A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis - RP103-04

Phase 1

• Conditions: Cystinosis; MedDRA version: 12.1Level: LLTClassification code 10011777Term: Cystinosis

• Registration Number: EUCTR2010-018365-34-FR
• Lead Sponsor: Raptor Therapeutics Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06-18
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1.Male and female subjects must have completed the last visit of Study RP103-03 and be willing to continue with RP103 treatment.
OR for patients who did not complete the RP103-03 study:
2. Male and female subject with a documented diagnosis of nephropathic cystinosis.
3. Subject on a stable dose of Cystagon® at least 21 days prior to Screening.
4. Subject must be able to swallow their typically administered Cystagon® capsule with the capsule intact.
5. Within the last 6 months, no clinically significant change in liver function [i.e., 1.5 times ULN for ALT and AST, and/or 1.5 times ULN for total bilirubin] and renal function [i.e., estimated GFR (corrected for body surface area)] at Screening as determined by the Investigator.
6. Subject must have an estimated GFR (corrected for body surface area) > 30 mL/minute/1.73 m2.
7. Sexually active female subjects of childbearing potential (i.e., not surgically sterile [tubal ligation, hysterectomy, or bilateraloophorectomy] or at least 2 years naturally postmenopausal) must agree to utilize the same acceptable form of contraception from Screening through completion of the Study. The acceptable forms of contraception for this Study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to Screening, barrier (spermicidal condom, diaphragm with spermicide), IUD, or a partner who has been vasectomized for at least 6 months. For pre-pubescent children, a documented attestation of abstinence from their parent or guardian will be acceptable.
8. Subject must be willing and able to comply with the Study restrictions and requirements.
9. Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the Study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Exclusion Criteria:
1.Patients enrolled in the previous Study RP103-03 who did not complete their last scheduled Study visit or who do not wish to continue on treatment with RP103.
AND for patients who did not complete the RP103-03 study:
2.Subjects who cannot take intact Cystagon® capsules orally.
3. Subjects with current history of the following conditions or any other health issues that make it, in the opinion of the Investigator, unsafe for them to participate:
• Inflammatory bowel disease (if currently active) or prior resection of small intestine;
•Heart disease (e.g., myocardial infarction, heart failure, unstable arrhythmias, or poorly controlled hypertension) 90 days prior to Screening;
•Active bleeding disorder 90 days prior to Screening;
• History of malignant disease within the last 2 years.
4.Subject with a hemoglobin level of < 10 g/dL at Screening or, in the opinion of the Investigator, a hemoglobin level that would make it unsafe for the subject to participate.
5. Subjects receiving any form of cysteamine medication through a gastric tube.
6. Subjects with known hypersensitivity to cysteamine and penicillamine.
7.Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive serum pregnancy screen.
8. Subjects who, in the opinion of the Investigator, are not able orwilling to comply with the protocol.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified