Trials / Completed
CompletedNCT03734263
Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
Pilot Clinical Trial to Investigate the Safety and Efficacy of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency.
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- Fondazione Telethon · Academic / Other
- Sex
- All
- Age
- 3 Months – 18 Years
- Healthy volunteers
- Not accepted
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.
Detailed 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).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | sodium phenylbutyrate | Enrolled subjects will receive a four-week period of treatment with sodium phenylbutyrate (oral use) |
Timeline
- Start date
- 2018-10-01
- Primary completion
- 2019-07-30
- Completion
- 2020-12-30
- First posted
- 2018-11-07
- Last updated
- 2021-10-13
Locations
1 site across 1 country: Italy
Source: ClinicalTrials.gov record NCT03734263. Inclusion in this directory is not an endorsement.