PDH-RAVICTI - A PHASE II, MULTICENTRIC, PROSPECTIVE, NON-COMPARATIVE CLINICAL TRIAL TO ASSESS THE EFFICACY AND SAFETY OF THE TREATMENT OF PYRUVATE DEHYDROGENASE DEFICIENCY (PDH) PATIENTS WITH GLYCEROL PHENYLBUTYRATE (RAVICTI®)

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2024-516410-38-00

Acronym

APHP230834

Enrollment

Registered

2025-04-22

Start date

2025-10-01

Completion date

Unknown

Last updated

2025-12-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pyruvate dehydrogenase deficiency

Brief summary

The efficacy of Glycerol Phenylbutyrate treatment on fatigue at 6 months will be evaluated by the Pediatric Quality of Life Inventory™ Multidimensional Fatigue Scale (PedsQL™ MFS, appendix 4 to 6). Difference between the total score at M0 and M6 will be calculated. Improving the overall score of 20% between M0 and M6 will reflect the impact of treatment on fatigue (success of the treatment).

Interventions

DRUGRAVICTI 1.1 g/ml oral liquid

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
The efficacy of Glycerol Phenylbutyrate treatment on fatigue at 6 months will be evaluated by the Pediatric Quality of Life Inventory™ Multidimensional Fatigue Scale (PedsQL™ MFS, appendix 4 to 6). Difference between the total score at M0 and M6 will be calculated. Improving the overall score of 20% between M0 and M6 will reflect the impact of treatment on fatigue (success of the treatment).	—

Countries

France

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026