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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Testing NMD670 for adults with Type 3 Spinal Muscular Atrophy.

Recruiting
18-75 years
All
Phase 2
  • Simplified description (AI rewritten)
  • Technical description (Original)

Study Objective: This study tests how well and how safely a new medicine, NMD670, works for adults who can walk and have Type 3 Spinal Muscular Atrophy (SMA). SMA is a disease that makes muscles weak because of a problem in a gene called SMN1.

Who Can Join: You can join if you have Type 3 SMA, can walk at least 50 meters without help, and have a confirmed genetic diagnosis. Your Body Mass Index (BMI) should be between 19 and 35. Both men and women can participate, but you must agree to use birth control if needed.

Who Cannot Join: You cannot join if you have had surgery that affects your movement or have other health issues that might affect the study. Also, you cannot join if you have taken another experimental medicine recently or if you have not followed previous SMA treatments as instructed.

  • Length: Find out how long you need to be in the study.
  • Visits: Check how many visits are required.
  • Risks: Learn about any possible risks involved.
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Study details
    Spinal Muscular Atrophy

NCT05794139

NMD Pharma A/S

15 March 2025

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