Clinical Research Alert: Phase 3 Study of Salanersen in Presymptomatic Newborns with SMA

Researchers at Biogen are working to better understand spinal muscular atrophy (SMA) and to evaluate a potentially new treatment for babies before they develop any symptoms.

The study

Newborns with SMA may be eligible to participate in a phase 3 clinical trial (STELLAR-1) to evaluate the safety and efficacy of the investigational therapy salanersen to treat presymptomatic SMA. Salanersen is designed to increase production of SMN protein from the SMN2 gene. SMN protein is essential for keeping motor neurons healthy and functional. This study aims to determine whether starting salanersen before the appearance of any signs or symptoms can prevent SMA from developing or reduce the severity of symptoms if they do occur.

This is an open-label, phase 3 study comprised of two parts. In part 1, all participants will receive two doses of salanersen, about 12 months apart. Participants in part 1 will engage in a screening visit, 11 clinic visits, and three phone calls over about 25-26 months. Participants who complete part 1 may enroll in part 2, during which they will receive three additional doses of salanersen, again 12 months apart. Part 2 will last up to 36 months, with seven clinic visits and 12 phone calls. The total study duration for participants will be about five years.

Salanersen will be administered through an intrathecal (into the spinal cord) injection once per year. The effects of the drug will be evaluated using a number of assessments including but not limited to:

• Physical and neurological examinations
• Blood and urine sample collections
• Vital sign measurements
• Electrocardiograms (ECGs)
• Assessments of development, strength, and movement
• Assessment of nerve health
• Questionnaires about how your child is feeling and acting

Study criteria

To be eligible for this study, individuals must meet the following inclusion criteria:

• ≤42 days of age at first dose of salanersen
• Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation
• Two or three copies of the survival motor neuron 2 (SMN2) gene
• Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose
• Body weight ≥3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent
• Meet other protocol-defined inclusion criteria

Individuals may not be eligible to participate if they are affected by another condition or have received another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel support will be available for eligible participants and their families.

People interested in learning more can reach out to the study coordinator by email at clinicaltrials@biogen.com or visit the sponsor’s website.