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A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
EU CTIS ID: 2023-505639-11-00
What this study is testing
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.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation
- Willing to stop risdiplam treatment
- Willing and able to start treatment with HD nusinersen
- Diagnosis of later-onset SMA with symptom onset at age >6 months.
- Aged ≥15 to ≤50 years at the time of informed consent
- Body weight >20 kg
You likely can't join if
- 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
- Permanent tracheostomy or permanent ventilation at Screening
- The medical necessity, as defined by the Investigator, for noninvasive ventilation such as bilevel positive airway pressure or continuous positive airway pressure outside of regular sleep hours for any reason other than proactive SMA management, at Screening
- History of bacterial meningitis, viral encephalitis, or hydrocephalus
See the full eligibility criteria
- Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation
- Willing to stop risdiplam treatment
- Willing and able to start treatment with HD nusinersen
- Diagnosis of later-onset SMA with symptom onset at age >6 months.
- Aged ≥15 to ≤50 years at the time of informed consent
- Body weight >20 kg
- Received oral risdiplam per the approved label or per the managed access program as follows 1. Nusinersen-naive participants must have had prior treatment with risdiplam for ≥6 months before enrollment 2. Nusinersen-experienced participants must have stopped nusinersen for ≥16 months and must have been on risdiplam for ≥12 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
- 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
- Permanent tracheostomy or permanent ventilation at Screening
- The medical necessity, as defined by the Investigator, for noninvasive ventilation such as bilevel positive airway pressure or continuous positive airway pressure outside of regular sleep hours for any reason other than proactive SMA management, at Screening
- 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 Inclusion/Exclusion criteria may apply.
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Japan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Japan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.