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 www.clinicaltrials.gov 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.

Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1

Also Known By: STR1VE
General Criteria*: Infants 6 months of age or less with 1-2 copies of SMN2
Phase: Phase 3
Clinicaltrials.gov Identifier: NCT03306277


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
Clinicaltrials.gov Identifier: NCT02644668


A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type 1 Spinal Muscular Atrophy

Also Known By: FIREFISH
General Criteria*: Infants age 1-7 months with SMA type I.
Phase: Phase 2
Clinicaltrials.gov Identifier: NCT02913482


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
Clinicaltrials.gov Identifier: NCT02908685


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

Also Known By: JEWELFISH
General Criteria*: Children, teens and adults age 12-60 who have previously been exposed to an SMN2-targeting therapy.
Phase: Phase 2
Clinicaltrials.gov Identifier: NCT03032172


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

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


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
Clinicaltrials.gov 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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