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TerminatedNCT02462759EMBRACEUpdated Feb 17, 2021Results posted

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

A Phase 2 interventional study of Nusinersen and Sham Procedure in Spinal Muscular Atrophy, sponsored by Biogen. Terminated at 7 sites in 2 countries. Per ClinicalTrials.gov, last updated 2021-02-17.

Sponsored by Biogen · Phase 2, Interventional, and Treatment

Why this study was terminated
The study was terminated early to roll over participants to open label extension study NCT02594124.
Phase
Phase 2
Study type
Interventional
Enrollment
21
Allocation
Randomized
Sex
All
01

Study summary

The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.

Read the detailed description

Part 2 is an Open Label extension phase.

02

Conditions studied

  • Spinal Muscular Atrophy

Keywords

  • EMBRACE
  • SMA
03

In context

Muscular Atrophy

494 studies on the registry are indexed under Muscular Atrophy; 94 are open to participants now.

This study's enrollment of 21 is below the median of 33 across 335 interventional studies indexed under Muscular Atrophy.

Browse Muscular Atrophy studies →

Lead sponsor

Biogen is the lead sponsor of 494 studies on the registry; 22 are open to participants now.

Of its 98 completed or terminated interventional studies of FDA-regulated products, 49 (50%) have results posted.

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

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

  • Genetic documentation of 5q SMA homozygous gene deletion, mutation, or compound heterozygote.
  • Onset of clinical signs and symptoms consistent with SMA at ≤6 months of age and have documentation of 3 SMN2 copies OR onset of clinical signs and symptoms consistent with SMA at ≤6 months of age, >7 months of age (211 days) at screening, and have documentation of 2 SMN2 copies OR onset of clinical signs and symptoms consistent with SMA at >6 months of age, are ≤18 months of age at screening, and have documentation of 2 or 3 SMN2 copies.
  • Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedures.
  • Medical care, such as routine immunizations meets and is expected to continue to meet guidelines set out in the Consensus Statement for Standard of Care in SMA, in the opinion of the Investigator.
  • Participants with 2 SMN2 copies must reside within approximately 9 hours' ground-travel distance from a participating study site for the duration of the study.

Key Exclusion Criteria:

  • Meets additional study related criteria.
  • Any previous exposure to ISIS 396443; previous dosing in this study or previous studies with ISIS 396443.
  • Signs or symptoms of SMA present at birth or within the first week after birth.
  • Ventilation for ≥16 hours per day continuously for >21 days at screening.
  • Permanent tracheostomy, implanted shunt for CSF drainage, or implanted central nervous system (CNS) catheter at screening.
  • History of brain or spinal cord disease that would interfere with the LP procedure, CSF circulation, or safety assessments.
  • Hospitalization for surgery (e.g., scoliosis surgery), pulmonary event, or nutritional support within 2 months prior to screening, or hospitalization for surgery planned during the study.
  • Clinically significant abnormalities in hematology or clinical chemistry parameters or Electrocardiogram (ECG), as assessed by the Investigator.
  • Treatment with an investigational drug for SMA (e.g., albuterol/salbutamol, riluzole, carnitine, sodium phenylbutyrate, valproate, hydroxyurea), biological agent, or device within 30 days prior to screening. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation.

For Part 2 only:

To be eligible to participate in Part 2 of this study, participants must meet the following eligibility criteria at the time of consent to participate in Part 2:

Participation in Part 1 and completion of the End of Part 1 Evaluation assessments.

Ability of parent(s) or legal guardian(s) to understand the purpose and risks of the study and to provide signed and dated informed consent on the Part 2 informed consent form (ICF) and authorization to use confidential health information in accordance with national and local participant privacy regulations.

Able to complete all study procedures, measurements, and visits, and parent or legal guardian/participant has adequately supportive psychosocial circumstances, in the opinion of the Investigator.

Participants will be excluded from the Part 2 if they meet the following exclusion criterion at the time of consent into Part 2 of the study:

Any significant change in clinical status, including laboratory tests that, in the opinion of the Investigator, would make them unsuitable to participate in Part 2. The Investigator must reassess the subject's medical fitness for participation and consider any diseases that would preclude treatment.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
21 participants (actual)

Study arms

  • Experimental
    Nusinersen

    Administered by intrathecal injection.

    Drug: Nusinersen

  • Sham comparator
    Sham Procedure

    Small needle prick on the lower back at the location where the IT injection is normally made.

    Procedure: Sham Procedure

Interventions

  • DrugNusinersen

    Administered by intrathecal injection.

    Also known as: BIIB058, ISIS SMNRx, ISIS 396443, Spinraza

  • ProcedureSham Procedure

    Small needle prick on the lower back at the location where the IT injection is normally made.

06

What researchers measure

Primary outcomes

  1. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly.

    Time frame: Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)

  2. Number of Participants With Change From Baseline in Clinical Laboratory Parameters

    Clinically significant changes in laboratory parameters were evaluated for assessing the safety of ISIS 396443.

    Time frame: Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)

  3. Number of Participants With Change From Baseline in Electrocardiograms (ECGs)

    Clinically significant changes in ECG measurements were evaluated for assessing the safety of ISIS 396443.

    Time frame: Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596

  4. Number of Participants With Change From Baseline in Vital Signs

    Clinically significant changes in vital signs were evaluated for assessing the safety of ISIS 396443. Vital signs that were assessed included resting systolic and diastolic blood pressure, pulse rate, respiratory rate, temperature, pulse oximetry, and transcutaneous carbon dioxide.

    Time frame: Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596

  5. Change From Baseline in Head Circumference

    Participants were analyzed for change in growth parameter of head circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the head circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  6. Change From Baseline in Chest Circumference

    Participants were analyzed for change in growth parameter of chest circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the chest circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days\>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  7. Change From Baseline in Arm Circumference

    Participants were analyzed for change in growth parameter of arm circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the arm circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  8. Change From Baseline in Weight for Age

    Participants were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight for age percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  9. Change From Baseline in Weight

    Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  10. Change From Baseline in Head to Chest Circumference (HCC) Ratio

    Participants were analyzed for change in growth parameter of HCC to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the HCC circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  11. Change From Baseline in Body Length

    Participants were analyzed for change in growth parameter of body length to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the body length percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  12. Number of Participants With Change From Baseline in Neurological Examination Outcomes

    Neurological examinations included assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, reflexes, mood, speech/language and hearing.

    Time frame: Part 1: Baseline to Day 422; Part 2: Baseline to Day 596

  13. Number of Participants With Change From Baseline in Activated Partial Thromboplastin Time [aPTT]

    Activated partial thromboplastin time was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of aPTT at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of aPTT at baseline to high values postbaseline.

    Time frame: Part 2: Up to 1080 days

  14. Number of Participants With Change From Baseline in Partial Thromboplastin Time [PTT]

    PTT was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of PTT at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of PTT at baseline to high values postbaseline.

    Time frame: Part 2: Up to 1080 days

  15. Number of Participants With Change From Baseline in International Normalized Ratio [INR])

    INR was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of INR at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of INR at baseline to high values postbaseline.

    Time frame: Part 2: Up to 1080 days

  16. Number of Participants With Presence of Urine Total Protein Post-baseline

    Urine total protein was evaluated to assess safety.

    Time frame: Part 2: Up to 1080 days

Secondary outcomes

  1. Plasma Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study

    Study days were windowed for integrated analysis and labelled as follows: Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659.

    Time frame: Pre-dose on Days 64, 183, 540 and 659

  2. Plasma Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study

    Study days were windowed for integrated analysis and labelled as follows: Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

    Time frame: Pre-dose on Days 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138

  3. Cerebrospinal Fluid (CSF) Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study

    CSF samples were analyzed for ISIS 396443 concentrations in participants. Study days were windowed for integrated analysis and labelled as follows: Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540.

    Time frame: Pre-dose on Days 15, 29, 64, 183, 302, 422 and 540

  4. CSF Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study

    CSF samples were analyzed for ISIS 396443 concentrations in participants. Study days were windowed for integrated analysis and labelled as follows: Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018.

    Time frame: Pre-dose on Days 15, 29, 64, 183, 302, 422, 540, 659, 778, 898 and 1018

  5. Number of Participants With Plasma Antibodies to ISIS 396443

    Time frame: Part 2: Baseline to Day 596

07

Results

Posted Jan 27, 2020
Limitations and caveats
Despite the early termination of both Parts of the study, the data from this study is of quality and reliable.

Participant flow

Participants were recruited from sites in the US and Germany. Part 1 was terminated early as positive efficacy results were observed in interim analysis of study NCT02193074 and it was considered unethical to continue this part of study. Part 2 was also terminated early to rollover and continue to follow participants in study NCT02594124.

Part 1: Double Blind
Participant flow — Part 1: Double Blind
MilestoneSham Procedure (Part 1)ISIS 396443 (Part 1)ISIS 396443 (Part 2)
Started7140
Completed6140
Not completed100
Withdrew: Death100
Part 2: Open-Label Phase
Participant flow — Part 2: Open-Label Phase
MilestoneSham Procedure (Part 1)ISIS 396443 (Part 1)ISIS 396443 (Part 2)
Started0020
Completed0020
Not completed000

Outcome measures

PrimaryNumber of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A SAE is any untoward medical occurrence that at any dose results in death, life-threatening event, requires inpatient hospitalization, significant disability/incapacity or congenital anomaly.

Time frame:
Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)
Reported as:
Count of participants · Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
ParticipantsSham Procedure in Part 1ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
AEs6614
SAEs349
PrimaryNumber of Participants With Change From Baseline in Clinical Laboratory Parameters

Clinically significant changes in laboratory parameters were evaluated for assessing the safety of ISIS 396443.

Time frame:
Part 1 and 2: From first dose/sham procedure to end of study (up to 1080 days)
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Clinical Laboratory Parameters
ParticipantsSham Procedure in Part 1ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Number of Participants With Change From Baseline in Clinical Laboratory Parameters000
PrimaryNumber of Participants With Change From Baseline in Electrocardiograms (ECGs)

Clinically significant changes in ECG measurements were evaluated for assessing the safety of ISIS 396443.

Time frame:
Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Electrocardiograms (ECGs)
ParticipantsSham Procedure in Part 1ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Number of Participants With Change From Baseline in Electrocardiograms (ECGs)000
PrimaryNumber of Participants With Change From Baseline in Vital Signs

Clinically significant changes in vital signs were evaluated for assessing the safety of ISIS 396443. Vital signs that were assessed included resting systolic and diastolic blood pressure, pulse rate, respiratory rate, temperature, pulse oximetry, and transcutaneous carbon dioxide.

Time frame:
Part 1: Day 2, 29 and 422; Part 2: Day 1 to 596
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Vital Signs
ParticipantsSham Procedure in Part 1ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Number of Participants With Change From Baseline in Vital Signs000
PrimaryChange From Baseline in Head Circumference

Participants were analyzed for change in growth parameter of head circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the head circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · centimeter (cm)
Change From Baseline in Head Circumference
centimeter (cm)ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline50.8 ± 3.8347.3 ± 1.51
Change at Day 15-0.7 ± 3.340.1 ± 0.36
Change at Day 290.0 ± 2.760.3 ± 0.65
Change at Day 640.1 ± 2.640.5 ± 0.73
Change at Day 1830.3 ± 3.101.0 ± 1.12
Change at Day 3021.0 ± 3.281.6 ± 1.05
Change at Day 4220.9 ± 2.472.0 ± 1.24
Change at Day 5401.3 ± 1.972.5 ± 1.24
Change at Day 6591.5 ± 2.532.6 ± 1.37
Change at Day 778—2.8 ± 0.92
Change at Day 898—3.5 ± 1.05
Change at Day 1018—3.5 ± 2.06
Change at Day 1138—4.0 ± 0
PrimaryChange From Baseline in Chest Circumference

Participants were analyzed for change in growth parameter of chest circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the chest circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days\>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · cm
Change From Baseline in Chest Circumference
cmISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline51.3 ± 5.4546.9 ± 3.91
Change at Day 15-0.2 ± 3.86-0.4 ± 1.53
Change at Day 29-1.1 ± 2.700.2 ± 1.39
Change at Day 64-0.9 ± 3.500.2 ± 1.92
Change at Day 1830.2 ± 3.041.4 ± 2.02
Change at Day 3021.2 ± 3.071.6 ± 2.86
Change at Day 4220.5 ± 3.722.8 ± 2.73
Change at Day 5401.8 ± 2.423.8 ± 3.23
Change at Day 6592.9 ± 3.855.1 ± 3.29
Change at Day 778—5.5 ± 3.52
Change at Day 898—7.1 ± 3.04
Change at Day 1018—9.7 ± 4.92
Change at Day 1138—9.1 ± 0
PrimaryChange From Baseline in Arm Circumference

Participants were analyzed for change in growth parameter of arm circumference to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the arm circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · cm
Change From Baseline in Arm Circumference
cmISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline16.2 ± 1.1914.5 ± 1.85
Change at Day 15-0.4 ± 1.040.3 ± 0.61
Change at Day 29-0.5 ± 1.08-0.1 ± 0.54
Change at Day 64-0.3 ± 0.49-0.4 ± 0.89
Change at Day 1830.0 ± 0.890.0 ± 0.85
Change at Day 302-0.6 ± 0.890.2 ± 1.17
Change at Day 422-0.6 ± 1.820.5 ± 1.71
Change at Day 540-0.2 ± 2.240.5 ± 1.89
Change at Day 6590.6 ± 0.930.6 ± 1.93
Change at Day 778—0.0 ± 2.18
Change at Day 898—0.8 ± 2.70
Change at Day 1018—1.0 ± 2.02
Change at Day 1138—1.5 ± 0
PrimaryChange From Baseline in Weight for Age

Participants were analyzed for change in growth parameter of weight for age to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight for age percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · kilogram (kg)
Change From Baseline in Weight for Age
kilogram (kg)ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline42.5 ± 40.8725.3 ± 28.41
Change at Day 15-1.3 ± 1.413.6 ± 12.12
Change at Day 29-2.4 ± 1.61-1.4 ± 4.82
Change at Day 64-2.9 ± 2.580.5 ± 8.17
Change at Day 183-3.8 ± 6.93-5.9 ± 8.73
Change at Day 302-10.1 ± 13.04-8.0 ± 15.42
Change at Day 422-6.3 ± 9.45-7.5 ± 17.52
Change at Day 540-7.0 ± 6.56-8.1 ± 17.26
Change at Day 659-8.5 ± 17.64-10.5 ± 22.23
Change at Day 778—-13.2 ± 16.95
Change at Day 898—-10.5 ± 24.10
Change at Day 1018—-7.0 ± 34.14
Change at Day 1138—0.3 ± 0
PrimaryChange From Baseline in Weight

Participants were analyzed for change in growth parameter of weight to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the weight percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · kg
Change From Baseline in Weight
kgISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline13.0 ± 2.009.5 ± 1.37
Change at Day 15-0.1 ± 0.230.2 ± 0.44
Change at Day 29-0.1 ± 0.260.1 ± 0.23
Change at Day 640.0 ± 0.390.4 ± 0.42
Change at Day 1830.8 ± 0.610.7 ± 0.48
Change at Day 3020.7 ± 1.041.3 ± 0.87
Change at Day 4221.8 ± 1.031.9 ± 1.02
Change at Day 5402.3 ± 1.332.2 ± 1.17
Change at Day 6593.1 ± 1.392.8 ± 1.41
Change at Day 778—3.2 ± 1.87
Change at Day 898—3.9 ± 2.86
Change at Day 1018—4.3 ± 1.96
Change at Day 1138—5.0 ± 0
PrimaryChange From Baseline in Head to Chest Circumference (HCC) Ratio

Participants were analyzed for change in growth parameter of HCC to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the HCC circumference percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · ratio
Change From Baseline in Head to Chest Circumference (HCC) Ratio
ratioISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline1.0 ± 0.051.0 ± 0.08
Change at Day 150.0 ± 0.030.0 ± 0.03
Change at Day 290.0 ± 0.040.0 ± 0.04
Change at Day 640.0 ± 0.040.0 ± 0.05
Change at Day 1830.0 ± 0.050.0 ± 0.05
Change at Day 3020.0 ± 0.030.0 ± 0.06
Change at Day 4220.0 ± 0.040.0 ± 0.06
Change at Day 5400.0 ± 0.030.0 ± 0.06
Change at Day 6590.0 ± 0.03-0.1 ± 0.06
Change at Day 778—-0.1 ± 0.07
Change at Day 898—-0.1 ± 0.06
Change at Day 1018—-0.1 ± 0.10
Change at Day 1138—-0.1 ± 0
PrimaryChange From Baseline in Body Length

Participants were analyzed for change in growth parameter of body length to evaluate clinical efficacy. WHO Child Growth Standards were used to determine the body length percentile. Study days were windowed for integrated analysis and labelled as follows: Days \<=1 as Baseline; Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Part 2: Baseline, Day 15, 29, 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · cm
Change From Baseline in Body Length
cmISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline95.0 ± 9.4879.9 ± 5.25
Change at Day 15-1.6 ± 3.19-0.1 ± 1.81
Change at Day 29-0.9 ± 3.490.9 ± 1.66
Change at Day 640.5 ± 4.771.8 ± 2.40
Change at Day 1833.0 ± 4.165.6 ± 2.36
Change at Day 3022.9 ± 5.767.1 ± 2.70
Change at Day 4224.2 ± 5.429.3 ± 3.09
Change at Day 5404.6 ± 7.1811.6 ± 3.93
Change at Day 65910.8 ± 4.1213.1 ± 3.79
Change at Day 778—14.8 ± 3.94
Change at Day 898—17.2 ± 5.62
Change at Day 1018—19.0 ± 6.67
Change at Day 1138—15.8 ± 0
PrimaryNumber of Participants With Change From Baseline in Neurological Examination Outcomes

Neurological examinations included assessment of mental status, level of consciousness, sensory function, motor function, cranial nerve function, reflexes, mood, speech/language and hearing.

Time frame:
Part 1: Baseline to Day 422; Part 2: Baseline to Day 596
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Neurological Examination Outcomes
ParticipantsSham Procedure in Part 1ISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Mental Status013
Level of consciousness012
Sensory function000
Motor function000
Cranial nerve function: Eye Movement001
Cranial nerve function: Vision001
Reflexes000
Mood5512
Speech/Language011
Hearing000
PrimaryNumber of Participants With Change From Baseline in Activated Partial Thromboplastin Time [aPTT]

Activated partial thromboplastin time was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of aPTT at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of aPTT at baseline to high values postbaseline.

Time frame:
Part 2: Up to 1080 days
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Activated Partial Thromboplastin Time [aPTT]
ParticipantsISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Shift to Low00
Shift to High00
PrimaryNumber of Participants With Change From Baseline in Partial Thromboplastin Time [PTT]

PTT was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of PTT at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of PTT at baseline to high values postbaseline.

Time frame:
Part 2: Up to 1080 days
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in Partial Thromboplastin Time [PTT]
ParticipantsISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Shift to Low00
Shift to High00
PrimaryNumber of Participants With Change From Baseline in International Normalized Ratio [INR])

INR was evaluated to assess safety. "Shift to low" measured change in normal, high and unknown values of INR at baseline to low values postbaseline. "Shift to high" measured change in normal, high and unknown values of INR at baseline to high values postbaseline.

Time frame:
Part 2: Up to 1080 days
Reported as:
Count of participants · Participants
Number of Participants With Change From Baseline in International Normalized Ratio [INR])
ParticipantsISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Shift to Low00
Shift to High00
PrimaryNumber of Participants With Presence of Urine Total Protein Post-baseline

Urine total protein was evaluated to assess safety.

Time frame:
Part 2: Up to 1080 days
Reported as:
Count of participants · Participants
Number of Participants With Presence of Urine Total Protein Post-baseline
ParticipantsISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Baseline01
High/Postive28
SecondaryPlasma Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study

Study days were windowed for integrated analysis and labelled as follows: Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659.

Time frame:
Pre-dose on Days 64, 183, 540 and 659
Reported as:
Mean · nanogram per milliliter (ng/mL)
Plasma Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study
nanogram per milliliter (ng/mL)ISIS 396443 Part 2(Participants on Sham in Part 1)
Day 641.983 ± 0.7320
Day 1830.776 ± 0.3994
Day 5400.425 ± 0.2200
Day 6590.365 ± 0.1146
SecondaryPlasma Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study

Study days were windowed for integrated analysis and labelled as follows: Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018;Days \>1078 to \<= 1198 as Day 1138.

Time frame:
Pre-dose on Days 64, 183, 302, 422, 540, 659, 778, 898, 1018 and 1138
Reported as:
Mean · ng/mL
Plasma Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study
ng/mLISIS 396443 Part 1 & 2
Day 642.139 ± 0.8811
Day 1831.059 ± 0.5569
Day 3020.667 ± 0.1852
Day 4220.858 ± 0.4636
Day 5400.608 ± 0.2736
Day 6590.739 ± 0.2812
Day 7780.590 ± 0.3414
Day 8980.661 ± 0.2558
Day 10180.329 ± 0.1020
Day 11380.423 ± 0
SecondaryCerebrospinal Fluid (CSF) Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study

CSF samples were analyzed for ISIS 396443 concentrations in participants. Study days were windowed for integrated analysis and labelled as follows: Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540.

Time frame:
Pre-dose on Days 15, 29, 64, 183, 302, 422 and 540
Reported as:
Mean · ng/mL
Cerebrospinal Fluid (CSF) Concentration of ISIS 396443 in Part 2 of Study in Participants Who Received Sham Procedure in Part 1 of the Study
ng/mLISIS 396443 Part 2(Participants on Sham in Part 1)
Day 1NA ± NA
Day 153.094 ± 1.2172
Day 294.805 ± 2.6706
Day 644.357 ± 2.3229
Day 1834.110 ± 2.4535
Day 3025.397 ± 2.7503
Day 4226.460 ± 2.9428
Day 5408.405 ± 6.2423
SecondaryCSF Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study

CSF samples were analyzed for ISIS 396443 concentrations in participants. Study days were windowed for integrated analysis and labelled as follows: Days \>1 to \<= 22 as Day 15;Days \>22 to \<=47 as Day 29;Days \>47 to \<= 123 as Day 64;Days \>123 to \<=242 as Day 183;Days \>242 to \<=362 as Day 302;Days \>362 to \<=482 as Day 422;Days \>482 to \<= 600 as Day 540;Days \>600 to \<= 719 as Day 659;Days \>719 to \<= 838 as Day 778;Days \>838 to \<= 958 as Day 898;Days \>958 to \<= 1078 as Day 1018.

Time frame:
Pre-dose on Days 15, 29, 64, 183, 302, 422, 540, 659, 778, 898 and 1018
Reported as:
Mean · ng/mL
CSF Concentration of ISIS 396443 in Part 1 and 2 of Study in Participants Who Received ISIS 396443 in Part 1 of the Study
ng/mLISIS 396443 Part 1 & 2
Day 1NA ± NA
Day 153.925 ± 2.0525
Day 297.273 ± 4.4786
Day 647.176 ± 2.8487
Day 1838.226 ± 3.6450
Day 3028.968 ± 3.1188
Day 4229.251 ± 4.0631
Day 5409.026 ± 2.6864
Day 6599.785 ± 3.3725
Day 7788.632 ± 2.4131
Day 89811.321 ± 8.9351
Day 10187.010 ± 0
SecondaryNumber of Participants With Plasma Antibodies to ISIS 396443
Time frame:
Part 2: Baseline to Day 596
Reported as:
Count of participants · Participants
Number of Participants With Plasma Antibodies to ISIS 396443
ParticipantsISIS 396443 Part 2(Participants on Sham in Part 1)ISIS 396443 Part 1 & 2
Number of Participants With Plasma Antibodies to ISIS 39644300

Adverse events

Collected over From start to end of study (up to 1133 days). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Sham Procedure in Part 11/7 (14.3%)3/7 (42.9%)6/7 (85.7%)
ISIS 396443 Part 1 & 20/14 (0%)9/14 (64.3%)14/14 (100%)
ISIS 396443 Part 2(Participants on Sham in Part 1)0/6 (0%)4/6 (66.7%)6/6 (100%)
Most frequent serious events
Showing 10 of 38
Most frequent serious events
EventSham Procedure in Part 1ISIS 396443 Part 1 & 2ISIS 396443 Part 2(Participants on Sham in Part 1)
PneumoniaInfections and infestations0/77/143/6
Rhinovirus infectionInfections and infestations1/72/142/6
Respiratory distressRespiratory, thoracic and mediastinal disorders1/72/142/6
Pneumonia aspirationRespiratory, thoracic and mediastinal disorders0/73/141/6
Bronchitis moraxellaInfections and infestations0/70/141/6
Enterovirus infectionInfections and infestations1/70/141/6
GastroenteritisInfections and infestations0/71/141/6
Parainfluenzae virus infectionInfections and infestations0/71/141/6
Pneumonia haemophilusInfections and infestations1/70/141/6
Pneumonia moraxellaInfections and infestations0/70/141/6
Most frequent other events
Showing 10 of 187
Most frequent other events
EventSham Procedure in Part 1ISIS 396443 Part 1 & 2ISIS 396443 Part 2(Participants on Sham in Part 1)
PyrexiaGeneral disorders1/712/144/6
CoughRespiratory, thoracic and mediastinal disorders1/711/143/6
Upper respiratory tract infectionInfections and infestations2/79/143/6
VomitingGastrointestinal disorders1/77/142/6
PneumoniaInfections and infestations0/77/142/6
Pain in extremityMusculoskeletal and connective tissue disorders0/76/142/6
Nasal congestionRespiratory, thoracic and mediastinal disorders0/76/142/6
RhinorrhoeaRespiratory, thoracic and mediastinal disorders0/76/141/6
DiarrhoeaGastrointestinal disorders0/75/141/6
GastroenteritisInfections and infestations0/75/140/6

Baseline characteristics

The Intent-to-treat (ITT) population include all participants who were randomized, receive at least 1 dose of ISIS 396443 or sham procedure.

Age, Continuous
Age, Continuous(months)Sham Procedure (Part 1)ISIS 396443 (Part 1)Total
Mean24.4 ± 13.8319.4 ± 10.1221.1 ± 11.39
Sex: Female, Male
Sex: Female, Male(Participants)Sham Procedure (Part 1)ISIS 396443 (Part 1)Total
Female5510
Male2911
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Sham Procedure (Part 1)ISIS 396443 (Part 1)Total
Ethnicity: Hispanic or Latino213
Ethnicity: Not Hispanic or Latino4913
Ethnicity: Not reported due to confidentiality145
Race: Asian325
Race: White279
Race: Other112
Race: Not reported due to confidentiality145
08

Study locations

7 sites
  • David Geffen School of Medicine at UCLA
    Los Angeles, California 90095-8344, United States
  • Connecticut Childrens Medical
    Hartford, Connecticut 06106, United States
  • The Johns Hopkins Hospital
    Baltimore, Maryland 21287, United States
  • Gillette Children's Specialty Healthcare
    Saint Paul, Minnesota 55101, United States
  • The University of Texas Southwestern Medical Center
    Dallas, Texas 75235, United States
  • Seattle Children's Research Institute
    Seattle, Washington 98105, United States
  • LMU-Campus Innenstadt
    Muenchen, 80337, Germany
09

References and documents

Publications

  • Acsadi G, Crawford TO, Muller-Felber W, Shieh PB, Richardson R, Natarajan N, Castro D, Ramirez-Schrempp D, Gambino G, Sun P, Farwell W. Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study. Muscle Nerve. 2021 May;63(5):668-677. doi: 10.1002/mus.27187. Epub 2021 Feb 16. PubMed 33501671 ↗

Study documents

  • Statistical analysis plan · Jul 16, 2018
  • Study protocol · Jun 16, 2017

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 17, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02462759
Lead sponsor
Biogen
Responsible party
Sponsor
First posted
Jun 4, 2015
Start date
Aug 19, 2015
Primary completion
Sep 24, 2018
Completion
Sep 24, 2018
Results posted
Jan 27, 2020
Last update
Feb 17, 2021

Study contacts

Medical Director
study director · Biogen

Oversight

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

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