Spinal Muscular Atrophy Trial, Recruiting NCT04708431 Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Condition: Spinal Muscular Atrophy
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Spinal Muscular Atrophy Trial, Recruiting

NCT04708431
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:

  • You are between 8 and 99 years old and have been diagnosed with androgen insensitivity syndrome (AIS), either through a genetic test showing a change in the androgen receptor gene, or through a clinical diagnosis based on specific physical and biological signs
  • You identify as male or female
  • You have any form of AIS — complete, partial, or mild forms are all eligible
  • You are willing and able to follow all study requirements for the full length of the study
  • You (or your parent/guardian if you are a child) are able to understand and sign a consent form agreeing to take part
  • Adult family members of someone with AIS may also be eligible to participate

Who may not be able to join:

  • People who have a similar but different condition, such as partial or complete gonadal dysgenesis, 5-alpha reductase deficiency, or another reason for a 46 XY difference of sexual development that is not AIS (confirm with trial site)
  • People who have serious medical conditions that are not related to hormone or endocrine issues
  • Adult relatives of AIS patients who have serious non-endocrine medical conditions

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: Veronica Gomez-Lobo, M.D., Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Phone: (301) 435-7567

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
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Registry
ClinicalTrials.gov
Start date
29 April 2021
Est. completion
1 February 2040

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

To define and describe a comprehensive phenotype in 500 patients with androgen insensitivity

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