NCT05808764 A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
| NCT ID | NCT05808764 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Hoffmann-La Roche |
| Condition | Muscular Atrophy, Spinal |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2024-04-26 |
| Primary Completion | 2026-11-30 |
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.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 10 participants in total. It began in 2024-04-26 with a primary completion date of 2026-11-30.
⚠ 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 study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
Eligibility Criteria
Inclusion Criteria: * Male or female newborn infant aged \<20 days at first dose * Newborn infants with genetic diagnosis of 5q-autosomal recessive SMA or newborn infants identified as positive for SMA via newborn screening or via prenatal testing. * Gestational age equal to or greater than 37 weeks * Receiving adequate nutrition and hydration at the time of screening * Adequately recovered from any acute illness at baseline and considered well enough to participate in the study * Parent/caregiver is willing to consider nasogastric, nasojejunal, or gastrostomy tube placement during the study to maintain safe hydration, nutrition, and treatment delivery, if recommended by the investigator. Exclusion Criteria: * Presence of clinical symptoms or signs consistent with SMA Type 0 * In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures * Systolic blood pressure or diastolic blood pressure or heart rate abnormalities * Presence of clinically relevant electrocardiogram (ECG) abnormalities * The infant (or the person breastfeeding the infant) taking any of the following: any inhibitor of CYP3A4 taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing, any inducer of CYP3A4 taken within 4 weeks (or within 5 times the elimination half-life, whichever is longer prior to dosing, and/or use of any multidrug and toxin extrusion (MATE) substrates taken within 2 weeks (or within 5 times the elimination half-life, whichever is longer) prior to dosing * Concurrent or previous administration of nusinersen or onasemnogene abeparvovec * Clinically significant abnormalities in laboratory test
Contact & Investigator
Reference Study ID Number: BN44619 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 NCT05808764 clinical trial?
This trial is open to participants of all sexes, up to 19 Days, 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 NCT05808764 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05808764 currently recruiting?
Yes, NCT05808764 is actively recruiting participants. Contact the research team at global-roche-genentech-trials@gene.com for enrollment information.
Where is the NCT05808764 trial being conducted?
This trial is being conducted at Chicago, United States, Ann Arbor, United States, Gordonville, United States, Brussels, Belgium and 9 additional locations.
Who is sponsoring the NCT05808764 clinical trial?
NCT05808764 is sponsored by Hoffmann-La Roche. The principal investigator is Clinical Trials at Hoffmann-La Roche. The trial plans to enroll 10 participants.