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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00645879 |
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Date of registration:
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25/03/2008 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Anaplerotic Therapy in Propionic Acidemia
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Scientific title:
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Safety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia. |
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Date of first enrolment:
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July 2008 |
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Target sample size:
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3 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00645879 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Nicola Longo, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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University of Utah |
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Key inclusion & exclusion criteria
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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
Age minimum:
5 Years
Age maximum:
12 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Propionic Acidemia
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Intervention(s)
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Drug: disodium citrate
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Drug: glutamine
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Drug: ornithine alpha ketoglutarate
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Primary Outcome(s)
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Define safety and efficacy of nutritional therapy with these investigational products: L-Ornithine alpha-ketoglutarate, Glutamine, or disodium citrate (anaplerotic therapy) on hyperammonemia and outcome in patients with propionic acidemia.
[Time Frame: end of study]
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Secondary Outcome(s)
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Define the effect of citrate, alpha-ketoglutarate and glutamine on plasma amino acids, acylcarnitines, ammonia, lactic acid and urine organic acids in patients with propionic acidemia.
[Time Frame: end of study]
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Evaluate the effect of investigational products on the developmental quotient and medical complications in patients with propionic acidemia.
[Time Frame: end of study]
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Secondary ID(s)
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24806
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1R21DK077415-01A1
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ANA-PA-001
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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