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Trial record 6 of 76 for:    Long-chain fatty acids

Triheptanoin Treatment Trial for Patients With Long-chain Fatty Acid Beta-oxidation Defects

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ClinicalTrials.gov Identifier: NCT02201368
Recruitment Status : Withdrawn (IMP management difficulties)
First Posted : July 28, 2014
Last Update Posted : July 28, 2014
Sponsor:
Collaborator:
Fundación Ramón Domínguez
Information provided by (Responsible Party):
Maria Luz Couce Pico, Hospital Clinico Universitario de Santiago

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 treatment and follow-up will be 16 months.

Condition or disease Intervention/treatment Phase
Long-chain Fatty Acid Transport Deficiency Drug: Triheptanoin (SpezialölÒ 107®) Dietary Supplement: MCT (Medium-Chain Triglycerides) Phase 3

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, reducing symptoms and serious complications of their disease, like cardiomyopathy, Reye-like syndrome episodes and rhabdomyolysis. This result would occur by the effect of propionyl CoA primer on the Krebs cycle and, at the same time, would produce a gluconeogenic effect.

This treatment opens the door to be used in other diseases such as pyruvate carboxylase deficiency, glycogen storage disease and other diseases with energy problems.

All patients will be followed up until 16 months.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects
Study Start Date : November 2009
Actual Primary Completion Date : March 2010
Actual Study Completion Date : October 2011

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Triheptanoin Drug: Triheptanoin (SpezialölÒ 107®)
  • Randomization: Treatment with Triheptanoin for 6 months
  • Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
  • Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months

Dietary Supplement: MCT (Medium-Chain Triglycerides)
  • Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
  • Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
  • Crossover: Treatment with Triheptanoin for 6 months

Active Comparator: MCT (Medium-Chain Triglycerides) Drug: Triheptanoin (SpezialölÒ 107®)
  • Randomization: Treatment with Triheptanoin for 6 months
  • Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
  • Crossover: Treatment with MCT(Medium-Chain Triglycerides)for 6 months

Dietary Supplement: MCT (Medium-Chain Triglycerides)
  • Randomization: Treatment with MCT(Medium-Chain Triglycerides)for 6 months
  • Washout Period: Treatment with MCT(Medium-Chain Triglycerides)for 2 months
  • Crossover: Treatment with Triheptanoin for 6 months




Primary Outcome Measures :
  1. Number of metabolic decompensation. [ Time Frame: up to 16 months ]
    This is a combined endpoint, including the number and/or severity of episodes of hypoglycemia, rhabdomyolysis, cardiomyopathy and liver failure after starting treatment with trihepatnoin.


Secondary Outcome Measures :
  1. Differences in the profiles of acylcarnitines with control. [ Time Frame: 6 months and 6 months in each arm treatment ]
  2. Average values of transaminase and creatin kinase. [ Time Frame: 6 months and 6 months in each arm treatment ]
  3. Differences in the fatty acid composition of plasma and red blood cells. [ Time Frame: 6 months and 6 months in each arm treatment ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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 Criteria:

  • No patient/family collaboration or the application of dietary treatment.
  • No in vitro test response.
  • Do not meet the inclusion criteria.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02201368


Locations
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Spain
Hospital Clínico Universitario de Santiago
Santiago de Compostela, Spain
Sponsors and Collaborators
Maria Luz Couce Pico
Fundación Ramón Domínguez
Investigators
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Study Chair: Mª Luz Couce Pico Hospital Clínico Universitario de Santiago
Principal Investigator: Jaume Campistol Plana Hospital Sant Joan de Déu. Barcelona
Principal Investigator: Mercedes Martínez-Pardo Hospital Universitario Ramon y Cajal
Principal Investigator: Mónica Ruiz Pons Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife
Principal Investigator: Mª Teresa García Silva Hospital 12 de Octubre. Madrid
Principal Investigator: Pablo Sanjurjo Hospital de Cruces de Baracaldo - Bilbao
Principal Investigator: Koldo Aldamiz Hospital de Cruces de Baracaldo - Bilbao
Principal Investigator: Inmaculada García Jiménez Hospital Universitario Miguel Servet - Zaragoza

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Responsible Party: Maria Luz Couce Pico, MD, Hospital Clinico Universitario de Santiago
ClinicalTrials.gov Identifier: NCT02201368     History of Changes
Other Study ID Numbers: 2007/084
2007-005578-29 ( EudraCT Number )
First Posted: July 28, 2014    Key Record Dates
Last Update Posted: July 28, 2014
Last Verified: July 2014

Keywords provided by Maria Luz Couce Pico, Hospital Clinico Universitario de Santiago:
Triglycerides; Lipid Metabolism; Fatty Acids.