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

Long-chain Fatty Acid Transport Deficiency Clinical Trial

Official title:

Study to Evaluate the Effectiveness of Dietary Treatment With Triheptanoin in Patients With Long-chain Fatty Acid Beta-oxidation Defects

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02201368
• Other study ID #: 2007/084
• Secondary ID: 2007-005578-29
• Status: Withdrawn
• Phase: Phase 3
• First received: March 22, 2013
• Last updated: July 25, 2014
• Start date: November 2009
• Est. completion date: October 2011

Study information

• Verified date: July 2014
• Source: Hospital Clinico Universitario de Santiago
• Contact: n/a
• Is FDA regulated: No
• Health authority: Spain: Ethics CommitteeSpain: Spanish Agency of Medicines
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 2011
• Est. primary completion date: March 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Long-chain Fatty Acid Transport Deficiency

Intervention

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

Locations

Country	Name	City	State
Spain	Hospital Clínico Universitario de Santiago	Santiago de Compostela

Sponsors (2)

Lead Sponsor	Collaborator
Maria Luz Couce Pico	Fundación Ramón Domínguez

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Secondary	Differences in the profiles of acylcarnitines with control.		6 months and 6 months in each arm treatment	No
Secondary	Average values of transaminase and creatin kinase.		6 months and 6 months in each arm treatment	No
Secondary	Differences in the fatty acid composition of plasma and red blood cells.		6 months and 6 months in each arm treatment	No