NCT02201368

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.

Trial Health

30
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Nov 2009

Geographic Reach
1 country

1 active site

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2009

Completed
4 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2010

Completed
1.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2011

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

March 22, 2013

Completed
1.4 years until next milestone

First Posted

Study publicly available on registry

July 28, 2014

Completed
Last Updated

July 28, 2014

Status Verified

July 1, 2014

Enrollment Period

4 months

First QC Date

March 22, 2013

Last Update Submit

July 25, 2014

Conditions

Long-chain Fatty Acid Transport Deficiency

Keywords

Triglycerides; Lipid Metabolism; Fatty Acids.

Outcome Measures

Primary Outcomes (1)

  • Number of metabolic decompensation.

    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.

    up to 16 months

Secondary Outcomes (3)

  • Differences in the profiles of acylcarnitines with control.

    6 months and 6 months in each arm treatment

  • Average values of transaminase and creatin kinase.

    6 months and 6 months in each arm treatment

  • Differences in the fatty acid composition of plasma and red blood cells.

    6 months and 6 months in each arm treatment

Study Arms (2)

Triheptanoin

EXPERIMENTAL
Drug: Triheptanoin (SpezialölÒ 107®)Dietary Supplement: MCT (Medium-Chain Triglycerides)

MCT (Medium-Chain Triglycerides)

ACTIVE COMPARATOR
Drug: Triheptanoin (SpezialölÒ 107®)Dietary Supplement: MCT (Medium-Chain Triglycerides)

Interventions

Triheptanoin (SpezialölÒ 107®)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)Triheptanoin
MCT (Medium-Chain Triglycerides)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)Triheptanoin

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • No patient/family collaboration or the application of dietary treatment.
  • No in vitro test response.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hospital Clínico Universitario de Santiago

Santiago de Compostela, Spain

Location

MeSH Terms

Interventions

triheptanoin

Study Officials

  • Mª Luz Couce Pico

    Hospital Clínico Universitario de Santiago

    STUDY CHAIR

  • 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

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

March 22, 2013

First Posted

July 28, 2014

Study Start

November 1, 2009

Primary Completion

March 1, 2010

Study Completion

October 1, 2011

Last Updated

July 28, 2014

Record last verified: 2014-07

Locations

ES(1)