Cystinosis MedDRA version: 12.1 Level: LLT Classification code 10011777 Term: Cystinosis
Conditions
Interventions
Product Name: Cysteamine bitartrate (INN: mercaptamine bitartrate )
Product Code: RP103
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: mercaptamine bitartrate
CAS Number: CAS 27761-19
Current
Sponsors
Raptor Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female subject with a documented diagnosis of nephropathic cystinosis. 2. Subject must be on a stable dose of Cystagon® sufficient to maintain their white blood cell (WBC) cystine level at = 1.0 nmol/half-cystine/mg protein. 3. Subject must be able to swallow their typically administered Cystagon® capsule with the capsule intact. 4. Within the last 6 months, no clinically significant change from normal in liver function tests [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. 5. Subject must have an estimated GFR (corrected for body surface area) > 30 mL/minute/1.73 m2. 6. Sexually active female subjects of childbearing potential (i.e., not surgically sterile [tubal ligation, hysterectomy, or bilateral oophorectomy] 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. 7. Subject must be willing and able to comply with the study restrictions and requirements. 8. 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: 2. 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. 3. 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. 4. Subjects receiving any form of cysteamine medication through a gastric tube. 5. Subjects who are receiving maintenance dialysis or who have had a kidney transplant. 6. Subjects who are on an active kidney transplant list or who are planning to receive a kidney transplant within 3 months of Screening. 7. Subjects with known hypersensitivity to cysteamine and penicillamine. 8. Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive serum pregnancy screen. 9. Subjects who have a made a blood donation within 30 days of Screening. 10. Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate that at steady-state, patients receiving every 6 hour (Q6H) treatment of Cystagon® can maintain a comparable depletion of white blood cell (WBC) cystine levels after receiving every 12 hour (Q12H) treatment regimen of RP103;Secondary Objective: To assess safety and tolerability of RP103. To assess the steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of RP103 compared to Cystagon®.;Primary end point(s): Comparable depletion of steady-state cysteamine-trough WBC cystine levels (Days 5, 6 and 7 of Week 6 (Period 1) and Week 9 (Period 2)) between Cystagon® and RP103. If the one-sided test of non-inferiority, conducted at the nominal level of 0.02104, is rejected at a non-inferiority margin of 0.3, we will conclude that RP103 is non-inferior to Cystagon® with an overall significance level of 0.025. | — |
Countries
France, Netherlands
Outcome results
None listed