Skip to main content
MedPath Trial logoMedPath TrialMedPath Trial Home
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:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
152
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

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

Similar Trials

Active, not recruiting
Phase 1
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalMedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
EUCTR2019-002663-10-HUBiogen Idec Research Limited100
Not yet recruiting
Phase 2
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular DiseasesG12.9Spinal Muscular Atrophy
LBCTR2021064778Biogen Idec Research Limited2
Active, not recruiting
Phase 1
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalMedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
EUCTR2019-002663-10-NLBiogen Idec Research Limited172
Active, not recruiting
Phase 1
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalMedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
EUCTR2019-002663-10-FRBiogen Idec Research Limited152
Active, not recruiting
Phase 1
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyMuscular Atrophy, SpinalMedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
EUCTR2019-002663-10-ITBIOGEN IDEC RESEARCH LIMITED152