Status:
COMPLETED
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Lead Sponsor:
Amgen
Conditions:
Cystinosis
Eligibility:
All Genders
6+ years
Phase:
PHASE3
Brief 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 ta...
Detailed Description
This is a multi-center, open-label, randomized, cross-over study to determine whether steady-state, twice a day treatment with Cysteamine Bitartrate Delayed-release Capsules(RP103) results in comparab...
Eligibility Criteria
Inclusion
- 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
- 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) or have had prior resection of small intestine; Heart disease (e.g., myocardial infarction, heart failure, arrhythmias or poorly controlled hypertension) 90 days prior to Screening; Active bleeding disorder 90 days prior to Screening; Malignant disease within the last 2 years.
- Patients with a hemoglobin level \< 10 g/dL at Screening or a level that, in the opinion of the investigator, makes 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.
- Subjects with known hypersensitivity to cysteamine or penicillamine.
- Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or have a positive serum pregnancy screen.
- Subjects who have a made a blood donation within 30 days of Screening.
- Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
Key Trial Info
Start Date :
June 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2011
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT01000961
Start Date
June 1 2010
End Date
August 1 2011
Last Update
December 19 2024
Active Locations (8)
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1
Stanford University Medical School
Stanford, California, United States, 94305
2
Emory Children's Center
Atlanta, Georgia, United States, 30322
3
Ann & Robert H. Lurie Children's Hospital of Chicago (formerly Children's Memorial Hospital)
Chicago, Illinois, United States, 60614
4
Hospices Civils de Lyon
Lyon, France