Study comparing the effectiveness of cysteamine bitartrate delayed-release capsules (RP103) in Patients with Cystinosis (Adult and Children 12 years and older)

Phase 1

Active, not recruiting

• Conditions: Cystinosis; MedDRA version: 20.0Level: PTClassification code 10011777Term: CystinosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2012-002773-64-NL
• Lead Sponsor: Horizon Pharma USA, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-11-01
• Last Update Posted: 30 October 2017

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

1. Male or female with a documented diagnosis of cystinosis.
2. On a stable dose of Cystagon® for at least 21 days prior to Screening.
3. WBC cystine level > 1 nmol ½ cystine/mg of protein on average over
at least 2 measurements collected during the 2 years prior to Screening.
4. 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.
5. No clinically significant change in renal function, i.e. estimated GFR
within 6 months prior to Screening.
6. Must have an estimated GFR > 20 mL/minute/1.73 m2 (using the
equation from Schwartz 2009 J Am Soc Nephrol 20:629-637) 1.
7. 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.
8. Subject or their parent or guardian must provide written informed
consent and assent (where applicable) prior to participation in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 45
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Younger than 12 years of age.
2. 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 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.
3. Hemoglobin level of < 9 g/dL at Screening or, in the opinion
of the Investigator, a hemoglobin level that would make it
unsafe for study participation.
4. Known hypersensitivity to cysteamine and penicillamine.
5. Female subjects who are nursing, planning a pregnancy, or
are known or suspected to be pregnant.
6. Subjects who, in the opinion of the Investigator, are not able or willing to comply with study requirements.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified