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: NMD670-02-0000

    PHASE: 2a

    STATUS: Active

    RECRUITING PATIENTS: Yes

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Upcoming Trials

Past Trials

  • TRIAL TITLE: A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)

    DESCRIPTION: The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.

    The secondary objectives of this study are to evaluate the safety and tolerability; and clinical outcomes following treatment with nusinersen in participants with SMA who previously received onasemnogene abeparvovec.

    SPONSOR: Biogen

    INDICATION: Spinal Muscular Atrophy

    STUDY PROTOCOL: RESPOND 232SM404

    PHASE: 4

    STATUS: Complete

    RECRUITING PATIENTS: No

  • TRIAL TITLE: An expanded access program for Risdiplam in patients with type 1 or type 2 spinal muscular atrophy

    DESCRIPTION: This program is designed to provide access to Risdiplam for eligible patients with Type 1 or Type 2 SMA prior to commercial availability of Risdiplam.

    SPONSOR: Genentech, Inc.

    INDICATION: Type 1 and Type 2 Spinal Muscular Atrophy

    STUDY PROTOCOL: AL41887

    PHASE: Expanded access program

    STATUS: Complete

    RECRUITING PATIENTS: No