NL-OMON34074
Recruiting
Phase 3
A Randomized, Crossover, Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients with Nephropathic Cystinosis - RP103-03
Raptor Pharmaceuticals Europe BV0 sites5 target enrollmentTBD
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- metabolic disorder in which the transport of cystine out of the lysosomes is abnormal
- Sponsor
- Raptor Pharmaceuticals Europe BV
- Enrollment
- 5
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Male or female subject with a documented diagnosis of nephropathic cystinosis.
- •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.
- •Subject must be able to swallow their typically administered Cystagon® capsule with the capsule intact.
- •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.
- •Subject must have an estimated GFR (corrected for body surface area) \> 30 mL/minute/1\.73 m2\.
- •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.
Exclusion Criteria
- •Subject\*s age \< 6 years old or subjects weight \< 21 kg.
- •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.
- •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.
- •Subjects receiving any form of cysteamine medication through a gastric tube.
- •Subjects who are receiving maintenance dialysis or who have had a kidney transplant.
- •Subjects who are on an active kidney transplant list or who are planning to receive a kidney transplant within 3 months of Screening.
Outcomes
Primary Outcomes
Not specified
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