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

Phase 1

• Conditions: Muscular Atrophy, Spinal; MedDRA version: 20.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

• Registration Number: EUCTR2019-002663-10-ES
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-05-10
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 100

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 = 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:
- 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: 132
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

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
- Dosing with onasemnogene abeparvovec-xioi (Zolgensma) within 6 months prior to Screening

Part A:
- 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 prior to Screening or anytime during the study; prior treatment with risdiplam or branaplam; or any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation

Part B:
- 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 prior to Screening or anytime during the study; prior treatment with risdiplam or branaplam; or any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation

Part C:
- Concurrent participation and/or administration of nusinersen in another clinical study

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified