Phase 2
N=36
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Inherited Mitochondrial Disease, Including Leigh Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02023866 ↗Enrolled (actual)
36
Serious AEs
30.6%
Results posted
Nov 2017
Primary outcome: Primary: Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV — -0.3; 0.1; -0.6; 0.0 units on a scale — p=0.1875
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Cysteamine Bitartrate (Drug)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV |
-0.3; 0.1; -0.6; 0.0 | 0.1875 |
| SECONDARY Change From Baseline in Glutathione |
16.8; 141.5; 10.2; 88.7; 51.4; 122.4 | — |
| SECONDARY Change From Baseline in Glutathione Disulfide |
29.4; -9.8; 18.8; -11.2; 0.8; -11.5 | — |
| SECONDARY Change From Baseline in Lactic Acid |
0.2; 0.4; 0.3; 0.1; 0.1; 0.0 | — |
| SECONDARY Change From Baseline in 6 Minute Walk Test |
-23.5; -36.2; -14.5; -21.3; 21.3; -18.9 | — |
| SECONDARY Change From Baseline in Jamar Dynamometer Hand Strength |
1.3; 1.1; 1.3; 0.0; 0.7; 1.3 | — |
| SECONDARY Change From Baseline in Barry-Albright Dystonia Scale Total Score |
1.3; 0.7; 0.3; 0.3; -0.7; 0.7 | — |
| SECONDARY Change From Baseline in Friedreich Ataxia Rating Scale |
16.9; 13.9; 15.9; 19.4; 16.5; 20.5 | — |
| SECONDARY Change From Baseline in Gross Motor Function |
0.4; 0.9; 0.5; 4.4; 4.0; 2.2 | — |
| SECONDARY Change From Baseline in Modified Lansky Play Performance Scale |
-2.0; -6.0; -2.0; 2.0; -4.0; -6.0 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Age ≥ 6 years and 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
- Patients with suspected elevated intracranial pressure, pseudotumor cerebri (PTC) and/or papilledema
- Severe gastrointestinal disease including gastroparesis
- History of angina, myocardial infarction, or cardiac surgery within 2 years prior to the Screening Visit
- Any clinically significant electrocardiogram (ECG), including dysrhythmia, or clinically significant abnormal laboratory finding not already listed above at the Screening Visit
- History of drug or alcohol abuse
- History of pancreatitis
- Participated in an investigational drug trial within 30 days or, within 90 days for a biologic, device, or surgical treatment, for inherited mitochondrial diseases prior to the Screening Visit
- Known or suspected hypersensitivity to cysteamine and penicillamine
- Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive serum pregnancy test at the Screening Visit
- Subject's who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
Data sourced from ClinicalTrials.gov (NCT02023866). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.