Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. — TIGEM2-PDH

Pyruvate Dehydrogenase Complex Deficiency Clinical Trial

Official title:
Pilot Clinical Trial to Investigate the Safety and Efficacy of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.

Status Completed

Clinical Trial Summary

In this study phenylbutyrate is used for patients with pyruvate dehydrogenase complex deficiency. The aim of the study is to investigate the safety and efficacy of therapy.

Clinical Trial Description

The Investigator will evaluate the safety and efficacy of a 4-weeks treatment with sodium phenylbutyrate in patients with pyruvate dehydrogenase complex deficiency. Efficacy will be evaluated based on biochemical endpoints (blood lactate and pyruvate). ;

Study Design

Related Conditions & MeSH terms

Pyruvate Dehydrogenase Complex Deficiency
Pyruvate Dehydrogenase Complex Deficiency Disease

Clinical Trial Details

NCT number	NCT03734263
Study type	Interventional
Source	Fondazione Telethon
Contact
Status	Completed
Phase	Phase 2
Start date	October 1, 2018
Completion date	December 30, 2020

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05257005` - Natural History Study of Pyruvate Dehydrogenase Deficiency
Recruiting	`NCT06340685` - Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency	Phase 1
Active, not recruiting	`NCT02616484` - Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:	Phase 3