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 early · Phase 2

Conditions studied: Mitochondrial Diseases

In brief

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).

Key facts

Study ID

NCT02473445

Run by

Amgen

People needed

22

Starts

2015-05-19

Expected to finish

2017-03-06

Last updated by the study team

2024-12-27

Who can join

Age: 6 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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):
• Hormonal contraception: birth control pills, injection, patch, vaginal ring or implant;
• Condom or diaphragm, with spermicide;
• Intrauterine device (IUD);
• Sterile male partner (vasectomy performed at least 6 months prior to the study).
• Patient's legally authorized representative must provide written informed consent; Patient must provide assent, if required by local/institutional requirements.

You may not qualify if…

• Documented diagnosis of concurrent inborn errors of metabolism.
• 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.
• Patients with suspected elevated intracranial pressure, pseudotumor cerebri (PTC) and/or papilledema.
• Severe gastrointestinal disease including gastroparesis.
• History of drug or alcohol abuse.
• History of pancreatitis.
• Participated in an investigational drug trial (except the RP103-MITO-001 study) within 30 days or, within 90 days for a biologic, device, or surgical treatment, for inherited mitochondrial diseases prior to the Baseline 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 Baseline visit.
• Patients who, in the opinion of the Investigator, are not able or willing to comply with the protocol.

Where it is running

• University of California at San Diego (UCSD) — San Diego, California, United States
• Stanford University School of Medicine — Stanford, California, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Baylor College of Medicine — Houston, Texas, United States
• University of Utah — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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