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CompletedNCT03771898EMBOLDENUpdated Jul 10, 2026Results posted

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

A Phase 2 interventional study of SHP611 in Metachromatic Leukodystrophy (MLD), sponsored by Shire. Completed at 30 sites in 14 countries. Open to participants aged 6 Months to 72 Months. Per ClinicalTrials.gov, last updated 2026-07-10.

Sponsored by Shire (part of Takeda) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
36
Allocation
Not applicable
Ages
6 Months to 72 Months
Sex
All
01

Study summary

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated.

Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.

02

Conditions studied

  • Metachromatic Leukodystrophy (MLD)
03

In context

Leukodystrophy, Metachromatic

49 studies on the registry are indexed under Leukodystrophy, Metachromatic; 11 are open to participants now.

This study's enrollment of 36 is above the median of 19 across 28 interventional studies indexed under Leukodystrophy, Metachromatic.

Browse Leukodystrophy, Metachromatic studies →

Lead sponsor

Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.

Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.

First submitted before Shire became part of Takeda.

Browse Shire studies →All Takeda studies →

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

04

Who can participate

Ages eligible
6 Months to 72 Months
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • The participant must have a documented diagnosis of MLD (Groups A-F):

    1. Low ASA activity in leukocytes (compared to laboratory normal range).
    2. Elevated sulfatides in urine.
  • The participant must have a gait disorder due to spastic ataxia or weakness attributable to MLD by the investigator and documented by a primary care physician or a specialist physician by 30 months of age (Groups A-C, and F), or be minimally symptomatic and greater than or equal to (> =) 6 to less than (\<) 18 months of age (Group D) or be early symptomatic and > =12 to \< 18 months of age (Group E). Participants in Group E must have neurological symptoms either documented by either a primary care physician or a specialist physician.
  • The participant's age at the time of , must be: Group A: 18 to 48 months of age; Group B: 18 to 72 months of age; Group C: 18 to 72 months of age; Group D: >= 6 to \< 18 months of age; Group E: > = 12 to \< 18 months of age; Group F: 18 to 72 months of age.
  • The participant's GMFC-MLD category at screening must be: Group A: GMFC-MLD category of 1 or 2; Group B: GMFC-MLD category of 3; Group C: GMFC-MLD category of 4; Group D: minimally symptomatic, >= 6 to \< 18 months of age, with the same arylsulfatase (ASA) allelic constitution as an older sibling with confirmed late infantile or juvenile onset MLD; Group E: early symptomatic, >= 12 to \< 18 months of age with a GMFC-MLD category of 1 or 2 with a history of achieving stable walking (defined as at least 1 month of independent walking); Group F: GMFC-MLD category of 5 or 6.
  • The participant and his/her parent/representative(s) must have the ability to comply with the clinical protocol.
  • Participant's parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the participant.

Exclusion criteria

Exclusion Criteria:

  • Multiple sulfatase disorder as determined by abnormal activity of another lysosomal sulfatase (based upon the reference laboratory's normal range) or a known genetic disorder other than MLD.
  • History of bone marrow transplant (BMT), hematopoietic stem cell transplantation (), or gene therapy; or undergoes BMT, HSCT, or gene therapy: at any point during the study.
  • Primary presentation of MLD was behavioral or cognitive symptoms (per investigator's clinical judgment); behavioral symptoms that are secondary to motor deficits (example [eg], tantrums in response to loss of motor skills) are not exclusionary.
  • The participant has any known or suspected to agents used for anesthesia or has history of difficult airway or potential for airway compromise.
  • Any other medical condition or serious comorbid illness that in the opinion of the investigator would preclude participation in the study.
  • Participants with laboratory, or vital sign abnormalities reflecting intercurrent illness that may compromise their safety during the trial should not be enrolled. Abnormal laboratory, vital sign and ECG results at screening should be reviewed with the Takeda medical monitor.
  • The participant is enrolled in another clinical study that involves use of any product (drug or device) within 30 days or 5 half-lives (whichever is longer) prior to study enrollment or at any time during the study.
  • The participant has had prior exposure to SHP611.
  • The participants must weigh > 11 pound (lbs) (5 kilograms [kg]).

  • The participant has a condition that is as described in the SOPH-A-PORT Mini S IDDD Instructions for Use (IFU)

    1. The participant has had, or may have, an allergic reaction to the materials of construction.
    2. The participant has shown an intolerance to an implanted device.
    3. The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port.
    4. The participant's drug therapy requires substances known to be incompatible with the materials of construction.
    5. The participant has a known or suspected local or general infection.
    6. The participant is at risk of abnormal bleeding due to a medical condition or therapy.
    7. The participant has one or more spinal abnormalities that could complicate safe implantation or fixation.
    8. The participant has a functioning Cerebro spinal fluid(CSF) shunt device .

Matched External Control Participants for Group A from Global Leukodystrophy Initiative of Metachromatic Leukodystrophy (GLIA-MLD) The matched external control group must have data for at least baseline gross motor function evaluation. Selection of the external control participants from GLIA-MLD will follow a set of criteria as similar as possible to the inclusion criteria for Group A in the SHP611-201 study protocol.

A filtering process will be applied to select the external control participants from the GLIA-MLD database, by meeting all of the following 3 filtering criteria:

  1. Filtering criterion 1: requiring documented diagnosis of MLD, based on

    • low arylsulfatase A (ASA) activity in leukocytes AND elevated sulfatides in urine. OR
    • biallelic variants in arylsulfatase A gene (ARSA) AND (either low ASA activity in leukocytes OR elevated sulfatides in urine).
  2. Filtering criterion 2: requiring documented gait disorder. Participants will be considered qualifying if they present with a gait disorder before 2.5 years (30 months) of age and have a medical record reporting a gait abnormality including, but not limited to, the following terms: ataxia, spasticity, and hyper/hypotonia.
  3. Filtering criterion 3: participants will be considered qualifying if they have at least 1 clinical encounter occurring between the age of 18 to 48 months with a GMFC-MLD category either 1 or 2.
Terms in these criteria, in plain language(6)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

ECG (electrocardiogram)

A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
36 participants (actual)

Study arms

  • Experimental
    SHP611

    Participants will receive 150 milligrams (mg) of SHP611 intrathecally (IT) via intrathecal drug delivery device (IDDD) or lumbar puncture (LP) once weekly for 106 weeks in six groups (Group A, B, C, D, E, and F) based on participant's age and motor dysfunction.

    Drug: SHP611

Interventions

  • DrugSHP611

    Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks.

    Also known as: HGT-1110, TAK-611, recombinant human arylsulfatase A [rhASA]

06

What researchers measure

Primary outcomes

  1. Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control

    Loss of locomotion was estimated using interval censoring survival analysis. Survival probability free of loss of locomotion based on GMFC-MLD was estimated up to Week 106 (or two years), with associated 2-sided 95 percent (%) confidence interval (CI). GMFC-MLD scale consists of 7 categories, scores ranging from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control). Higher scores mean a worse outcome. The data was reported in terms of Mean as survival function was quantified using a weighted average of percentage of participants not reaching the event of interest, with weights derived from the relative size of treated and control units in the strata used for the stratified log-rank test in the primary analysis.

    Time frame: Baseline up to Week 106

Secondary outcomes

  1. Group A: Number of Participants Who Maintained Their Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106 Compared With Matched External Control Group Data

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  2. Number of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  3. Group A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  4. Group A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  5. Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  6. Percent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  7. Group A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  8. Time to Unreversed Decline From Baseline in GMFM-88 Total Score of Greater Than (>) 20 Points or Unreversed Decline to Less Than (<) 40 Points Whichever Occurs First

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  9. Change From Baseline in GMFM-88 Total Score at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  10. Group A: Number of Participants With GMFM-88 Total Score Decrease of <= 20 Points From Baseline and a Total Score >=40 Points at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  11. Number of Participants With Change From Baseline in Expressive Language Evaluated by Using the Expressive Language Function Classification in Metachromatic Leukodystrophy (ELFC-MLD) Scale Categories at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline, Week 106

  12. Number of Participants With Treatment-emergent Adverse Event (TEAEs)

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: From start of study drug administration up to Week 106

  13. Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Values at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  14. Number of Participants With Clinically Significant Change From Baseline in Physical Examination Findings at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  15. Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Values at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  16. Number of Participants With Clinically Significant Change From Baseline in Cerebrospinal Fluid (CSF) Laboratory Parameters at Week 106

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  17. Number of Participants With Anti-Drug Antibody (ADA) Response to SHP611

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  18. Number of Participants With IDDD Implantations

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  19. Number of Participants With Any IDDD Related Malfunctions

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  20. IDDD Longevity as Assessed by Time to IDDD Failure

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

  21. Number of Participants With TEAE Related to IDDD and Surgical Procedure

    Data collection is ongoing and detailed reporting will be available after the study completion date.

    Time frame: Baseline up to Week 106

07

Results

Posted Jun 6, 2024

Participant flow

The study was conducted at 19 investigational sites. A total of 36 participants were enrolled in this two-period study (primary treatment and extension periods) of which 35 received SHP611 or had intrathecal drug delivery device (IDDD) implanted. Data was reported till the primary treatment period. The study is still ongoing, additional data will be reported after the completion of the study. Study consisted of six groups (Group A,B,C,D, E, F) based on participant's age and motor dysfunction.

Participant flow — Overall Study
MilestoneGroup A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611GLIA-MLD Matched External Control for Group A
Started1556933
Completed123590
Not completed321033
Withdrew: Adverse event10000
Withdrew: Lost to follow-up11100
Withdrew: Withdrawal by parent/guardian11000
Withdrew: Ongoing (participants in glia-mld, [part of glia-ctn study])000033

Outcome measures

PrimaryPercent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control

Loss of locomotion was estimated using interval censoring survival analysis. Survival probability free of loss of locomotion based on GMFC-MLD was estimated up to Week 106 (or two years), with associated 2-sided 95 percent (%) confidence interval (CI). GMFC-MLD scale consists of 7 categories, scores ranging from 0 (walking without support with quality of performance normal for age) to 6 (loss of any locomotion as well as loss of any head and trunk control). Higher scores mean a worse outcome. The data was reported in terms of Mean as survival function was quantified using a weighted average of percentage of participants not reaching the event of interest, with weights derived from the relative size of treated and control units in the strata used for the stratified log-rank test in the primary analysis.

Time frame:
Baseline up to Week 106
Reported as:
Mean · percent probability
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control
percent probabilityGroup A, SHP611GLIA-MLD Matched External Control for Group A
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control7.1 (0 to 20.2)9.4 (0 to 24.4)
Statistical analysis
  • Group A, SHP611 vs GLIA-MLD Matched External Control for Group A · Log Rank · p = =0.585 (One-sided, over time intervals during entire follow-up. Stratified generalized log-rank test was used, where matching identification created from matching process in SAS PSMATCH Procedure used as strata.) · Difference in survival probability (%): -2.2 · 95% CI -22.2 to 17.7For the shared time interval up to Week 106 (or two years).
SecondaryGroup A: Number of Participants Who Maintained Their Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106 Compared With Matched External Control Group Data

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Change From Baseline in Gross Motor Function Evaluated by Using the GMFC-MLD at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryGroup A: Number of Participants With Decline From Baseline in GMFC-LMD of More Than 2 Categories

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryGroup A: Time to Decline From Baseline in GMFC-MLD of More Than 2 Categories

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryChange From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryPercent Change From Baseline in Cerebrospinal Fluid (CSF) Sulfatides Levels at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryGroup A: Number of Participants With Maintenance of Gross Motor Function at Week 106 Using Gross Motor Function Measure 88 (GMFM-88) Total Score

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryTime to Unreversed Decline From Baseline in GMFM-88 Total Score of Greater Than (>) 20 Points or Unreversed Decline to Less Than (<) 40 Points Whichever Occurs First

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryChange From Baseline in GMFM-88 Total Score at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryGroup A: Number of Participants With GMFM-88 Total Score Decrease of <= 20 Points From Baseline and a Total Score >=40 Points at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Change From Baseline in Expressive Language Evaluated by Using the Expressive Language Function Classification in Metachromatic Leukodystrophy (ELFC-MLD) Scale Categories at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline, Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Treatment-emergent Adverse Event (TEAEs)

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
From start of study drug administration up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Values at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Clinically Significant Change From Baseline in Physical Examination Findings at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Values at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Clinically Significant Change From Baseline in Cerebrospinal Fluid (CSF) Laboratory Parameters at Week 106

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Anti-Drug Antibody (ADA) Response to SHP611

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With IDDD Implantations

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With Any IDDD Related Malfunctions

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryIDDD Longevity as Assessed by Time to IDDD Failure

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

SecondaryNumber of Participants With TEAE Related to IDDD and Surgical Procedure

Data collection is ongoing and detailed reporting will be available after the study completion date.

Time frame:
Baseline up to Week 106

Results for this outcome have not been posted.

Adverse events

Collected over From start of study drug administration up to Week 106. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Group A, SHP6110/15 (0%)14/15 (93.3%)15/15 (100%)
Group B, SHP6111/5 (20%)4/5 (80%)5/5 (100%)
Group C, SHP6110/6 (0%)5/6 (83.3%)6/6 (100%)
Group F, SHP6110/9 (0%)9/9 (100%)9/9 (100%)
Most frequent serious events
Showing 10 of 75
Most frequent serious events
EventGroup A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611
SeizureNervous system disorders3/151/54/65/9
Status epilepticusNervous system disorders2/150/53/62/9
PyrexiaGeneral disorders4/151/52/64/9
DysphagiaGastrointestinal disorders3/152/51/60/9
PainGeneral disorders1/152/50/60/9
Febrile convulsionNervous system disorders2/150/52/60/9
PneumoniaInfections and infestations1/151/51/63/9
Respiratory syncytial virus infectionInfections and infestations0/150/50/63/9
Device failureProduct Issues4/150/50/61/9
SepsisInfections and infestations0/150/50/62/9
Most frequent other events
Showing 10 of 360
Most frequent other events
EventGroup A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611
PyrexiaGeneral disorders13/153/54/69/9
VomitingGastrointestinal disorders8/152/53/68/9
ConstipationGastrointestinal disorders12/153/54/65/9
Muscle spasticityNervous system disorders11/153/52/66/9
COVID-19Infections and infestations10/150/53/61/9
CSF protein increasedInvestigations7/151/53/66/9
Febrile convulsionNervous system disorders0/151/54/60/9
GastroenteritisInfections and infestations5/151/54/61/9
Gastrooesophageal reflux diseaseGastrointestinal disorders5/153/52/63/9
Salivary hypersecretionGastrointestinal disorders3/153/53/63/9

Baseline characteristics

Age, Continuous
Age, Continuous(months)Group A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611GLIA-MLD Matched External Control for Group ATotal
Mean18.27 ± 4.00820.80 ± 4.65817.67 ± 7.44817.44 ± 4.74614.76 ± 3.75116.59 ± 4.692
Sex: Female, Male
Sex: Female, Male(Participants)Group A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611GLIA-MLD Matched External Control for Group ATotal
Female114241536
Male41451832
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Group A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611GLIA-MLD Matched External Control for Group ATotal
Hispanic or Latino311128
Not Hispanic or Latino8428931
Unknown or Not Reported40302229
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Group A, SHP611Group B, SHP611Group C, SHP611Group F, SHP611GLIA-MLD Matched External Control for Group ATotal
American Indian or Alaska Native000011
Asian200114
Native Hawaiian or Other Pacific Islander000000
Black or African American100113
White125571342
More than one race000000
Unknown or Not Reported00101718
08

Study locations

30 sites
  • Los Angeles Biomedical Research Institute at Harbor-UCLA
    Torrance, California 90502, United States
  • Childrens Hospital Colorado
    Aurora, Colorado 80045, United States
  • Rare Disease Research, LLC
    Atlanta, Georgia 30303, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • University of Iowa Stead Family Children's Hospital
    Iowa City, Iowa 52242, United States
  • Mayo Clinic - PPDS
    Rochester, Minnesota 55905, United States
  • New York University Langone Medical Center
    New York, New York 10016, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Children's Hospital of Pittsburgh
    Pittsburgh, Pennsylvania 15224, United States
  • University of Utah
    Salt Lake City, Utah 84108, United States
  • Hospital Universitario Austral - PIN
    Ciudad Autónoma Buenos Aires, Buenos Aires B1629AHJ, Argentina
  • UZ Antwerpen
    Edegem, 2650, Belgium
  • Hospital de Clínicas de Porto Alegre
    Porto Alegre, 90035-903, Brazil
  • Stollery Children's Hospital University of Alberta
    Edmonton, Alberta T6G 2R7, Canada
  • British Columbia Children's Hospital
    Vancouver, British Columbia V6H 3V4, Canada
  • Hospital for Sick Children
    Toronto, Ontario M5G 1X8, Canada
  • Montreal Children's Hospital
    Montreal, Quebec H3H 1P3, Canada
  • Hôpital Bicêtre - Paris Sud
    Le Kremlin-Bicêtre, 94275, France
  • CHU Lenval
    Nice, 06200, France
  • Universitätsklinikum Hamburg Eppendorf
    Hamburg, 20246, Germany
  • Universitätsklinikum Tübingen
    Tübingen, 72076, Germany
  • Attikon University General Hospital
    Chaïdári, Attica 124 62, Greece
  • Tel Aviv Sourasky Medical Center
    Tel Aviv, 64239, Israel
  • IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
    Roma, 165, Italy
  • Kanazawa University Hospital
    Kanazawa, 920-8641, Japan
  • VU Medisch Centrum
    Amsterdam, 1081 HV, Netherlands
  • Hospital Universitario Cruces
    Barakaldo, Vizcaya 48903, Spain
  • Hospital Vall d'Hebrón
    Barcelona, 8035, Spain
  • Birmingham Children's Hospital NHS Foundation Trust
    Birmingham, B4 6NH, United Kingdom
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report

Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement

Data sharing details ↗ · vivli.org

10

Updates

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

Key details

Study ID
NCT03771898
Lead sponsor
Shire
Collaborators
Takeda Development Center Americas, Inc.
Responsible party
Sponsor
First posted
Dec 11, 2018
Start date
May 13, 2019
Primary completion
Mar 8, 2023
Completion
May 6, 2026
Results posted
Jun 6, 2024
Last update
Jul 10, 2026

Study contacts

Study Director
study director · Shire

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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