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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):
Horizon Orphan LLC
ClinicalTrials.gov Identifier:
NCT01197378
First received: September 3, 2010
Last updated: February 10, 2017
Last verified: February 2017

September 3, 2010
February 10, 2017
August 2010
June 2017   (Final data collection date for primary outcome measure)
Safety and tolerability of long-term repeat dosing of RP103 in patients with nephropathic cystinosis. [ Time Frame: 30 days after Last Patient Last Visit ]
Same as current
Complete list of historical versions of study NCT01197378 on ClinicalTrials.gov Archive Site
  • Steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of RP103. [ Time Frame: 30 days after Last Patient Last Visit ]
  • Long term quality of life using either PedsQL™ or SF-36® instruments. [ Time Frame: 30 days after Last Patient Last Visit ]
Same as current
Not Provided
Not Provided
 
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients With Cystinosis
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®.
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 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, NCT01000961) and won't be offered to others until data analysis of the RP103-03 subjects has been completed.
Interventional
Phase 3
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Cystinosis
Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
RP103 every 12H, supplied in 75 and 25mg capsules
Other Name: RP103
Experimental: Cysteamine Bitartrate Delayed-release Capsules (RP103)
RP103 every 12H, supplied in 75 and 25mg capsules
Intervention: Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
60
June 2017
June 2017   (Final data collection date for primary outcome measure)

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 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.
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact information is only displayed when the study is recruiting subjects
United States,   France,   Netherlands
 
 
NCT01197378
RP103-04
2010-018365-34 ( EudraCT Number )
No
Not Provided
Not Provided
Not Provided
Horizon Orphan LLC
Horizon Orphan LLC
Not Provided
Study Director: Evelyn Olson, BS Horizon Orphan LLC
Horizon Orphan LLC
February 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP