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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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21 December 2021 |
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Main ID: |
NCT01461304 |
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Date of registration:
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25/10/2011 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Dietary Therapy for Inherited Disorders of Energy Metabolism
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Scientific title:
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Dietary Therapy for Inherited Disorders of Energy Metabolism |
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Date of first enrolment:
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April 17, 2009 |
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Target sample size:
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Recruitment status: |
No longer available |
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URL:
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https://clinicaltrials.gov/show/NCT01461304 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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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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Jerry Vockley, 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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Children's Hospital of Pittsburgh, University of Pittsburgh |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Ages 1 month and up
- Diagnosis of disorder in long chain fatty acid oxidation, glycogen storage disease,
pyruvate carboxylase deficiency, or Barth Syndrome
- Currently receiving triheptanoin as result of participation in previous study will be
eligible if they have one of the included diagnoses
- Prefer 2 of following 3: acylcarnitine profile, fibroblast acylcarnitine profile or
positive medical genetic test
Exclusion Criteria:
- Pregnant females
- MCAD deficiency
- disorder of short and medium chain fatty acid oxidation or ketone body metabolism
Age minimum:
1 Month
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Pyruvate Carboxylase Deficiency Disease
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Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency
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Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency
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ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of
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Mitochondrial Trifunctional Protein Deficiency
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Glycogen Storage Disorders
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Barth Syndrome
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Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)
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Intervention(s)
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Drug: triheptanoin
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Secondary ID(s)
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PRO08020019
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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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