Rare Disease Trial, Recruiting NCT03409003 Sponsor: Nicholas Ah Mew Condition: Rare Disease
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Rare Disease Trial, Recruiting

NCT03409003
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 have been diagnosed with, or are suspected to have, a condition called NAGS deficiency (a rare disorder affecting how the body processes protein).
  • You are currently taking a medication called Carbaglu (carglumic acid) to treat NAGS deficiency.
  • You are already enrolled in a separate long-term research study about urea cycle disorders (known as RDCRN protocol #5101) (confirm with trial site).

Who may not be able to join:

  • Your high ammonia levels are caused by a different urea cycle disorder, not NAGS deficiency.
  • You have a condition called organic acidemia or lysinuric protein intolerance (disorders affecting how the body breaks down certain nutrients).
  • You have a mitochondrial disorder (a condition affecting how cells produce energy).
  • You have a condition called congenital lactic acidemia (a disorder where too much lactic acid builds up in the body from birth).
  • You have a fatty acid oxidation defect (a condition where the body cannot properly break down fats for energy).
  • You have a primary liver disease (a condition where the liver itself is the main source of the problem).
  • You were born weighing less than 1,500 grams (about 3.3 pounds), which is considered extremely low birth weight.

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: Nicholas Ah Mew, MD, Children's National Research Institute

Phone: 2023066489

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
Nicholas Ah Mew
Registry
ClinicalTrials.gov
Start date
1 April 2012
Est. completion
1 July 2026

Where this trial is recruiting

🇺🇸 United States

Primary endpoints

Carbaglu related adverse events and adverse reactions

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