Investigating the safety and pharmacokinetics of risdiplam in infants with spinal muscular atrophy
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study is testing how safe the drug risdiplam is and how it works in newborns with spinal muscular atrophy to make sure it’s safe for them.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | N/A to 19 Days |
| Sex | All |
| Sponsor | Hoffmann-La Roche Industry-sponsored |
| Locations | 13 sites (Chicago, Illinois and 12 other locations) |
| Trial ID | NCT05808764 on ClinicalTrials.gov |
What this trial studies
This study evaluates how the drug risdiplam is processed in the body and its safety profile in newborn infants diagnosed with spinal muscular atrophy (SMA). Participants must be under 20 days old at the time of the first dose and have a confirmed genetic diagnosis of SMA. The study aims to ensure that these infants receive adequate nutrition and hydration while monitoring their health throughout the treatment. The findings could provide critical insights into the drug's effectiveness and safety for this vulnerable population.
Who should consider this trial
Good fit: Ideal candidates are newborn infants under 20 days old with a genetic diagnosis of 5q-autosomal recessive SMA.
Not a fit: Patients with clinical symptoms consistent with SMA Type 0 may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved treatment options for infants with spinal muscular atrophy.
How similar studies have performed: Other studies have shown promise in similar approaches for treating SMA, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Chicago, Illinois and 12 other locations
- Ann and Robert H. Lurie Children Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- University Of Michigan — Ann Arbor, Michigan, United States (Recruiting)
- Clinic for Special Children. — Gordonville, Pennsylvania, United States (Recruiting)
- Hopital Universitaire des Enfants Reine Fabiola — Brussels, Belgium (Recruiting)
- CHR Citadelle — Liège, Belgium (Recruiting)
- Children'S Hospital of Eastern Ontario — Ottawa, Ontario, Canada (Recruiting)
- Universitatsklinikum Essen — Essen, Germany (Recruiting)
- Fondazione Serena Onlus - CENTRO CLINICO NEMO — Milano, Emilia-Romagna, Italy (Recruiting)
- Fondazione Policlinico Univeristario A. Gemelli — Roma, Emilia-Romagna, Italy (Recruiting)
- UMC Utrecht — Utrecht, Netherlands (Recruiting)
- OUS (Oslo University Hospital), Rikshospitalet — Oslo, Norway (Recruiting)
- Uniwersyteckie Centrum Kliniczne — Gdansk, Poland (Recruiting)
- Instytut Pomnik - Centrum Zdrowia Dziecka — Warsaw, Poland (Recruiting)
Study contacts
- Study coordinator: Reference Study ID Number: BN44619 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728 (U.S. Only)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.