At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Risdiplam (drug)
- How long the study runs
- Study runs about 58 months (dates as stated)
- About the drug or intervention
- Risdiplam — drug: Participants will receive risdiplam orally at the currently approved dose.
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 3 Months to 24 Months
- Who
- All
- Number of participants
- 28
- Started
- 2024-05-30
- Last checked
- 2026-10
Plain English Summary
What is this study?
- • Testing a new treatment for muscular atrophy, spinal
- • Phase4 - 28 participants
- • This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec
Who can take part?
- • Ages 3 Months to 24 Months
- • Diagnosed with muscular atrophy, spinal
Where?
- • London - Great Ormond Street Hospital For Children
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 3 Months - 24 Months
- Who can join: All genders
What the study is looking for
- ✓\<2 years of age at the time of agreement to take part
- ✓Confirmed presence of two SMN2 gene copies as documented through laboratory testing
- ✓Administration of onasemnogene abeparvovec pre-symptomatically or post-symptomatically
- ✓Has received onasemnogene abeparvovec for SMA no less than 13 weeks, but not more than months 30 weeks, prior to...
- ✓If treated with risdiplam prior to onasemnogene abeparvovec, risdiplam treatment must not have exceeded 3 weeks and...
Who cannot take part
- ✗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
- ✗Concomitant or previous use of an anti-myostatin agent
- ✗Participants requiring invasive ventilation or tracheostomy
See the full criteria
Where Is This Study? (1 UK site)
Great Ormond Street Hospital For Children
London WC1N 3JH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700How to Get in Touch
Reference Study ID Number: BN44620 https://forpatients.roche.com/ No attachments to email below.
Sponsor contactCONTACT
Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
Sponsor contactCONTACT
