Condition: Pyruvate Dehydrogenase Complex Deficiency Disease · Sponsor: Assistance Publique - Hôpitaux de Paris
This is a phase II, multicenter, prospective, non-comparative clinical trial to assess the efficacy and safety of the treatment of pyruvate dehydrogenase deficiency (PDH) patients with glycerol phenylbutyrate (Ravicti®). The trial will be conducted with three visits: 3 day hospitalizations including clinical consultations and paramedical procedures at Month 0 (M0), Month 3 (M3), Month 6 (M6). During all the research, AE/SAE and treatment compliance will be recorded. Patients will keep their usual treatment during the study time: vitamin B1, ketogenic diet, possible anti-epileptic and/or dystonic treatment(s). The efficacy on fatigue, polyhandicap, neurodevelopmental functioning, quality of life and seizure amount for epileptic patients will be evaluated at 0, 3 and 6 months. Biological balance will be assed with regular quantification of PDH deficiency markers, lactate concentration and amino acid plasma quantification.
This description comes directly from the study's public registry record.
Pascale De Lonlay, MD, PhD · 01 44 49 58 52 · pascale.delonlay@aphp.fr
Gael Plastow, Project advisor · 01 44 38 18 57 · gael.plastow@aphp.fr
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| Hôpital Universitaire Necker - Enfants Malades | Paris, France, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06887777