CrYSTobs A cohort of patients with cystinosis : compliance to cysteamine and neurological complicationsAn auxiliary study to Raptor RP103 03 and RP103 04 - CrYSTobs

• Conditions: nephropatic cystinosis; MedDRA version: 12.1Level: LLTClassification code 10011777Term: Cystinosis

• Registration Number: EUCTR2010-020098-18-FR
• Lead Sponsor: HOSPICES CIVILS DE LYO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-07-12
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

Patients with nephropathic cystinosis

- Male and female subjects with confirmed diagnosis of nephropathic cystinosis (defined by clinical signs and WBC cystine level).
- Age > 6 years.
- Subjects receiving any oral cysteamine treatment: Cystagon? or RP103.
- Subjects must be able to swallow their typically administered cysteamine treatment.
- Sexually active female subjects of childbearing potential must agree to utilize the same acceptable form of contraception from day 1 through completion of the study.
- Subjects or their parent or legal guardian must provide written informed consent prior to participation in the study.
- Subjects covered by or having the right to social security.

Controls

- Age and sex matched to study population :
Age range : patients aged 6 to 11 years , patients aged 12 to 17 years, adult patients: 18 or above.
Sex : male or female
- Subjects or their parent or legal guardian must provide written informed consent prior to participation in the study.
- Subjects covered by or having the right to social security.

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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients with nephropathic cystinosis
- Subjects receiving any form of cysteamine medication through a gastric tube.
- Subjects with known hypersensitivity to cysteamine and penicillamine.
- Females who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive urinary pregnancy test.
- Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
Contra-indication to MRI assessment

Controls for metabonomic assessment
- Any uropathology or nephropathology.

Study & Design

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