Rare Disease Trial, Recruiting
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.
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
Where this trial is recruiting
Primary endpoints
Carbaglu related adverse events and adverse reactions
Can't join this trial?
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.