Spinal Muscular Atrophy Trial, Recruiting NCT04944940 Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) Condition: Spinal Muscular Atrophy
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Spinal Muscular Atrophy Trial, Recruiting

NCT04944940
Recruiting Not Applicable

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

If you have SBMA (Spinal Bulbar Muscular Atrophy):

  • You are willing to follow all study procedures and are available for the full duration of the study
  • You are male and over 18 years old
  • Your SBMA diagnosis has been confirmed through genetic testing
  • You are able to understand the study and are willing to sign a consent form
  • You are able to travel to the NIH Clinical Center

If you are joining as a Healthy Control (comparison participant):

  • You are willing to follow all study procedures and are able to travel to the NIH for the duration of the study
  • You are male and over 18 years old
  • You have no history of SBMA or any other neuromuscular (nerve or muscle) condition
  • You have no history of facial palsy (weakness or drooping of the face)
  • You are able to understand the study and are willing to sign a consent form
  • You are able to travel to the NIH Clinical Center

Who may not be able to join:

SBMA participants may not be eligible if:

  • You cannot have an MRI scan — for example, if you have a pacemaker, defibrillator, insulin pump, metal clips, non-removable metal jewellery, or if you experience severe claustrophobia (fear of enclosed spaces)
  • You are unable to walk
  • You have used hormone-reducing medications (androgens) in the past two years

SBMA participants may not be able to take part in the spinal fluid (lumbar puncture) procedure if:

  • Your blood clotting test results are outside of the normal range by a certain amount (confirm with trial site)
  • You have a low platelet count, abnormal bleeding time, or certain abnormal blood test results (confirm with trial site)
  • You have a history of a bleeding disorder
  • You are taking blood-thinning medications

SBMA participants may not be able to take part in the muscle biopsy (small muscle sample) procedure if:

  • The muscle in your lower leg that would be sampled has wasted away too much to safely take a sample
  • You have taken aspirin or anti-inflammatory painkillers (such as ibuprofen) in the 3 days before the procedure

SBMA participants may not be able to take part in the whole-body MRI if:

  • You have previously been treated with hormone-reducing medications, including certain types of injections or tablets used to lower androgen (male hormone) levels
  • You are unable to complete strength and walking tests as part of the study
  • You are planning to make significant changes to your diet or exercise routine during the study period

Healthy Control participants may not be eligible if:

  • Your blood clotting test results are outside of the normal range by a certain amount (confirm with trial site)
  • You have a low platelet count, abnormal bleeding time, or certain abnormal blood test results (confirm with trial site)
  • You have a history of a bleeding disorder
  • You are taking blood-thinning medications

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Christopher Grunseich, M.D., National Institute of Neurological Disorders and Stroke (NINDS)

Phone: (301) 451-8146

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Not Applicable
Registry
ClinicalTrials.gov
Start date
25 October 2021
Est. completion
30 December 2026

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Disease progression as measured by clinical and molecular tests

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov