Status:
COMPLETED
Anaplerotic Therapy in Propionic Acidemia
Lead Sponsor:
Nicola Longo
Collaborating Sponsors:
National Institutes of Health (NIH)
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Conditions:
Propionic Acidemia
Eligibility:
All Genders
5-12 years
Phase:
PHASE1
Brief Summary
The objective of this project is to define whether nutritional supplements (ornithine alpha-ketoglutarate, glutamine, or citrate) capable of filling-up the citric acid cycle (anaplerotic therapy) can ...
Detailed Description
Propionic acidemia is caused by deficiency of propionyl CoA carboxylase that impairs the supply of succinyl CoA to the citric acid (Krebs) cycle. The Krebs cycle is responsible for obtaining energy fr...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of propionic acidemia (propionyl CoA carboxylase deficiency)
Exclusion
- Severe illness with cardiac or hepatic compromise that could affect study results
- Use of other investigative therapies
- Inability to comply with study directions
Key Trial Info
Start Date :
July 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2010
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00645879
Start Date
July 1 2008
End Date
February 1 2010
Last Update
January 21 2015
Active Locations (1)
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1
University of Utah, Department of Pediatrics
Salt Lake City, Utah, United States, 84132