Muscular Atrophy, Spinal MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Spinraza
Product Name: .Nusinersen
Product Code: [ ISIS 396443, BIIB058]
Pharmaceutical Form: Solution for injection
CAS Number: 125894-36-9
Current Sponsor code: ISIS 396443 (BIIB058)
Oth
Sponsors
BIOGEN IDEC RESEARCH LIMITED
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: - Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation. - Diagnosis of later-onset SMA with symptom onset at age >6 months. - Aged =5 to =39 years at the time of informed consent for nusinersen-naïve participants. - Aged =18 to =39 years at time of informed consent for nusinersen-experienced participants. - Body weight >20 kg. - Received oral risdiplam per the approved label or per the managed access program as follows Nusinersen-naive participants must have had prior treatment with risdiplam for =6 months and =12 months before enrollment. Nusinersen-experienced participants must have stopped nusinersen for =16 months and have been on risdiplam for =12 months and =18 months before enrollment. - Able to perform the age-appropriate functional assessments in the study. - RULM entry item A score =3. - RULM total score =5 and =30 at Screening. - Nonambulatory, defined as not able to walk 15 feet (4.57 meters) independently without support. - Willing to stop risdiplam treatment. - Willing and able to start treatment with nusinersen. NOTE: Other protocol defined Inclusion criteria may apply. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 35 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: - Any major illness within 1 month before the Screening examination or within 1 week prior to Screening and up to first dose administration. - 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 catheter. - History of bacterial meningitis, viral encephalitis, or hydrocephalus. - Ongoing medical condition that according to the Investigator would interfere with the conduct and assessments of the study. An example is a medical disability (e.g., wasting or cachexia, severe anemia, and respiratory parameters) that would interfere with the assessment of safety or would compromise the ability of the participant to undergo study procedures. - Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study. - Treatment with an investigational drug, 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 gene therapy for the treatment of SMA. NOTE: Other protocol defined Exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.;Secondary Objective: The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with risdiplam.;Primary end point(s): Change in Total Revised Upper Limb Module (RULM) Score;Timepoint(s) of evaluation of this end point: Up to Day 855 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1) Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) 2) Number of Participants With Change from Baseline in Clinical Laboratory Parameters, Electrocardiogram (ECG), Vital Signs and Pulse Oximetry;Timepoint(s) of evaluation of this end point: 1) Up to Day 855 2) Up to Day 855 | — |
Countries
Australia, Belgium, Brazil, Canada, Germany, Hungary, Italy, Japan, Poland, Spain, Switzerland
Contacts
Public ContactMedical Director
Biogen Idec Research Limited
Outcome results
None listed