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CompletedNCT01114737Updated Feb 1, 2016Results posted

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

A Phase 3 interventional study of Sapropterin dihydrochloride and Placebo in Phenylketonuria, sponsored by BioMarin Pharmaceutical. Completed at 36 sites in 2 countries. Open to participants aged 8 Years to 65 Years. Per ClinicalTrials.gov, last updated 2016-02-01.

Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
206
Allocation
Randomized
Ages
8 Years to 65 Years
Sex
All
01

Study summary

This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.

Read the detailed description

Phenylketonuria (PKU) results from deficient phenylalanine hydroxylase (PAH) activity and leads to toxic phenylalanine (Phe) accumulation in patients with PKU causing mental retardation, microcephaly, delayed speech, seizures, psychiatric symptoms and behavioral abnormalities. Although for most PKU patients early initiation of dietary treatment prevents severe complications, discontinuation of dietary restrictions at an early age is associated with poor cognitive development and neuropsychiatric disorders are present even in early-treated and well controlled PKU patients.

This study, PKU-016, will be conducted in PKU patients to evaluate the therapeutic effects of sapropterin dihydrochloride on the symptoms of attention deficit hyperactivity disorder (ADHD), depression, and anxiety.

02

Conditions studied

  • Phenylketonuria

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Keywords

  • Phenylketonuria
  • PKU
  • Kuvan
  • Sapropterin dihydrochloride
  • Neuropsychiatric disorder
  • ADHD
03

In context

Phenylketonurias

183 studies on the registry are indexed under Phenylketonurias; 38 are open to participants now.

This study's enrollment of 206 is above the median of 25 across 114 interventional studies indexed under Phenylketonurias.

Browse Phenylketonurias studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 110 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
8 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • ≥ 8 years of age
  • Confirmed diagnosis of PKU
  • Willing to continue current diet (typical diet for the 3 months prior to study entry) unchanged while participating in the study
  • Willing and able to provide written, signed informed consent or in the case of subjects under the age of 18, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
  • Sexually active subjects must be willing to use an acceptable method of contraception while participating in the study and for at least 30 days following the last dose of sapropterin dihydrochloride
  • Females of childbearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to screening, or have had total hysterectomy.
  • Willing and able to comply with all study procedure

Exclusion criteria

Exclusion Criteria:

  • Has known hypersensitivity to sapropterin dihydrochloride or its excipients
  • Subject breastfeeding at screening or planning to become pregnant (subject or partner) at any time during the study
  • Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to the completion of all scheduled study assessments
  • Received sapropterin dihydrochloride within 16 weeks of randomization
  • Have initiated or adjusted medication for treatment of ADHD, depression, or anxiety ≤ 8 weeks prior to randomization
  • Taking medication known to inhibit folate synthesis (eg, methotrexate)
  • Any condition requiring treatment with levodopa or any PDE-5 inhibitor
  • Concurrent disease or condition that would interfere with study participation, compliance or safety as determined by the Investigator
  • Any condition that, in the view of the Investigator, places the subject at high risk of poor treatment compliance or of not completing the study
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
206 participants (actual)

Study arms

  • Experimental
    Sapropterin dihydrochloride

    Drug: Sapropterin dihydrochloride

  • Placebo comparator
    Tablet without active ingredient

    Drug: Placebo

Interventions

  • DrugSapropterin dihydrochloride

    A dose of 20 mg/kg/day will be administered. Route of administration is oral (intact).

    Also known as: Kuvan, Phenoptin, BH4, 6R BH4

  • DrugPlacebo

    Placebo (tablet without active ingredient) is dosed once/day for the first 13 weeks of the study.

06

What researchers measure

Primary outcomes

  1. Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13

    Effects of 6R-BH4 on symptoms of ADHD in PKU subjects who had symptoms of ADHD at screening in the subjects that had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

    Time frame: Baseline to Week 13

  2. Number of Participants With a Score of 1 or 2 in Global Function Evaluation (CGI-I) From Baseline to Week 13.

    Effects of 6R-BH4 on global function in PKU subjects in subjects that had a blood Phe level reduction after treatment with 6R-BH4 at screening. The CGI-I is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse.

    Time frame: 13 weeks

Secondary outcomes

  1. Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 13

    Effects of 6R-BH4 on symptoms of anxiety in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

    Time frame: Baseline to Week 13

  2. Change in Hamilton Depression Rating Scale (HAM-D) Score From Baseline to Week 13

    Effects of 6R-BH4 on symptoms of depression in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

    Time frame: Baseline to Week 13

  3. Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 13

    Effects of 6R-BH4 on global function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

    Time frame: Baseline to Week 13

  4. Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 13

    Effects of 6R-BH4 on executive function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Baseline to Week 13

  5. Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 13

    Effects of 6R-BH4 on executive function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Baseline to Week 13

  6. Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on ADHD through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

    Time frame: Week 13 to Week 26

  7. Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on anxiety through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

    Time frame: Week 13 to Week 26

  8. Change in Hamilton Depression Rating Scale (HAM-D) Score From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on depression through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

    Time frame: Week 13 to Week 26

  9. Change in Clinical Global Impression-Severity (CGI-S) From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on global function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

    Time frame: Week 13 to Week 26

  10. Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Week 13 to Week 26

  11. Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Week 13 to Week 26

    Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Week 13 to Week 26

  12. Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on ADHD through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

    Time frame: Baseline to Week 26

  13. Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on anxiety through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

    Time frame: Baseline to Week 26

  14. Change in Hamilton Rating Scale For Depression (HAM-D) Score From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on depression through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

    Time frame: Baseline to Week 26

  15. Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on global function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

    Time frame: Baseline to Week 26

  16. Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Baseline to Week 26

  17. Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 26

    Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

    Time frame: Baseline to Week 26

07

Results

Posted Feb 1, 2016
Limitations and caveats
As specified in the SAP, no multiplicity adjustment was made in the analyses; therefore, the family-wise error rate was not controlled in this study

Participant flow

Randomized Treatment Period
Participant flow — Randomized Treatment Period
MilestonePlacebo6R-BH4 20 mg/kg/Day
Started10898
Completed10597
Not completed31
Withdrew: Withdrawal by subject10
Withdrew: Lost to follow-up11
Withdrew: Pregnancy10
Open-Label Treatment Period
Participant flow — Open-Label Treatment Period
MilestonePlacebo6R-BH4 20 mg/kg/Day
Started10597
Completed10095
Not completed52
Withdrew: Withdrawal by subject11
Withdrew: Lost to follow-up31
Withdrew: Difficult personal events10

Outcome measures

PrimaryChange in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13

Effects of 6R-BH4 on symptoms of ADHD in PKU subjects who had symptoms of ADHD at screening in the subjects that had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · units on a scale
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13
units on a scaleResponders in Placebo Arm With ADHD SymptomsResponders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 13-4.9 (-8.9 to -0.9)-9.1 (-13.5 to -4.7)
Statistical analysis
  • Responders in Placebo Arm With ADHD Symptoms vs Responders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms · ANCOVA · p = 0.085 · Mean difference (final values): -4.2 · 95% CI -8.9 to 0.6Adjusted for baseline ADHD-RS/ASRS total score, age group, and ADHD medication.
SecondaryChange in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 13

Effects of 6R-BH4 on symptoms of anxiety in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · units on a scale
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 13
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 13-3.6 (-5.4 to -1.9)-3.2 (-5.1 to -1.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.669 · Mean difference (final values): 0.4 · 95% CI -1.5 to 2.3Adjusted for baseline HAMA Anxiety Scale Total Score, age group, ADHD symptom, and ADHD medication.
SecondaryChange in Hamilton Depression Rating Scale (HAM-D) Score From Baseline to Week 13

Effects of 6R-BH4 on symptoms of depression in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · units on a scale
Change in Hamilton Depression Rating Scale (HAM-D) Score From Baseline to Week 13
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Depression Rating Scale (HAM-D) Score From Baseline to Week 13-2.5 (-3.9 to -1.1)-2.1 (-3.6 to -0.6)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.588 · Mean difference (final values): 0.4 · 95% CI -1.1 to 1.9Adjusted for Baseline HAM-D Rating Scale Total Score, age group, ADHD symptom, and ADHD medication.
SecondaryChange in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 13

Effects of 6R-BH4 on global function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · units on a scale
Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 13
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 13-0.5 (-0.8 to -0.2)-0.6 (-0.9 to -0.3)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.531 · Mean difference (final values): -0.1 · 95% CI -0.4 to 0.2Adjusted for baseline CGI-Severity Response, age group, ADHD symptom, and ADHD medication.
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 13

Effects of 6R-BH4 on executive function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 13
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 13-8.1 (-12.6 to -3.6)-9.1 (-14.6 to -3.5)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.661 · Mean difference (final values): -1.0 · 95% CI -5.5 to 3.6Adjusted for Baseline BRIEF Adult-GEC T Score, ADHD symptom, and ADHD medication.
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 13

Effects of 6R-BH4 on executive function in PKU subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Baseline to Week 13
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 13
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 13-0.7 (-4.0 to 2.7)-4.8 (-8.0 to -1.6)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.034 · Mean difference (final values): -4.1 · 95% CI -7.9 to -0.3Adjusted for Baseline BRIEF Parent-GEC T Score, ADHD symptom, and ADHD medication.
SecondaryChange in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on ADHD through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · units on a scale
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Week 13 to Week 26
units on a scaleResponders in Placebo Arm With ADHD SymptomsResponders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Week 13 to Week 26-4.0 (-8.2 to 0.2)-1.4 (-6.1 to 3.3)
Statistical analysis
  • Responders in Placebo Arm With ADHD Symptoms vs Responders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms · ANCOVA · p = 0.312 · Mean difference (final values): 2.6 · 95% CI -2.6 to 7.8Adjusted for Week 13 ADHD RS/ASRS Total Score, age group, and ADHD medication.
SecondaryChange in Hamilton Anxiety Rating Scale (HAM-A) Score From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on anxiety through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · units on a scale
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Week 13 to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Week 13 to Week 260.1 (-1.7 to 1.8)-0.5 (-2.3 to 1.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.590 · Mean difference (final values): -0.5 · 95% CI -2.4 to 1.4Adjusted for Week 13 HAMA Anxiety Rating Scale Total Score, age group, ADHD symptom, and ADHD medication.
SecondaryChange in Hamilton Depression Rating Scale (HAM-D) Score From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on depression through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · units on a scale
Change in Hamilton Depression Rating Scale (HAM-D) Score From Week 13 to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Depression Rating Scale (HAM-D) Score From Week 13 to Week 260.1 (-1.3 to 1.5)0.5 (-1.1 to 2.0)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.636 · Mean difference (final values): 0.4 · 95% CI -1.2 to 1.9Adjusted for Week 13 HAMD Rating Scale Total Score, age group, ADHD symptom, and ADHD
SecondaryChange in Clinical Global Impression-Severity (CGI-S) From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on global function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · units on a scale
Change in Clinical Global Impression-Severity (CGI-S) From Week 13 to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Clinical Global Impression-Severity (CGI-S) From Week 13 to Week 26-0.0 (-0.3 to 0.3)0.1 (-0.2 to 0.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.510 · Mean difference (final values): 0.1 · 95% CI -0.2 to 0.5Adjusted for Week 13 Global Impression-Severity Score, ADHD symptom, and ADHD medication.
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Week 13 to Week 26
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Week 13 to Week 26-0.7 (-4.5 to 3.1)0.9 (-3.7 to 5.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.395 · Mean difference (final values): 1.6 · 95% CI -2.1 to 5.2Adjusted for Week 13 BRIEF Adult-GEC T Score, ADHD symptom, and ADHD medication.
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Week 13 to Week 26

Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Week 13 to Week 26
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Week 13 to Week 26
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Week 13 to Week 26-1.3 (-4.3 to 1.7)0.0 (-2.7 to 2.7)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.443 · Mean difference (final values): 1.3 · 95% CI -2.1 to 4.7Adjusted for Week 13 BRIEF Parent-GEC T Score, ADHD symptom, and ADHD medication.
SecondaryChange in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on ADHD through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The total ADHD-RS score and the corrected total ARS score range from 0 to 54, with higher scores corresponding to worse severity of ADHD symptoms.

Time frame:
Baseline to Week 26
Reported as:
Least squares mean · units on a scale
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 26
units on a scaleResponders in Placebo Arm With ADHD SymptomsResponders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms
Change in Attention-Deficit Hyperactivity Disorder Rating Scale-IV (ADHD-RS) / Adult ADHD Self-Report Scale (ASRS) Total Score From Baseline to Week 26-9.5 (-14.2 to -4.8)-9.3 (-14.5 to -4.2)
Statistical analysis
  • Responders in Placebo Arm With ADHD Symptoms vs Responders in 6R-BH4 20 mg/kg/Day Arm With ADHD Symptoms · ANCOVA · p = 0.953 · Mean difference (final values): 0.2 · 95% CI -5.5 to 5.8Adjusted for Baseline ADHD-RS/ASRS Total Score, ADHD symptom, and ADHD medication.
SecondaryChange in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on anxiety through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-A Score is a total score ranging from 0 to 56 with higher scores corresponding to worse severity of anxiety symptoms. The HAM-A has 14 items, each measuring specific anxiety symptom clusters. Each item is given a 5-point-score as: 0, absent; 1, mild; 2, moderate; 3, severe; or 4, incapacitating.

Time frame:
Baseline to Week 26
Reported as:
Least squares mean · units on a scale
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Anxiety Rating Scale (HAM-A) Score From Baseline to Week 26-3.9 (-5.8 to -2.0)-4.2 (-6.3 to -2.2)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.733 · Mean difference (final values): -0.4 · 95% CI -2.4 to 1.7Adjusted for Baseline Hamilton Anxiety Rating Scale (HAM-A) Score, ADHD symptom, and ADHD medication.
SecondaryChange in Hamilton Rating Scale For Depression (HAM-D) Score From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on depression through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. HAM-D Score is a total score ranging from 0 to 48 with higher scores corresponding to worse severity of depression. The HAM-D is a 17-item depression rating scale. Nine of the items are scored on a 5-point scale as: 0, absence of depressive symptom being measured; 1, doubt concerning the presence of the symptom; 2, mild symptoms; 3, moderate symptoms; or 4, severe symptoms. The remaining 8 items are scored on a 3-point scale as: 0, absence; 1, doubt on the presence of the symptom; or 2, clear presence of symptoms.

Time frame:
Baseline to Week 26
Reported as:
Least squares mean · units on a scale
Change in Hamilton Rating Scale For Depression (HAM-D) Score From Baseline to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Hamilton Rating Scale For Depression (HAM-D) Score From Baseline to Week 26-2.6 (-4.1 to -1.0)-2.0 (-3.7 to -0.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.522 · Mean difference (final values): 0.5 · 95% CI -1.1 to 2.2Adjusted for Baseline Hamilton Rating Scale For Depression (HAM-D) Score, ADHD symptom, and ADHD medication.
SecondaryChange in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on global function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. CGI-S is a 7-point scale that requires the clinician to rate the severity of the subject's mental illness at the time of assessment, relative to clinician's past experience with subjects who have the same diagnosis. Considering total clinical experience, a subject is assessed on the severity of mental illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 6, severely ill; or 7, among the most extremely ill.

Time frame:
Baseline to Week 26
Reported as:
Mean · units on a scale
Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 26
units on a scaleResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Clinical Global Impression-Severity (CGI-S) From Baseline to Week 26-0.6 (-0.9 to -0.3)-0.5 (-0.8 to -0.2)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.564 · Mean difference (final values): 0.1 · 95% CI -0.2 to 0.4Adjusted for Baseline Clinical Global Impression-Severity (CGI-S), ADHD symptom, and ADHD medication.
PrimaryNumber of Participants With a Score of 1 or 2 in Global Function Evaluation (CGI-I) From Baseline to Week 13.

Effects of 6R-BH4 on global function in PKU subjects in subjects that had a blood Phe level reduction after treatment with 6R-BH4 at screening. The CGI-I is a 7-point scale that requires the clinician to assess how much the participant's illness has improved or worsened relative to a baseline state at the beginning of the intervention and rated as: 1=very much improved; 2=much improved; 3=minimally improved; 4=no change; 5=minimally worse; 6=much worse; 7=very much worse.

Time frame:
13 weeks
Reported as:
Number · Number of participants with scale 1 or 2
Number of Participants With a Score of 1 or 2 in Global Function Evaluation (CGI-I) From Baseline to Week 13.
Number of participants with scale 1 or 2Responders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Number of Participants With a Score of 1 or 2 in Global Function Evaluation (CGI-I) From Baseline to Week 13.1513
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · Cochran-Mantel-Haenszel · p = 0.67 · Relative risk: 0.87 · 95% CI 0.46 to 1.64Adjusted for age group, ADHD symptom, and ADHD medication
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Baseline to Week 26
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 26
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Adult-Global Executive Composite (GEC) T Score From Baseline to Week 26-7.1 (-12.6 to -1.6)-7.7 (-14.5 to -0.9)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.833 · Mean difference (final values): -0.6 · 95% CI -6.2 to 5.0Adjusted for Baseline BRIEF Adult-GEC T Score, ADHD symptom, and ADHD medication.
SecondaryChange in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 26

Durability of the therapeutic effect of 6R-BH4 on executive function through 26 weeks in subjects who had a blood Phe level reduction after treatment with 6R-BH4. The scoring for the GEC T Score is complex and is achieved using proprietary software designed to generate scores based on raw data collected. Higher scores suggest a higher level of dysfunction.

Time frame:
Baseline to Week 26
Reported as:
Least squares mean · T score
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 26
T scoreResponders in Placebo ArmResponders in 6R-BH4 20 mg/kg/Day Arm
Change in Behavior Rating Inventory of Executive Function (BRIEF) Parent-Global Executive Composite (GEC) T Score From Baseline to Week 26-2.6 (-6.2 to 1.0)-4.8 (-8.2 to -1.4)
Statistical analysis
  • Responders in Placebo Arm vs Responders in 6R-BH4 20 mg/kg/Day Arm · ANCOVA · p = 0.279 · Mean difference (final values): -2.2 · 95% CI -6.3 to 1.8Adjusted for Baseline BRIEF Parent-GEC T Score, ADHD symptom, and ADHD medication.

Adverse events

Collected over Study Period. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Randomized Treatment Period - Placebo—3/108 (2.8%)80/108 (74.1%)
Randomized Treatment Period - 6R-BH4—0/98 (0%)79/98 (80.6%)
Randomized Treatment Period - Overall—3/206 (1.5%)159/206 (77.2%)
Open-Label Treatment Period - Placebo-6R-BH4—2/104 (1.9%)79/104 (76%)
Open-Label Treatment Period - 6R-BH4—0/95 (0%)71/95 (74.7%)
Open-Label Treatment Period - Overall—2/199 (1%)150/199 (75.4%)
6R-BH4 Treatment Period - 6R-BH4—0/98 (0%)86/98 (87.8%)
6R-BH4 Treatment Period - Combined—2/202 (1%)165/202 (81.7%)
Most frequent serious events
Most frequent serious events
EventRandomized Treatment Period - PlaceboRandomized Treatment Period - 6R-BH4Randomized Treatment Period - OverallOpen-Label Treatment Period - Placebo-6R-BH4Open-Label Treatment Period - 6R-BH4Open-Label Treatment Period - Overall6R-BH4 Treatment Period - 6R-BH46R-BH4 Treatment Period - Combined
Animal biteInjury, poisoning and procedural complications0/1080/980/2061/1040/951/1990/981/202
Petit mal epilepsyNervous system disorders0/1080/980/2061/1040/951/1990/981/202
Necrotising fasciitisInfections and infestations1/1080/981/2060/1040/950/1990/980/202
ConcussionInjury, poisoning and procedural complications1/1080/981/2060/1040/950/1990/980/202
Amino acid level increasedInvestigations1/1080/981/2060/1040/950/1990/980/202
Most frequent other events
Showing 10 of 13
Most frequent other events
EventRandomized Treatment Period - PlaceboRandomized Treatment Period - 6R-BH4Randomized Treatment Period - OverallOpen-Label Treatment Period - Placebo-6R-BH4Open-Label Treatment Period - 6R-BH4Open-Label Treatment Period - Overall6R-BH4 Treatment Period - 6R-BH46R-BH4 Treatment Period - Combined
HeadacheNervous system disorders28/10825/9853/20616/10417/9533/19932/9848/202
NasopharyngitisInfections and infestations9/10811/9820/20612/10411/9523/19919/9831/202
Nasal congestionRespiratory, thoracic and mediastinal disorders11/1087/9818/2064/10412/9516/19917/9821/202
Oropharyngeal painRespiratory, thoracic and mediastinal disorders10/1086/9816/20611/10411/9522/19916/9827/202
DiarrhoeaGastrointestinal disorders4/10810/9814/2068/1044/9512/19913/9821/202
VomitingGastrointestinal disorders14/1084/9818/20612/1043/9515/1996/9818/202
NauseaGastrointestinal disorders10/1084/9814/20610/1047/9517/19910/9820/202
CoughRespiratory, thoracic and mediastinal disorders8/1087/9815/2068/1048/9516/19910/9818/202
Upper respiratory tract infectionInfections and infestations7/1084/9811/20610/1043/9513/1997/9817/202
Abdominal pain upperGastrointestinal disorders5/1084/989/2062/1047/959/1999/9811/202

Baseline characteristics

Age, Continuous
Age, Continuous(years)Placebo6R-BH4 20 mg/kg/DayTotal
Mean22.5 ± 10.4223.6 ± 12.6923.1 ± 11.54
Age, Customized
Age, Customized(participants)Placebo6R-BH4 20 mg/kg/DayTotal
<18 years434386
>=18 years6555120
Sex: Female, Male
Sex: Female, Male(Participants)Placebo6R-BH4 20 mg/kg/DayTotal
Female544195
Male5457111
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Placebo6R-BH4 20 mg/kg/DayTotal
Hispanic or Latino358
Not Hispanic or Latino10593198
Unknown or Not Reported000
Race/Ethnicity, Customized
Race/Ethnicity, Customized(participants)Placebo6R-BH4 20 mg/kg/DayTotal
American Indian or Alaska Native101
Asian112
Native Hawaiian or Other Pacific Islander000
Black or African American202
White10296198
Other213
Region of Enrollment
Region of Enrollment(participants)Placebo6R-BH4 20 mg/kg/DayTotal
Canada383169
United States7067137
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Study locations

36 sites
  • La Jolla, California, United States
  • Los Angeles, California, United States
  • Palo Alto, California, United States
  • San Francisco, California, United States
  • Aurora, Colorado, United States
  • Washington DC, District of Columbia, United States
  • Gainesville, Florida, United States
  • Tampa, Florida, United States
  • Atlanta, Georgia, United States
  • Chicago, Illinois, United States
  • Indianapolis, Indiana, United States
  • Boston, Massachusetts, United States
  • Minneapolis, Minnesota, United States
  • Albany, New York, United States
  • Buffalo, New York, United States
  • Rochester, New York, United States
  • Chapel Hill, North Carolina, United States
  • Cleveland, Ohio, United States
  • Portland, Oregon, United States
  • Hershey, Pennsylvania, United States
  • Philadelphia, Pennsylvania, United States
  • Pittsburgh, Pennsylvania, United States
  • Nashville, Tennessee, United States
  • Dallas, Texas, United States
  • Madison, Wisconsin, United States
  • Milwaukee, Wisconsin, United States
  • Calgary, Alberta, Canada
  • Edmonton, Alberta, Canada
  • Vancouver, British Columbia, Canada
  • Winnipeg, Manitoba, Canada
  • Halifax, Nova Scotia, Canada
  • Hamilton, Ontario, Canada
  • Kingston, Ontario, Canada
  • London, Ontario, Canada
  • Toronto, Ontario, Canada
  • Montreal, Quebec, Canada
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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 1, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01114737
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
May 3, 2010
Start date
Aug 2010
Primary completion
Mar 2013
Completion
Mar 2013
Results posted
Feb 1, 2016
Last update
Feb 1, 2016

Study contacts

Suyash Prasad, MD
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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