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Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
NCT05794139 · View on ClinicalTrials.gov ↗
Study Summary
The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
Conditions Studied
Interventions
- DRUG Placebo
- DRUG NMD670
Study Locations (20)
Other
- UZ Leuven - Neurochirurgie Campus Gasthuisberg — Leuven
- CHR de la Citadelle - Neurologie — Liège
- Heritage Medical Research Clinic — Calgary
- Genge Partners Inc. — Montreal
- Aarhus Universitetshospital, Neurologisk Afdeling — Aarhus
- Rigshospitalet - Neurologisk Afdeling — Copenhagen
- Charite - Campus Virchow-Klinikum (CVK) — Berlin
- Universitätsklinikum Essen - Klinik Für Neurologie — Essen
California
- UCLA David Geffen School Of Medicine - Neurology — Los Angeles
- Stanford University Medical Center — Palo Alto
Missouri
- Roy Blunt NextGen Precision Health Institute — Columbia
- Washington University School of Medicine — St Louis
Colorado
- University of Colorado Neurology Clinic — Aurora
Florida
- UF Fixel Institute for Neurological Diseases — Gainesville
Georgia
- Rare Disease Center — Atlanta
Kansas
- University of Kansas Medical Center — Kansas City
Maryland
- The Johns Hopkins Medicine, Spinal Muscular Atrophy Center — Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 54 participants |
| Start Date | 2023-09-21 |
| Est. Completion | 2026-05 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05794139
The ClinicalTrials.gov registry entry for NCT05794139 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 54 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is NMD Pharma A/S, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05794139 reports 20 study locations spanning 11 distinct geographic areas — top geographies include Other, California, Missouri. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05794139 about?
NCT05794139 is a clinical study titled "Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy". The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3
What is the current status of trial NCT05794139?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 54 participants. The study started on 2023-09-21. Estimated completion is 2026-05.
What conditions does trial NCT05794139 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05794139?
The interventions under investigation include: Placebo (DRUG), NMD670 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05794139?
This trial is sponsored by NMD Pharma A/S, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05794139 being conducted?
This trial has 20 study locations across California, Colorado, Florida, Georgia, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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