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

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.

Conditions

• Long-chain Fatty Acid Transport Deficiency

Interventions

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

Sponsors & Collaborators

• Fundación Ramón Domínguez

  collaborator OTHER

• Maria Luz Couce Pico

  lead OTHER

Principal Investigators

• Mª Luz Couce Pico · Hospital Clínico Universitario de Santiago

• Jaume Campistol Plana · Hospital Sant Joan de Déu. Barcelona

• Mercedes Martínez-Pardo · Hospital Universitario Ramon y Cajal

• Mónica Ruiz Pons · Hospital Clínico Universitario Ntra. Sra. de Candelaria. Tenerife

• Mª Teresa García Silva · Hospital 12 de Octubre. Madrid

• Pablo Sanjurjo · Hospital de Cruces de Baracaldo - Bilbao

• Koldo Aldamiz · Hospital de Cruces de Baracaldo - Bilbao

• Inmaculada García Jiménez · Hospital Universitario Miguel Servet, Zaragoza

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

CROSSOVER

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2009-11-30

Primary Completion

2010-03-31

Completion

2011-10-31

Countries

• Spain

Study Locations