Glibenclamide treatment in hypotonia-cystinuria syndrome

• Conditions: Hypotonia-cystinuria syndrome/PREPL deficiency; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2012-000814-13-BE
• Lead Sponsor: niversity Hospital Leuven

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-02-17
• Last Update Posted: 7 October 2014

Recruitment & Eligibility

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

Inclusion Criteria

Diagnosis of hypotonia-cystinuria syndrome/PREPL deficiency, confirmed by molecular genetic analysis.
Age <16y.
Written informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range: 8
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Intolerance for glibenclamide.
History of hyperinsulinism or other contraindications for glibenclamide.

Study & Design

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