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Trial record 13 of 32 for:    CYSTEAMINE

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
Sponsor:
Information provided by (Responsible Party):
Horizon Pharma USA, Inc.

Tracking Information
First Submitted Date  ICMJE October 22, 2009
First Posted Date  ICMJE October 23, 2009
Results First Submitted Date  ICMJE November 2, 2012
Results First Posted Date  ICMJE November 19, 2014
Last Update Posted Date May 19, 2017
Study Start Date  ICMJE June 2010
Actual Primary Completion Date June 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 18, 2014)
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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: October 22, 2009)
To assess the steady-state white blood cell cystine levels of RP103 compared to Cystagon® [ Time Frame: Periodically over a 3 day window for each drug ]
Change History Complete list of historical versions of study NCT01000961 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 18, 2014)
  • 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. ]
Original Secondary Outcome Measures  ICMJE
 (submitted: October 22, 2009)
  • To assess the safety and tolerability of RP103 compared to Cystagon® [ Time Frame: 27-37 days (duration of study) ]
  • To assess the steady state pharmacokinetics and pharmacodynamics of RP103 compared to Cystagon®. [ Time Frame: Periodically over a 3 day window for each drug ]
  • To compare the incidence of concomitant gastric acid reduction therapies [e.g., proton pump inhibitors (PPI)] between Cystagon® and RP103. [ Time Frame: 27-37 days (duration of study) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Official Title  ICMJE 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
Brief Summary 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®.
Detailed Description 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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystinosis
Intervention  ICMJE
  • 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

  • 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

Study Arms  ICMJE
  • Experimental: RP103 Q12H
    Intervention: Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)
  • Active Comparator: Cystagon® Q6H
    Intervention: Drug: Cystagon® (Cysteamine Bitartrate)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 17, 2012)
43
Original Estimated Enrollment  ICMJE
 (submitted: October 22, 2009)
20
Actual Study Completion Date  ICMJE August 2011
Actual Primary Completion Date June 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Netherlands,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01000961
Other Study ID Numbers  ICMJE RP103-03
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Horizon Pharma USA, Inc.
Study Sponsor  ICMJE Horizon Pharma USA, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Evelyn Olson, BS Horizon Pharma USA, Inc.
PRS Account Horizon Pharma USA, Inc.
Verification Date April 2017

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