Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01197378
First received: September 3, 2010
Last updated: June 25, 2014
Last verified: June 2014
  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: Cysteamine Bitartrate Delayed-release Capsules (RP103)
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis

Resource links provided by NLM:


Further study details as provided by Raptor Pharmaceuticals Inc.:

Primary Outcome Measures:
  • Safety and tolerability of long-term repeat dosing of RP103 in patients with nephropathic cystinosis. [ Time Frame: 30 days after Last Patient Last Visit ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of RP103. [ Time Frame: 30 days after Last Patient Last Visit ] [ Designated as safety issue: No ]
  • Long term quality of life using either PedsQL™ or SF-36® instruments. [ Time Frame: 30 days after Last Patient Last Visit ] [ Designated as safety issue: No ]

Enrollment: 60
Study Start Date: August 2010
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cysteamine Bitartrate Delayed-release Capsules (RP103)
RP103 every 12H, supplied in 75 and 25mg capsules
Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
RP103 every 12H, supplied in 75 and 25mg capsules
Other Name: RP103

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 visits followed by up to 6 quarterly clinic visits for the duration of the study and home use of RP103. Enrollment will be offered first to those patients who have completed the previous Phase 3 Study (RP103-03)and won't be offered to others until data analysis of the RP103-03 subjects has been completed.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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., 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 parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study.

Exclusion Criteria:

  • 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 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 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01197378

Locations
United States, California
California Pacific Medical Center (CPMC) Research Institute
San Francisco, California, United States, 94115
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
Chicago, Illinois, United States, 60614
United States, Texas
Texas Children's Hospital/Baylor University
Houston, Texas, United States, 77030
France
Villeneuve-Lapeyronie Hospital
Montpellier, France
Robert Debre Hospital
Paris, France
Hopital Necker
Paris, France
Netherlands
Radboud University Nijmegen Medical Center
Nijmegen, Netherlands
Sponsors and Collaborators
Raptor Pharmaceuticals Inc.
Investigators
Principal Investigator: Craig Langman, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

Additional Information:
Publications:
Responsible Party: Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01197378     History of Changes
Other Study ID Numbers: RP103-04
Study First Received: September 3, 2010
Last Updated: June 25, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Raptor Pharmaceuticals Inc.:
cystinosis
cysteamine
inheritable disease
orphan disease
CTNS protein, human
metabolic disease
nephropathic cystinosis

Additional relevant MeSH terms:
Cystinosis
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Cysteamine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 21, 2014