A Long-Term Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis

Phase 3

Completed

Conditions: cystinosis
10047954
Lignac-Fanconi Syndrom

Registration Number: NL-OMON43669

Lead Sponsor: Raptor Pharmaceuticals Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 6

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 * 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.

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.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>- To demonstrate superiority of RP103 versus Cystagon® in controlling white<br /><br>blood cell cystine (WBC) levels over 24 hours in patients with<br /><br>cystinosis.<br /><br>- To assess long-term safety and tolerability of RP103.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>- To compare WBC cystine measurements provided by a central laboratory versus<br /><br>sites* local (regional, not associated with the clinical trial)<br /><br>laboratories.<br /><br>- To assess the long term quality of life and sleep using instruments<br /><br>appropriate to the subjects* age and region (US or Europe).<br /><br>- To assess dosing compliance with two different cysteamine bitartrate<br /><br>formulations, Q6H immediate-release Cystagon® vs. Q12H delayedrelease<br /><br>RP103.<br /><br>- To assess halitosis during Cystagon® and RP103 treatment in a subgroup of<br /><br>subjects.</p><br>