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Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

This study has been completed.
Information provided by (Responsible Party):
Horizon Pharma USA, Inc. Identifier:
First received: December 5, 2012
Last updated: April 11, 2017
Last verified: April 2017
This is a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who are naïve to any form of cysteamine treatment. Subjects will receive RP103 treatment for at least 12 months. US subjects will transition to the commercially approved drug PROCYSBI®. In Brazil, after at least 12 months of study participation and upon approval by the Brazilian regulatory authorities, subjects will be eligible to transition to a post study drug supply program, and continue to receive the drug at no personal cost.

Condition Intervention Phase
Cystinosis Drug: RP103 Q12H Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Safety and Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naïve Patients With Cystinosis

Resource links provided by NLM:

Further study details as provided by Horizon Pharma USA, Inc.:

Primary Outcome Measures:
  • White Blood Cell (WBC) Cystine Levels [ Time Frame: 12 months minimum ]
    Steady-state cysteamine-trough WBC cystine levels 30 minutes post RP103 dose at each study visit.

Secondary Outcome Measures:
  • Long-Term Safety and Tolerability [ Time Frame: 12 months minimum ]
    The safety profile of RP103 will be investigated by changes from the last study visit as noted in the following parameters: physical examination, vital signs, ECG and clinical laboratory testing. Adverse events (including attribution of treatment-emergent non-serious and serious adverse events) will be recorded.

  • Pharmacokinetic Assessment (Cmax, maximum concentration) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of Cmax.

  • Pharmacokinetic Assessment (Tmax, time to maximum concentration) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of Tmax.

  • Pharmacokinetic Assessment (AUC, area under the curve) [ Time Frame: 12 months minimum ]
    Plasma cysteamine concentration of AUC.

Enrollment: 17
Actual Study Start Date: December 19, 2012
Study Completion Date: December 13, 2016
Primary Completion Date: December 13, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RP103 Q12H
From Day 1 and throughout the duration of participation, subjects will take RP103 (Cysteamine Bitartrate Delayed-release Capsules) every 12 hours, supplied in 75mg and 25mg capsules.
Drug: RP103 Q12H
Other Name: (Cysteamine Delayed-release Capsules)

Detailed Description:

The purpose of this study is to gather information about the safety and effectiveness (how well it works to treat cystinosis) of a new drug called RP103.

In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of an older, already approved drug called Cystagon® (cysteamine bitartrate) reduces cystine in the body. RP103 has the same active ingredient as Cystagon® and is designed to reduce cystine in a similar way that Cystagon® does. RP103 is also different from Cystagon®: Instead of the cysteamine bitartrate being absorbed from the stomach, RP103 is designed to be absorbed from the small intestine. This may make the effects of the drug last longer, so that it can be taken twice a day instead of four times a day like Cystagon®.

To decide if RP103 is effective, the study will look at two types of blood tests. One test is pharmacodynamics (PD), which measures the amount of white blood cell (WBC) cystine after taking study drug. WBC cystine is a laboratory test used to find out if cysteamine bitartrate is reducing cystine levels in the body. The second test is pharmacokinetics (PK), which measures the amount of cysteamine in the blood after taking the drug.


Ages Eligible for Study:   up to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female with a documented diagnosis of cystinosis
  • No clinically significant change in liver function tests, i.e. 1.5 times ULN for ALT and AST, and/or 1.5 times ULN for total bilirubin, within 6 months prior to Screening
  • No clinically significant change in renal function, i.e. estimated GFR within 6 months prior to Screening
  • Must have an estimated GFR > 20 mL/minute/1.73m2 (using the equation from Schwartz 2009 J Am Soc Nephrol 20:629-647)
  • Female subjects who are sexually active and of childbearing potential, i.e. not surgically sterile (tubal ligation, bilateral oopherectomy, or hysterectomy) or at least 2 years naturally postmenopausal must agree to use an acceptable form of contraception from Screening through completion of the study. Acceptable forms of contraception for this study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to Screening, barrier (spermicidal condom or diaphragm with spermicide), IUD, or a partner who has been vasectomized for at least 6 months. [NB: Childbearing potential is defined as a female who has reached menarche.]
  • Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study
  • Has not taken any form of cysteamine bitartrate in the past

Exclusion Criteria:

  • Current history of the following conditions or any other health issues that make it, in the opinion of the investigator, unsafe for study participation:
  • Inflammatory bowel disease if currently active, or prior resection of the small intestine
  • Heart disease (e.g., myocardial infarction, heart failure, unstable arrhythmias, or poorly controlled hypertension) within 90 days prior to Screening
  • Active bleeding disorder within 90 days prior to Screening
  • History of malignant disease within 2 years prior to Screening
  • Hemoglobin level of < 10 g/dL at Screening or, in the opinion of the investigator, a hemoglobin level that would make it unsafe for study participation
  • Known hypersensitivity to penicillamine
  • Female subjects who are nursing, planning a pregnancy, or are known or suspected to be pregnant
  • Subjects who, in the opinion of the investigator, are not able or willing to comply with study requirements
  • Has received a kidney transplant or is currently on dialysis
  • Is 6 years of age or older at the time of the Screening visit
  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 identifier: NCT01744782

United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo
Sao Paulo, SP, Brazil
Sponsors and Collaborators
Horizon Pharma USA, Inc.
Study Director: Evelyn Olson, BS Horizon Pharma USA, Inc.
  More Information

Responsible Party: Horizon Pharma USA, Inc. Identifier: NCT01744782     History of Changes
Other Study ID Numbers: RP103-08
Study First Received: December 5, 2012
Last Updated: April 11, 2017

Keywords provided by Horizon Pharma USA, Inc.:
Nephropathic Cystinosis
Delayed-release Cysteamine
CTNS Protein, Human
Orphan Disease

Additional relevant MeSH terms:
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action processed this record on September 20, 2017