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Current Status
Recruiting
Verified Feb 9, 2026
Phase
Clinical Trial
Sponsor
Hoffmann-La Roche
Enrollment Target
28
Start Date
May 2024
Interested in learning more? Complete the form below.
No-Cost Study Care
Advance Medical Science
Contribute to groundbreaking research
Cutting-Edge Treatment
Access to innovative therapies
Expert Medical Care
Close monitoring by specialists
Possible Compensation*
For time and travel (varies by study)
Help Others
Benefit future patients
*Compensation varies by study. Confirm details with coordinator.
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.
Study Type
INTERVENTIONAL
Target Enrollment
28 participants
Age Range
3 Months - 24 Months
Gender
ALL
Sponsor
Hoffmann-La Roche
Choose your preferred location or select flexible during enrollment
📍 And 2 more locations available
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Browse all pulmonary hypertension clinical trials in these cities — not just this study.
Join others in Arkansas exploring innovative treatment options through clinical research
If you're searching for Spinal Muscular Atrophy treatment in Little Rock, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Little Rock, Aurora, Gainesville and surrounding areas.
Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Spinal Muscular Atrophy. All study-related care is provided at no cost to participants.
Important Clinical Trial Information
This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.
Study identifier: NCT05861986. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.