Trials Currently Recruiting

Please note that this is not a complete list of all ongoing SMA clinical trials. This only covers drug trials (often called "interventional" trials or studies) that are currently recruiting participants.

All the information here is drawn from and is accurate to the best of our knowledge. However, you should contact your healthcare provider and the study organizer to discuss your or your child's participation.

We provide resources to help you determine if participation in a clinical trial is the right choice for you and your family, and to guide you in asking the right questions about a clinical trial. Ultimately, your decision should be made after consulting with those who understand your unique situation.

Spinraza in Adult Spinal Muscular Atrophy 

Also Known By: SAS
General Criteria*: Adults with SMA who are ages 18-60 who are planning to initiate treatment with SPIRNAZA
Phase: Observational study designed to evaluate the safety, tolerability and effectiveness of SPINRAZA (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Identifier: NCT03709784

Pre-Symptomatic Study of Intravenous AVXS-101 in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (SPR1NT)

Also Known By: SPR1NT
General Criteria*: Infants under six weeks of age who are genetically diagnosed with SMA but not yet showing symptoms, and who have 2, 3 or 4 copies of SMN2
Phase: Phase 3 Identifier: NCT03505099

A Study of RO7034067 in Adult and Pediatric Participants with Spinal Muscular Atrophy

Also Known By: JEWELFISH
General Criteria*: Children, teens and adults with SMA Type 1, 2 or 3, aged 6 months to 60 years, in addition to those who have previously been treated with gene therapy, an SMN2-targeting therapy or olesoxime
Phase: Phase 2 Identifier: NCT03032172

Study of Intrathecal Administration of AVXS-101 for Spinal Muscular Atrophy (STRONG)

Also Known By: STRONG
General Criteria*: Children up to five years of age with SMA type 2 and 3 copies of SMN2
Phase: Phase 1 Identifier: NCT03381729

*All trials have additional criteria that must be met for enrollment.

If you are a clinical trial organizer looking for assistance in recruitment for clinical trials, please visit our For Researchers section.

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