Status:
WITHDRAWN
Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects
Lead Sponsor:
Maria Luz Couce Pico
Collaborating Sponsors:
Fundación Ramón Domínguez
Conditions:
Long-chain Fatty Acid Transport Deficiency
Eligibility:
All Genders
Phase:
PHASE3
Brief Summary
The purpose of this study is to determine if administration Triheptanoin is an effective treatment for defects of the long-chain fatty acid beta-oxidation in young adults or adults. Period of treatmen...
Detailed Description
Triheptanoin treatment, in patients with long-chain fatty acid beta-oxidation defects, could cause not only a great improvement in their quality of life, also could prevent life-threatening signs, red...
Eligibility Criteria
Inclusion
- All patients with any of the following conditions:
- Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (LCHAD).
- Very long-chain acyl-coenzyme A dehydrogenase deficiency (VLCAD.)
- Mitochondrial trifunctional protein (MTP).
- Carnitine palmitoyltransferase I deficiency (CPT I).
- Carnitine Palmitoyltransferase II (CPT II).
- Carnitine-acylcarnitine translocase deficiency (CACT).
- Positive skin biopsy: patients were deemed to commence the dietary treatment with Triheptanoin after evaluating the individual response in vitro in cultured fibroblasts. This response is based on the measurement of the production of propionyl-CoA, the incubation with fatty acids odd-chain, compared with control group fibroblasts.
- The informed consent must be signed by the patient or family, in the case of minors.
Exclusion
- No patient/family collaboration or the application of dietary treatment.
- No in vitro test response.
- Do not meet the inclusion criteria.
Key Trial Info
Start Date :
November 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2011
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT02201368
Start Date
November 1 2009
End Date
October 1 2011
Last Update
July 28 2014
Active Locations (1)
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1
Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain