Cysteamine Hydrochloride for nephrOpathic Cystinosis, open-label Phase III pivotal study

• Conditions: ephropatic cystinosis patients with cystine corneal deposits; MedDRA version: 16.0Level: LLTClassification code 10071112Term: Nephropathic cystinosisSystem Organ Class: 100000004850; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2009-012564-13-FR
• Lead Sponsor: Orphan Europe SAR

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-06-19
• Last Update Posted: 23 December 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

- Signed and dated written inform consent form in accordance with local regulations: Having freely given their written informed consent to participate in the study. For patients aged less than 18 years consent will be obtained from the two parents (or legal representatives),
- Diagnosis of cystinosis based on a previous white blood cells cystine concentration > 1.5 nmoles half-cystine per mg protein,
- Presence of corneal crystal deposits attested during a slit-lamp examination within 3 months prior inclusion,
- Ability to comply with their usual eye drops treatment in order to comply with the eyewash regimen of 4 instillations,
- Agreement to move to Ophthalmic Core Laboratory for the assessment visits,
- Likely to be able to participate in all scheduled evaluation and complete all required study procedures,
- In the opinion of the investigator, the patient will be compliant and have a high probability of completing the study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 16
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with uncontrolled hepatic disorder, cardiovascular disease, neurologic disease, or cancer,
- Laboratory tests out of normal range according to the reference laboratory values. Deviations may be accepted if the investigator considers that they are not clinically significant for the conduct of study,
- Patients with history or presence of alcohol abuse or drug addiction,
- Pregnant or breast-feeding women,
- Women of child-bearing potential without effective contraception (oral pill or IUCD),
- Patients likely to be non-compliant to the study procedures or for whom a long-term follow-up seems to be difficult to achieve (In the younger children, the confocal microscopy may not be feasible and therefore will not be excluded from the study but only those patients able to undergo the IVCM procedure should be included in the primary analysis).

Study & Design

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