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

NCT03734263 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2021-10-13

No results posted yet for this study

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.

Conditions

  • Pyruvate Dehydrogenase Complex Deficiency

Interventions

DRUG

sodium phenylbutyrate

Enrolled subjects will receive a four-week period of treatment with sodium phenylbutyrate (oral use)

Sponsors & Collaborators

  • Fondazione Telethon

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-10-01
Primary Completion
2019-07-30
Completion
2020-12-30

Countries

  • Italy

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03734263 on ClinicalTrials.gov