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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Raptor Pharmaceuticals Inc.
Sponsor:
Information provided by (Responsible Party):
Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier:
NCT01744782
First received: December 5, 2012
Last updated: July 2, 2014
Last verified: July 2014
  Purpose

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 and will continue to participate in the study and receive RP103, until it is available through the appropriate marketing approval (In the US, following FDA approval; In Brazil, once it is a regularly registered medication and available at no personal cost), or a subject withdraws or is withdrawn from the study, or the Sponsor terminates development of RP103 for cystinosis.

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.


Condition Intervention Phase
Cystinosis
Drug: RP103 Q12H
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Raptor Pharmaceuticals Inc.:

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

  • Long-Term Safety and Tolerability [ Time Frame: 12 months minimum ] [ Designated as safety issue: Yes ]
    The safety profile of RP103 will be investigated with the following assessments: physical exam, vital signs, ECG, clinical laboratory testing and adverse events.


Estimated Enrollment: 10
Study Start Date: December 2012
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: December 2015 (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)

  Eligibility

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

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

Exclusion Criteria:

  • Subjects with 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 cysteamine and 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
  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: NCT01744782

Contacts
Contact: Mary Jo Bagger, Clinical Operations, Raptor Pharmaceuticals Inc. 1-415-408-6210 mbagger@raptorpharma.com

Locations
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60614
Contact: Heather Price, MS    773-755-6368    hprice@luriechildrens.org   
Principal Investigator: Craig B Langman, MD         
Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de Sao Paulo Not yet recruiting
Sao Paulo, SP, Brazil
Contact: Maria Helena Vaisbich, MD    55-11-2661-8833    vaisbich@terra.com.br   
Contact: Juliiana de Oliveria Achili    55-11-2661-8833    juliana.caires@hc.fm.usp.br   
Sponsors and Collaborators
Raptor Pharmaceuticals Inc.
Investigators
Principal Investigator: Craig B. Langman, MD Ann & Robert H Lurie Children's Hospital of Chicago
  More Information

Publications:
Responsible Party: Raptor Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT01744782     History of Changes
Other Study ID Numbers: RP103-08
Study First Received: December 5, 2012
Last Updated: July 2, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Raptor Pharmaceuticals Inc.:
Nephropathic Cystinosis
Cysteamine
Delayed-release Cysteamine
CTNS Protein, Human
Orphan Disease

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 20, 2014