Spinal Muscular Atrophy (SMA)

Condition Description

Spinal muscular atrophy (SMA) is a group of hereditary diseases that progressively destroys motor neurons—nerve cells in the brain stem and spinal cord that control essential skeletal muscle activity such as speaking, walking, breathing, and swallowing, leading to muscle weakness and atrophy. Motor neurons control movement in the arms, legs, chest, face, throat, and tongue. When there are disruptions in the signals between motor neurons and muscles, the muscles gradually weaken, begin wasting away and develop twitching (called fasciculations).

Active Trials

  • TRIAL TITLE: Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

    DESCRIPTION: A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death.

    SPONSOR: Genentech, Inc.

    INDICATION: Spinal Muscular Atrophy

    STUDY PROTOCOL: ML43702

    PHASE: 4

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)

    DESCRIPTION: The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.

    The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with risdiplam.

    SPONSOR: Biogen

    INDICATION: Spinal Muscular Atrophy, Neuromuscular Diseases, SMA

    STUDY PROTOCOL: ASCEND 232SM303

    PHASE: 3b

    STATUS: Active

    RECRUITING PATIENTS: Yes

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  • TRIAL TITLE: A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non- Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension.

    DESCRIPTION: BHV2000-301 is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2 arm study designed to assess the efficacy and safety of taldefgrobep alfa in study participants with SMA who are stable on standard of care therapy including risdiplam, and/or nusinersen, and/or who have a history of treatment with onasemnogene abeparvovec-xioi.

    SPONSOR: Biohaven Pharmaceuticals, Inc.

    INDICATION: Spinal Muscular Atrophy

    STUDY PROTOCOL: BHV2000-301

    PHASE: 3

    STATUS: Active

    RECRUITING PATIENTS: No

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  • TRIAL TITLE: A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy.

    DESCRIPTION: The purpose of this study is to measure clinical efficacy on muscle strength and function, safety, and tolerability of NMD670 compared with placebo in ambulatory participants with Type 3 SMA.

    SPONSOR: NMD Pharma

    INDICATION: Type 3 Spinal Muscular Atrophy

    STUDY PROTOCOL: NMD670-02-0001

    PHASE: 2

    STATUS: Active

    RECRUITING PATIENTS: Yes

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