Status:
COMPLETED
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Lead Sponsor:
Amgen
Conditions:
Cystinosis
Eligibility:
All Genders
1+ 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 immediate release...
Detailed Description
This is a long-term, open-label, study to determine the safety and tolerability of twice a day treatment with cysteamine bitartrate delayed-release capsules (RP103). It will involve 6-9 monthly clinic...
Eligibility Criteria
Inclusion
- 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:
- Male and female subjects must have cystinosis.
- Subjects must be on a stable dose of Cystagon® at least 21 days prior to Screening.
- Within the last 6 months, no clinically significant change from normal in liver function tests (i.e., alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], total bilirubin) and renal function (i.e., estimated glomerular filtration rate \[eGFR\]) at Screening as determined by the Investigator.
- Subjects with an eGFR 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 parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study.
Exclusion
- 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:
- Subjects less than 1 year old
- 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, unstable 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 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, in the opinion of the Investigator, are not able or willing to comply with the protocol.
Key Trial Info
Start Date :
August 27 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 26 2017
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT01197378
Start Date
August 27 2010
End Date
June 26 2017
Last Update
December 27 2024
Active Locations (10)
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1
California Pacific Medical Center (CPMC) Research Institute
San Francisco, California, United States, 94115
2
Stanford University Medical School
Stanford, California, United States, 94305
3
Emory Children's Center
Atlanta, Georgia, United States, 30322
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60614