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.

SMA Drug Trials Currently Recruiting Participants

A Study of Multiple Doses of IONIS-SMNRx (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Also Known By: NURTURE
General Criteria*: Very young infants genetically diagnosed with SMA but not yet showing symptoms.
Phase: Phase 3 Identifier: NCT02386553

A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

Also Known By: N/A
General Criteria*: Teens and adults 12 years of age or older (ambulatory and non-ambulatory) with SMA type II, III or IV.
Phase: Phase 2 Identifier: NCT02644668

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants

Also Known By: SUNFISH
General Criteria*: Children, teens and adults age 2-25 with SMA type II or III.
Phase: Phase 2 Identifier: NCT02908685

An Open-label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA) - SUSPENDED

Also Known By: N/A
General Criteria*: Infants age seven months or less with SMA type I.
Phase: Phase 2 Identifier: NCT02268552

A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy - SUSPENDED

Also Known By: MOONFISH
General Criteria*: Individuals age 2-55 with SMA type I, II, or III.
Phase: Phase 1 Identifier: NCT02240355

Expanded Access Programs

Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

Also Known By: N/A
General Criteria*: Individuals of all ages with SMA type I Identifier: NCT02865109

*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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