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| Sponsor: | Raptor Pharmaceutical Corp. |
|---|---|
| Information provided by: | Raptor Pharmaceutical Corp. |
| ClinicalTrials.gov Identifier: | NCT00872729 |
Purpose
Cystinosis is an inheritable 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.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystinosis |
Drug: Cystagon® (Cysteamine Bitartrate) Capsules: 150 mg/50 mg Drug: RP103 |
Phase I |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Control: Active Control Endpoint Classification: Pharmacokinetics/Dynamics Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Pilot Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cysteamine Bitartrate (Cystagon®) in Patients With Nephropathic Cystinosis |
| Estimated Enrollment: | 10 |
| Study Start Date: | May 2009 |
| Study Completion Date: | October 2009 |
| Primary Completion Date: | October 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Cystagon®: Active Comparator
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules: 150 mg/50 mg
|
Drug: Cystagon® (Cysteamine Bitartrate) Capsules: 150 mg/50 mg
Reference Product: Cystagon® (Cysteamine Bitartrate) Capsules: 150 mg/50 mg Duration of Treatment: Reference Period up to four doses Q6H
|
|
RP103: Experimental
Test Product: RP103 (Cysteamine Bitartrate Delayed-release Capsules), 75 mg
|
Drug: RP103
Test Product: RP103 (Cysteamine Bitartrate Delayed-release Capsules), 75 mg Dose: Dose of RP103 equivalent to reference product. Single dose of Test Product.
|
This is a single-dose, open-labeled, non-randomized, two-period study of Cysteamine Bitartrate Delayed-release Capsules (RP103) and Cystagon® in up to 10 patients (male or female) with nephropathic cystinosis under fasting conditions. It will involve a 4 night check-in to a clinical research center.
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| United States, California | |
| University of California San Diego Medical Center | |
| San Diego, California, United States, 92103 | |
| Principal Investigator: | Bruce Barshop, MD, PhD | UCSD |
More Information
| Responsible Party: | Raptor Therapeutics Inc. ( Patrice P. Rioux , MD, PhD, Chief Medical Officer ) |
| ClinicalTrials.gov Identifier: | NCT00872729 History of Changes |
| Other Study ID Numbers: | RP103-01 |
| Study First Received: | March 27, 2009 |
| Last Updated: | July 13, 2010 |
| Health Authority: | United States: Food and Drug Administration |
|
cystinosis cysteamine inheritable disease |
orphan disease CTNS protein, human 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 |