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Clinical Trials/EUCTR2019-002663-10-GB
EUCTR2019-002663-10-GBActive, not recruitingPhase 1

Escalating Dose and Randomized, Controlled Study of Nusinersen(BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Biogen Idec Research Limited0 sites152 target enrollmentStarted: September 1, 2020Last updated:
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
152

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional clinical trial of medicinal product

Eligibility Criteria

Sex
All

Inclusion Criteria

  • Part A, B and C: - Genetic documentation of 5q SMA (homozygous gene
  • deletion, mutation, or compound heterozygote)
  • - 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
  • - Participants with SMA symptom onset = 6 months (= 180 days) of age (infantile onset) should have age = 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
  • - Participants = 18 years of age at Screening must be ambulatory
  • - Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening
  • Are the trial subjects under 18? yes
  • Number of subjects for this age range: 140
  • F.1.2 Adults (18-64 years) yes
  • F.1.2.1 Number of subjects for this age range 12
  • F.1.3 Elderly (>=65 years) no
  • F.1.3.1 Number of subjects for this age range 0

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 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
  • - 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
  • - 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
  • - 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
  • - 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
  • - Concurrent or previous participation and/or administration of nusinersen in another clinical study
  • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Investigators

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