Urea Cycle Disorders Clinical Trial
Official title:
Hypothermia Treatment in Hyperammonemia and Encephalopathy
Verified date | May 2015 |
Source | Columbia University |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Institutional Review Board |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 5 |
Est. completion date | May 2015 |
Est. primary completion date | May 2015 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 30 Days |
Eligibility |
Inclusion Criteria: - 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. Exclusion Criteria: - 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. |
Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | Medical College Wisconsin | Milwaukee | Wisconsin |
United States | Columbia University, Morgan Stanley Children's Hospital | New York | New York |
United States | Children's National Medical Center | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Uta Lichter-Konecki | Children's Research Institute, Medical College of Wisconsin |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Participants with unexpected Serious Adverse Events as a Measure of Safety and Tolerability | The DSMB meets within 3 weeks of each case, no later than 5 weeks after initiation of hypothermia therapy and assesses the safety and feasibility of adjunct hypothermia treatment in this patient group. The treatment of neonates and very young infants in hyperammonemic coma is very complex and adding hypothermia therapy to this treatment could not be feasible, the pilot study therefore also assesses the feasibility of adding hypothermia therapy to the standard of care treatment. | Participants will be followed for the duration of the hospital stay, an expected average of 5 weeks | Yes |
Primary | Feasibility of hypothermia therapy as adjunct therapy to the complex standard of care therapy | The standard of care therapy is very complex. It includes renal replacement therapy, metabolic diet intervention, and ammonia scavenger use. The pilot study will assess primarily for the first 72 hours of treatment and secondarily for the duration of the hospital stay, an expected average of 5 weeks, whether adding hypothermia to this already complex treatment is feasible. | During the first 72h of treatment | No |
Secondary | Time to normalization of ammonia level | One of the effects of hypothermia treatment is an overall slowing of metabolism which should cause less ammonia to be produced. If this is correct this should lead to a faster normalization of the ammonia level by renal replacement therapy. | During the first 72 hours of treatment | No |
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