Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochond… (NCT02023866) | Clinical Trial Compass
CompletedPhase 2
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
United States36 participantsStarted 2014-05
Plain-language summary
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.
Who can participate
Age range6 Years – 17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Age ≥ 6 years and \< 18 years
✓. Body weight ≥ 5 kg
✓. Documented (genetically confirmed known mutation, i.e. no variants of uncertain significance) diagnosis of inherited mitochondrial disease other than Friedreich's ataxia (FRDA)
✓. Moderate disease severity based on Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) score, with a score between 15 to 45 inclusive \[Leber's Hereditary Optic Neuropathy (LHON) subjects are exempt of this inclusion criteria\], if approved by the sponsor.
✓. For patients regularly taking dietary supplements such as creatinine, alpha-lipoic acid, coenzyme Q10 (CoQ10), vitamin B, carnitine, etc. they have to have been taking them for at least 3 months pre-study and will agree to keep these the same throughout the study (from the Screening Visit to Study Exit)
✓. With respect to concomitant medications, the subject must:
✓. Be willing to abstain from initiating dietary supplements and non-prescribed medications, except as allowed by the Investigator, throughout the study (from the Screening Visit to Study Exit);
✓. Be on a stable dose of medications prescribed for seizure management and prevention. Stable dose in this context means unchanged for at least 30 days prior to the Screening Visit.
Exclusion criteria
✕. Documented diagnosis of concurrent inborn errors of metabolism
✕. Non-elective hospitalization related to mitochondrial disease or direct complication of disease within 60 days prior to the Screening Visit.
What they're measuring
1
Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV
✕. Platelet count, lymphocyte count or hemoglobin below the lower limit of normal (LLN) at the Screening Visit
✕. Hepatic insufficiency with liver enzyme tests (alkaline phosphatase, aspartate aminotransferase \[AST\] or alanine aminotransferase \[ALT\]) greater than 2.5 times the upper limit of normal (ULN) at the Screening Visit
✕. Bilirubin \> 1.2 g/dL at the Screening Visit
✕. Inability to complete the elements of the study, e.g., coma, hemodynamic instability or requiring continuous ventilator support.
✕. Malabsorption requiring total parenteral nutrition (TPN), chronic diarrhea, bouts of pseudo obstruction
✕. Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in lactic acidosis