A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delay… (NCT02473445) | Clinical Trial Compass
TerminatedPhase 2
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
Stopped: Sponsor decision to end development of RP103 for mitochondrial disease due to lack of efficacy demonstrated in base study RP103-MITO-001.
United States22 participantsStarted 2015-05-19
Plain-language summary
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study RP103-MITO-001 (NCT02023866).
Who can participate
Age range6 Years – 17 Years
SexALL
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Inclusion criteria
✓. Completed all visits in Study RP103-MITO-001 (NCT02023866).
✓. Body weight ≥ 5 kg.
✓. 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 Day 1 to Study Exit).
✓. Willing and able to comply with study drug dosing requirements, i.e. ingest the RP103 capsules intact, or sprinkled in liquid or soft food, or using a G-tube.
✓. Sexually active female subjects of childbearing potential (i.e., not surgically sterile \[tubal ligation, hysterectomy, or bilateral oophorectomy\]) must agree to utilize two of the following acceptable forms of contraception throughout the study (from Day 1 to Study Exit):
✓. Patient's legally authorized representative must provide written informed consent; Patient must provide assent, if required by local/institutional requirements.
Exclusion criteria
✕. Documented diagnosis of concurrent inborn errors of metabolism.
✕
What they're measuring
1
Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score
Timeframe: Baseline, every 3 months and Study Exit (up to 24 Months)
. Platelet count, lymphocyte count or hemoglobin below the lower limit of normal (LLN) at the Baseline 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 Baseline Visit.
✕. Bilirubin \> 1.2 g/dL at the Baseline 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.