Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
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|ClinicalTrials.gov Identifier: NCT01000961|
Recruitment Status : Completed
First Posted : October 23, 2009
Results First Posted : November 19, 2014
Last Update Posted : May 19, 2017
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|Condition or disease||Intervention/treatment||Phase|
|Cystinosis||Drug: Cystagon® (Cysteamine Bitartrate) Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||43 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||None (Open Label)|
|Official Title:||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|
|Study Start Date :||June 2010|
|Actual Primary Completion Date :||June 2011|
|Actual Study Completion Date :||August 2011|
|Experimental: RP103 Q12H||
Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1:
Every 12H, supplied in 75 and 25mg capsules/Duration of Treatment: 3 weeks
|Active Comparator: Cystagon® Q6H||
Drug: Cystagon® (Cysteamine Bitartrate)
Run-in Period (Weeks 1, 2, 3) and Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1:
Every 6H, supplied in 150 and 50mg capsules/Duration of Treatment: 3 weeks each period used
- The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon® [ Time Frame: 4 weeks after the last subject has completed the study ]
- Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®. [ Time Frame: 4 weeks after the last subject has completed the study ]
- Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®. [ Time Frame: 4 weeks after the last subject has completed the study ]
- Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®. [ Time Frame: 6 hours post dosing for Cystagon®; 12 hours post dosing for RP103. ]
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|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01000961
|United States, California|
|Stanford University Medical School|
|Stanford, California, United States, 94305|
|United States, Georgia|
|Emory Children's Center|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago (formerly Children's Memorial Hospital)|
|Chicago, Illinois, United States, 60614|
|Hospices Civils de Lyon|
|Robert Debre Hospital|
|Radboud University Nijmegen Medical Center|
|Study Director:||Evelyn Olson, BS||Horizon Pharma USA, Inc.|
|Responsible Party:||Horizon Pharma USA, Inc.|
|Other Study ID Numbers:||
|First Posted:||October 23, 2009 Key Record Dates|
|Results First Posted:||November 19, 2014|
|Last Update Posted:||May 19, 2017|
|Last Verified:||April 2017|
CTNS protein, human
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action