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A Study to Evaluate the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients with Spinal Muscular Atrophy After Gene Therapy
EU CTIS ID: 2023-504508-26-00
What this study is testing
To evaluate, in pediatric patients, the effectiveness of risdiplam administered as an early intervention after onasemnogene abeparvovec
- Therapeutic use (Phase IV)
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
- Confirmed diagnosis of 5q-autosomal recessive SMA, including genetic confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the survival of motor neuron 1 (SMN1) telomeric gene
- Confirmed presence of two survival of motor neuron 2 (SMN2) centromeric gene copies as documented through laboratory testing
- Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically (≤3 months of symptoms)
- Has received onasemnogene abeparvovec for SMA no less than13 weeks (- 2 weeks),, but not more than, 30 weeks (+ 2 weeks), prior to enrollment
- If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and must be discontinued 1 day prior to onasemnogene abeparvovec administration
- Parent or caregiver of patient willing to consider the use of non-invasive ventilation during the study, as recommended by the investigator
You likely can't join if
- Previous or current enrolment in investigational study prior to initiation of study treatment
- Any unresolved standard-of-care laboratory abnormalities per the onasemnogene abeparvovec prescribing information
- Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide
- Patients requiring invasive ventilation or tracheostomy and patients requiring awake non-invasive ventilation or with awake hypoxemia (SaO2 < 95%) with or without ventilator support
- Any major illness requiring hospitalization within 1 month before the screening examination or any febrile illness within 1 week prior to screening and up to first dose administration
- Concomitant or previous use of an anti-myostatin agent
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
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. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. 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.