Who is sponsoring NCT03734263?

NCT03734263 is sponsored by Fondazione Telethon.

What drugs are being tested in NCT03734263?

Interventions in NCT03734263: sodium phenylbutyrate.

What condition does NCT03734263 study?

NCT03734263 studies Pyruvate Dehydrogenase Complex Deficiency.

Is NCT03734263 still recruiting?

NCT03734263 is currently completed. Last updated 13 October 2021.

Last reviewed 2021-10-13 · How we verify

NCT03734263: TIGEM2-PDH

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.

Completed Phase 2 Last updated 13 October 2021

What this trial tests

Phase 2 trial testing sodium phenylbutyrate in Pyruvate Dehydrogenase Complex Deficiency in 1 participant. Completed in 30 December 2020.

Timeline

1 October 2018

Primary endpoint
30 July 2019

30 December 2020

Quick facts

Lead sponsor	Fondazione Telethon
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	1
Start date	1 October 2018
Primary completion	30 July 2019
Estimated completion	30 December 2020
Sites	1 location across Italy

Drugs / interventions tested

sodium phenylbutyrate — full drug profile →

Conditions studied

Pyruvate Dehydrogenase Complex Deficiency — all drugs for Pyruvate Dehydrogenase Complex Deficiency →

Sponsor

Fondazione Telethon — full company profile →

Who can join

Adults 3 Months to 18, any sex, with Pyruvate Dehydrogenase Complex Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

Mitochondrial diseases: from molecular mechanisms to therapeutic advances.
Wen H, Deng H, Li B, Chen J, et al · · 2025 · cited 111× · PMID 39788934 · DOI 10.1038/s41392-024-02044-3
Moving towards clinical trials for mitochondrial diseases.
Pitceathly RDS, Keshavan N, Rahman J, Rahman S. · · 2021 · cited 52× · PMID 32618366 · DOI 10.1002/jimd.12281
Amino acid ratio combinations as biomarkers for discriminating patients with pyruvate dehydrogenase complex deficiency from other inborn errors of metabolism.
Verma A, Lehman AN, Gokcan H, Cropcho L, et al · · 2024 · cited 4× · PMID 37688338 · DOI 10.1002/mgg3.2283
Therapies for Mitochondrial Disease: Past, Present, and Future.
Ball M, van Bergen NJ, Compton AG, Thorburn DR, et al · · 2025 · cited 3× · PMID 40714961 · DOI 10.1002/jimd.70065

Verify or expand the search:

Other trials of sodium phenylbutyrate

Trials testing the same drug.

NCT05028803 — The Effects of Sodium Phenylbutyrate on Circulating Branched Chain Amino Acids · NA · unknown

Other recruiting trials for Pyruvate Dehydrogenase Complex Deficiency

Currently open trials in the same condition.

NCT06340685 — Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency · Phase 1 · recruiting
NCT02616484 — Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: · Phase 3 · active not recruiting

Other Fondazione Telethon trials

Trials by the same sponsor.

NCT04959890 — Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy · unknown
NCT03311074 — Retroviral Insertion Site Methodology Study · Phase 4 · withdrawn
NCT03814499 — Natural History Study in Subjects With Usher Syndrome · completed
NCT03232203 — Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs) · completed
NCT03173521 — Gene Therapy in Patients With Mucopolysaccharidosis Disease · Phase 1, PHASE2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03734263 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Fondazione Telethon
Last refreshed: 13 October 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03734263.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing