A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02473445
• Date of registration: 10/06/2015
• Prospective Registration: No
• Primary sponsor: Horizon Pharma USA, Inc.
• Public title: A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
• Scientific title: A Long-Term Open-Label Extension Study of RP103-MITO-001 to Assess the Safety, Tolerability and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103) for Treatment of Children With Inherited Mitochondrial Disease
• Date of first enrolment: May 19, 2015
• Target sample size: 22
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02473445
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Evelyn Olson, BS
• Affiliation: Horizon Pharma USA, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Completed all visits in Study RP103-MITO-001 (NCT02023866).

2. Body weight = 5 kg.

3. 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).

4. 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.

5. 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).

6. Patient's legally authorized representative must provide written informed consent;
Patient must provide assent, if required by local/institutional requirements.

Exclusion Criteria:

1. Documented diagnosis of concurrent inborn errors of metabolism.

2. Platelet count, lymphocyte count or hemoglobin below the lower limit of normal (LLN)
at the Baseline visit.

3. 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.

4. Bilirubin > 1.2 g/dL at the Baseline Visit.

5. Inability to complete the elements of the study, e.g., coma, hemodynamic instability
or requiring continuous ventilator support.

6. Malabsorption requiring total parenteral nutrition (TPN), chronic diarrhea, bouts of
pseudo obstruction.

7. Severe end-organ hypo-perfusion syndrome secondary to cardiac failure resulting in
lactic acidosis.

8. Patients with suspected elevated intracranial pressure, pseudotumor cerebri (PTC)
and/or papilledema.

9. Severe gastrointestinal disease including gastroparesis.

10. History of drug or alcohol abuse.

11. History of pancreatitis.

12. 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.

13. Known or suspected hypersensitivity to cysteamine and penicillamine.

14. 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.

15. Patients who, in the opinion of the Investigator, are not able or willing to comply
with the protocol.



Age minimum: 6 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Mitochondrial Diseases

Intervention(s)

Drug: Cysteamine Bitartrate

Primary Outcome(s)

Change in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Score [Time Frame: Baseline, every 3 months and Study Exit (up to 24 Months)]

Secondary Outcome(s)

Change Over Time in Two of the Most Pre-eminent Symptoms [Time Frame: Baseline, every 3 months and Study Exit (up to 24 Months)]

Change Over Time in Pharmacodynamic Biomarkers [Time Frame: Baseline, every 3 months and Study Exit (up to 24 Months)]

Secondary ID(s)

RP103-MITO-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/05/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02473445