Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

  • STATUS
    Recruiting
  • End date
    Jul 4, 2023
  • participants needed
    172
  • sponsor
    Biogen
Updated on 22 December 2021
muscle atrophy
gene deletion
nusinersen

Summary

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C).

The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).

Details
Condition Muscular Atrophy, Spinal
Treatment Nusinersen
Clinical Study IdentifierNCT04089566
SponsorBiogen
Last Modified on22 December 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Part A, B and C
Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote)
Part A
Onset of clinical signs and symptoms consistent with SMA at > 6 months (> 180 days) of age (i.e., later-onset SMA)
Age 2 to ≤ 15 years, inclusive, at the time of informed consent
Part B
Participants with SMA symptom onset ≤ 6 months (≤ 180 days) of age (infantile onset) should have age > 1 week to ≤ 7 months (≤ 210 days) at the time of informed consent
Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset)
Age 2 to < 10 years at the time of informed consent
Can sit independently but has never had the ability to walk independently
HFMSE score ≥ 10 and ≤ 54 at Screening
Part C
Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening
Part C Cohort 1
Participants of any age (individuals ≥18 years of age at Screening must be ambulatory)
Part C Cohort 2
Participants ≥18 years of age at Screening (can be ambulatory or nonambulatory)
HFMSE total score ≥4 points at Screening
RULM entry item A score ≥3 points at Screening

Exclusion Criteria

Part A, B and C
Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening period
Presence of an implanted shunt for the drainage of cerebrospinal fluid (CSF) or of an implanted central nervous system (CNS) catheter
Hospitalization for surgery, pulmonary event, or nutritional support within 2 months prior to Screening or planned within 12 months after the participant's first dose
Part A
Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
Treatment with an investigational drug given for the treatment of SMA, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with any survival motor neuron-2 (SMN2)-splicing modifier or gene therapy; or prior antisense oligonucleotide treatment, or cell transplantation
Medical necessity for a gastric feeding tube
Part B
Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset)
Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
Medical necessity for a gastric feeding tube
Part C
Treatment with an investigational drug including but not limited to the treatment of SMA, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with any SMN2-splicing modifier or gene therapy; or prior antisense oligonucleotide treatment, or cell transplantation
Concurrent or previous participation and/or administration of nusinersen in another clinical study
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
Participants with SMA symptom onset ≤ 6 months (≤ 180 days) of age (infantile onset)
Signs or symptoms of SMA present at birth or within the first week after birth
Concomitant or previous administration of any SMN2-splicing modifier (excluding nusinersen) or gene therapy, either in a clinical study or as part of medical care
Concurrent or previous participation in any interventional investigational study for any other drug or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening
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