Clin2
NCT04602325Possibly a fitRecruiting

Blood and brain injury biomarker study for inherited metabolic disorders

Urea Cycle DisorderOrganic AcidemiaMaple Syrup Urine DiseaseGlutaric Acidemia IFatty Acid Oxidation DisorderHypoxic-Ischemic Encephalopathy

Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.

This trial studies blood and other measurable signs (biomarkers) that may reflect brain injury after certain inherited metabolic conditions. Results may help doctors better detect and understand brain effects from these illnesses, especially episodes that affect the brain.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
24 people
Ages
7 years to 18 years
Study type
Observational

Who can take part

  • You have an inherited disorder that causes high ammonia (urea cycle disorders like OTCD, CPSD, or NAGS)
  • OR you have an inherited organic acidemia (like propionic acidemia or methylmalonic acidemia)
  • OR you have had certain metabolic emergencies (like MSUD or glutaric acidemia) with or without brain effects
  • OR you had hypoxic-ischemic encephalopathy (brain injury from lack of oxygen/blood flow)
  • You have not had a solid-organ transplant
  • You are not using other experimental treatments that would interfere with measuring the study biomarkers

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

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