Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients … (NCT01000961) | Clinical Trial Compass
CompletedPhase 3
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
United States, France43 participantsStarted 2010-06
Plain-language summary
Cystinosis is an inherited disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results to four times a day Cystagon®.
Who can participate
Age range6 Years
SexALL
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Inclusion Criteria:
* Male and female subjects must have nephropathic cystinosis.
* Subjects 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.
* Subjects must be able to swallow their typically administered Cystagon® capsule with the capsule intact.
* Within the last 6 months, no clinically significant change in liver function \[i.e., ALT, AST, total bilirubin\] and renal function \[i.e., estimated GFR\] at Screening as determined by the Investigator.
* Subjects with an estimated GFR (corrected for body surface area) \> 30 mL/min/1.73m2.
* 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.
* Subjects must be willing and able to comply with the study restrictions and requirements.
* Subjects or their or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study.
Exclusion Criteria:
* Subject's age \< 6 years old or subject's weight \< 21 kg.
* Subjects with a known history, currently of the following conditions or other health issues that make it, in the opinion of the investigator, unsafe for them to participate: inflammatory bowel disease (if currently active) o…
What they're measuring
1
The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®
Timeframe: 4 weeks after the last subject has completed the study