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

NCT ID: NCT02201368

Last Updated: 2014-07-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE3

Study Classification

INTERVENTIONAL

Study Start Date

2009-11-30

Study Completion Date

2011-10-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Long-chain Fatty Acid Transport Deficiency

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Triheptanoin

Group Type EXPERIMENTAL

Triheptanoin (SpezialölÒ 107®)

Intervention Type DRUG

* 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

MCT (Medium-Chain Triglycerides)

Intervention Type DIETARY_SUPPLEMENT

* 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

MCT (Medium-Chain Triglycerides)

Group Type ACTIVE_COMPARATOR

Triheptanoin (SpezialölÒ 107®)

Intervention Type DRUG

* 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

MCT (Medium-Chain Triglycerides)

Intervention Type DIETARY_SUPPLEMENT

* 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

Interventions

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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

Intervention Type DRUG

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

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fundación Ramón Domínguez

OTHER

Sponsor Role collaborator

Maria Luz Couce Pico

OTHER

Sponsor Role lead

Responsible Party

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Maria Luz Couce Pico

MD

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Mª Luz Couce Pico

Role: STUDY_CHAIR

Hospital Clínico Universitario de Santiago

Jaume Campistol Plana

Role: PRINCIPAL_INVESTIGATOR

Hospital Sant Joan de Déu. Barcelona

Mercedes Martínez-Pardo

Role: PRINCIPAL_INVESTIGATOR

Hospital Universitario Ramon y Cajal

Mónica Ruiz Pons

Role: PRINCIPAL_INVESTIGATOR

Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife

Mª Teresa García Silva

Role: PRINCIPAL_INVESTIGATOR

Hospital 12 de Octubre. Madrid

Pablo Sanjurjo

Role: PRINCIPAL_INVESTIGATOR

Hospital de Cruces de Baracaldo - Bilbao

Koldo Aldamiz

Role: PRINCIPAL_INVESTIGATOR

Hospital de Cruces de Baracaldo - Bilbao

Inmaculada García Jiménez

Role: PRINCIPAL_INVESTIGATOR

Hospital Universitario Miguel Servet, Zaragoza

Locations

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Hospital Clínico Universitario de Santiago

Santiago de Compostela, , Spain

Site Status

Countries

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Spain

Other Identifiers

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2007-005578-29

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2007/084

Identifier Type: -

Identifier Source: org_study_id

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