NCT05861999 A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
| NCT ID | NCT05861999 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Hoffmann-La Roche |
| Condition | Muscular Atrophy, Spinal |
| Study Type | INTERVENTIONAL |
| Enrollment | 28 participants |
| Start Date | 2024-08-14 |
| Primary Completion | 2028-03-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.
This trial targets 28 participants in total. It began in 2024-08-14 with a primary completion date of 2028-03-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.
Eligibility 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 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. * In the opinion of the investigator, has demonstrated a plateau or decline in function post-gene therapy (with a duration of 26 weeks or less) documented by 2 individual time points in the functions as follows: swallowing AND one additional function/ability (respiratory, motor function, other) per appropriate expectation. 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 * 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.
Contact & Investigator
Reference Study ID Number: BN44621 https://forpatients.roche.com/
✉ global-roche-genentech-trials@gene.com📞 888-662-6728 (U.S. Only)
Clinical Trials
STUDY DIRECTOR
Hoffmann-La Roche
Frequently Asked Questions
Who can join the NCT05861999 clinical trial?
This trial is open to participants of all sexes, aged 3 Months or older, up to 24 Months, studying Muscular Atrophy, Spinal. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05861999 trial and what does that mean for participants?
Phase 4 studies are conducted after a treatment has been approved. They monitor long-term safety and real-world effectiveness in a broader patient population.
Is NCT05861999 currently recruiting?
Yes, NCT05861999 is actively recruiting participants. Contact the research team at global-roche-genentech-trials@gene.com for enrollment information.
Where is the NCT05861999 trial being conducted?
This trial is being conducted at Little Rock, United States, Madera, United States, Palo Alto, United States, Aurora, United States and 11 additional locations.
Who is sponsoring the NCT05861999 clinical trial?
NCT05861999 is sponsored by Hoffmann-La Roche. The principal investigator is Clinical Trials at Hoffmann-La Roche. The trial plans to enroll 28 participants.