Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

Completed · Phase 2

Conditions studied: Urea Cycle Disorders, Organic Acidemias

In brief

This is a pilot study which will test the safety and feasibility of hypothermia treatment as adjunct therapy to conventional treatment of hyperammonemic encephalopathy (HAE) in neonates versus conventional treatment (dialysis, nutritional therapy, and ammonia scavenging drugs) only. The endpoint of the pilot study will be reached when either 24 patients have been enrolled and no serious adverse events were observed, when no patient has been enrolled in 5 years, or when serious adverse events occur which are clearly linked to the use of hypothermia. These would be serious complications not seen in patients on conventional therapy (dialysis , nutritional therapy, ammonia scavenging drugs) for HAE.

Key facts

Study ID

NCT01624311

Run by

Uta Lichter-Konecki

People needed

5

Starts

2007-08-01

Expected to finish

2015-05-01

Last updated by the study team

2015-06-01

Who can join

Age: 0 and older, up to 0. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Newborns >36 wks gestation and ≥2200g birth weight without co-morbidity (see exclusion criteria) that have clinical signs and symptoms of an Urea Cycle Disorders or Propionic , Methylmalonic, or Isovaleric acidemia and hyperammonemia and encephalopathy requiring renal replacement therapy.

You may not qualify if…

• Patients with hyperammonemia that have clinical signs and symptoms of lysinuric protein intolerance, mitochondrial disorders, congenital lactic acidosis, and fatty acid oxidation disorders, patients with rare and unrelated serious comorbidities and other genetic diseases, e.g., Down syndrome, intraventricular hemorrhage in the newborn period, traumatic brain injury, and low birth weight (<2,200 g at >36 wks gestation).
• Infants in extremis for which no additional intensive therapy will be offered by the attending neonatologist.

Where it is running

• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Columbia University, Morgan Stanley Children's Hospital — New York, New York, United States
• Medical College Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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