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Looking for participantsPhase4

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Sponsor: Hoffmann-La Roche

NCT ID: NCT05861986

View on ClinicalTrials.gov ↗

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.

Muscular Atrophy, Spinal

How this trial compares with your answers

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What we know so far

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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
Inclusion Criteria: * \<2 years of age at the time of informed consent * 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) gene * 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 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 * Has, in the opinion of the investigator, not experienced clinically significant decline in function from the time of onasemnogene abeparvovec administration Exclusion Criteria: * 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 * Participants requiring awake non-invasive ventilation or with awake hypoxemia (Arterial Oxygen Saturation \[SaO2\] \<95%) with or without ventilator support * Presence of feeding tube and an OrSAT score of 0 * Hospitalization for pulmonary event within the last 2 months, or any planned hospitalization at the time of screening * 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.

Where Is This Study? (1 UK site)

Great Ormond Street Hospital For Children

London WC1N 3JH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Main Email: Research.Governance@gosh.nhs.uk

Research.Governance@gosh.nhs.uk0207 905 2700

How to Get in Touch

Reference Study ID Number: BN44620 https://forpatients.roche.com/ No attachments to email below.

Sponsor contact

CONTACT

888-662-6728 (U.S. Only) global-roche-genentech-trials@gene.com

Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry

Sponsor contact

CONTACT

Data sourced from ClinicalTrials.gov · Last verified: 2026-10