Urea Cycle Disorders Clinical Trial
Official title:
Hypothermia Treatment in Hyperammonemia and Encephalopathy
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.
Children with neonatal onset Urea Cycle Disorders or Organic Acidemias develop hyperammonemia (high ammonia levels) and fall into coma often causing brain damage. For these children to be able to benefit maximally from available long-term treatment and solid organ transplant, outcome of the neonatal onset crisis must be improved. Animal experiments and small clinical trials have indicated that hypothermia protects the brain during hyperammonemia. This pilot study investigates whether adjunct hypothermia therapy in addition to standard of care treatment is feasible and safe in babies with high ammonia levels in coma. ;
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02252770 -
Nitric Oxide Supplementation in Argininosuccinic Aciduria
|
N/A | |
| Completed |
NCT01002469 -
Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment
|
N/A | |
| Completed |
NCT00986895 -
A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group
|
Phase 1 | |
| Completed |
NCT01257737 -
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
|
Phase 4 | |
| Recruiting |
NCT05671666 -
Ureagenesis Analysis in Healthy Subjects and in Urea Cycle Disorder Patients
|
N/A | |
| Completed |
NCT02311283 -
Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
|
N/A | |
| Completed |
NCT02489292 -
Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
|
Phase 2 | |
| Completed |
NCT00992459 -
Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders
|
Phase 3 | |
| Completed |
NCT00551200 -
Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders
|
Phase 2 | |
| Completed |
NCT00947297 -
Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)
|
Phase 3 | |
| Completed |
NCT02740153 -
PCORI Urea Cycle Disorder Study
|
||
| Completed |
NCT02051049 -
Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)
|
||
| Completed |
NCT01765283 -
Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)
|
Phase 1/Phase 2 | |
| Completed |
NCT00947544 -
Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
|
Phase 2 | |
| Completed |
NCT01347073 -
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
|
Phase 3 | |
| Completed |
NCT00718627 -
Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders
|
Phase 2 | |
| Completed |
NCT03797131 -
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
|
N/A | |
| Completed |
NCT01549015 -
Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function
|
N/A | |
| Terminated |
NCT01541722 -
Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders
|
N/A | |
| Completed |
NCT00345605 -
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
|
Phase 2 |