Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants
|ClinicalTrials.gov Identifier: NCT01624311|
Recruitment Status : Completed
First Posted : June 20, 2012
Last Update Posted : June 1, 2015
|Condition or disease||Intervention/treatment||Phase|
|Urea Cycle Disorders Organic Acidemias||Other: Therapeutic Hypothermia Other: Standard of Care Therapy||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Hypothermia Treatment in Hyperammonemia and Encephalopathy|
|Study Start Date :||August 2007|
|Actual Primary Completion Date :||May 2015|
|Actual Study Completion Date :||May 2015|
Experimental: Adjunct Hypothermia Arm
Patients that receive adjunct therapeutic hypothermia in addition to standard of care therapy
Other: Therapeutic Hypothermia
After obtaining written consent, patients are cooled to 33.5°C (+/- 1°C ) for 72 hours and and then rewarmed by 0.5°C per every 3 hours over 18 hours.
Patients that were treated with standard of care therapy for the same conditions at the sponsoring institution over the past 10 years.
Other: Standard of Care Therapy
Patients with hyperammonemia and encephalopathy requiring renal replacement therapy due to a urea cycle disorder or organic acidemia that were treated at Children's National Medical Center over the past 10 years.
- Number of Participants with unexpected Serious Adverse Events as a Measure of Safety and Tolerability [ Time Frame: Participants will be followed for the duration of the hospital stay, an expected average of 5 weeks ]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.
- Feasibility of hypothermia therapy as adjunct therapy to the complex standard of care therapy [ Time Frame: During the first 72h of treatment ]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.
- Time to normalization of ammonia level [ Time Frame: During the first 72 hours of treatment ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01624311
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, New York|
|Columbia University, Morgan Stanley Children's Hospital|
|New York, New York, United States, 10027|
|United States, Wisconsin|
|Medical College Wisconsin|
|Milwaukee, Wisconsin, United States, 53226|
|Principal Investigator:||Uta Lichter-Konecki, MD, PhD||Columbia University|