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| Sponsor: | Raptor Pharmaceutical Corp. |
|---|---|
| Information provided by: | Raptor Pharmaceutical Corp. |
| ClinicalTrials.gov Identifier: | NCT01000961 |
Purpose
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®.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystinosis |
Drug: Cystagon® (Cysteamine Bitartrate) Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103) |
Phase III |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
| Estimated Enrollment: | 20 |
| Study Start Date: | June 2010 |
| Estimated Study Completion Date: | July 2011 |
| Primary Completion Date: | June 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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 |
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 comparable depletion of white blood cell (WBC) cystine levels compared to the existing four times a day cysteamine treatment. It will involve up to 20 clinic visits plus intermittent home use of the RP103. Most of these clinic visits occur in clusters of 3-4 consecutive days. Eligible patients will be offered enrollment into a long-term follow up study.
Eligibility| Ages Eligible for Study: | 6 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| 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 | |
| Children's Memorial Hospital | |
| Chicago, Illinois, United States, 60614 | |
| United States, Texas | |
| Texas Children's Hospital/Baylor University | |
| Houston, Texas, United States, 77030 | |
| France | |
| Hospices Civils de Lyon | |
| Lyon, France | |
| Villeneuve-Lapeyronie Hospital | |
| Montpellier, France | |
| Hopital Necker | |
| Paris, France | |
| Robert Debre Hospital | |
| Paris, France | |
| Netherlands | |
| Radboud University Nijmegen Medical Center | |
| Nijmegen, Netherlands | |
| Principal Investigator: | Craig Langman, MD | Children's Memorial Hospital |
More Information
| Responsible Party: | Mary Jo Bagger, 707-494-9176, mbagger@raptorpharma.com, Raptor Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT01000961 History of Changes |
| Other Study ID Numbers: | RP103-03 |
| Study First Received: | October 22, 2009 |
| Last Updated: | June 10, 2011 |
| Health Authority: | United States: Food and Drug Administration; France: Afssaps - French Health Products Safety Agency |
|
cystinosis cysteamine inheritable disease orphan disease |
CTNS protein, human metabolic disease nephropathic cystinosis |
|
Cystinosis Fanconi Syndrome Lysosomal Storage Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases Kidney Diseases |
Urologic Diseases Renal Tubular Transport, Inborn Errors Cysteamine Radiation-Protective Agents Protective Agents Physiological Effects of Drugs Pharmacologic Actions |