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-GR
• Lead Sponsor: Biogen Idec Research Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2020-11-02
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 145

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
Are the trial subjects under 18? yes
Number of subjects for this age range: 121
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
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.
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 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
Part B:
- Treatment with an investigational drug including but not limited to the
treatment of SMA, biological agent, or device within 30 days or 5 halflives
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
- 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
Part C:
- Concurrent or previous participation and/or administration of
nusinersen in another clinical study
- 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
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

Study & Design

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