Study comparing the effectiveness of cysteamine bitartrate delayed release capsules (RP103) to Cystagon in Patients with Cystinosis(Adults and Children 12 years and older).

Phase 1

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-GB

Lead Sponsor: Horizon Pharma USA, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

Male or female, 12 years and older, with a documented diagnosis of cystinosis.
(Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study)

On a stable dose of Cystagon® for at least 21 days prior to Screening.

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.

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/1.73 m2 (using the equation from Schwartz 2009 J Am Soc Nephrol 20:629-637) 1.
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 0

Exclusion Criteria

Younger than 12 years of age.

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.

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.

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.