Overview

Anaplerotic Therapy in Propionic Acidemia

Status:
Completed
Trial end date:
2010-02-01
Target enrollment:
0
Participant gender:
All
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 improve hyperammonemia, glutamine levels, and outcome in patients with propionic acidemia. Ornithine alpha-ketoglutarate, glutamine, and citrate are commonly used as nutritional supplements specially by athletes to increase muscle strength. They can be mixed with formula or other foods.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Nicola Longo
Collaborators:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
National Institutes of Health (NIH)
Treatments:
Citric Acid
Sodium Citrate
Criteria
Inclusion Criteria:

- Confirmed diagnosis of propionic acidemia (propionyl CoA carboxylase deficiency)

Exclusion Criteria:

- Severe illness with cardiac or hepatic compromise that could affect study results

- Use of other investigative therapies

- Inability to comply with study directions