Efficacy and safety of treatment with N-butyl-deoxynojirimycin (NB-DNJ-miglustat) in patients with Niemann-Pick disease type C. - ND

• Conditions: IEMANN-PICK DISEASE TYPE C; MedDRA version: 9.1Level: LLTClassification code 10029403

• Registration Number: EUCTR2006-005842-35-IT
• Lead Sponsor: Dipartimento di Pediatria Universita` di Napoli Federico II

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-10-28
• Last Update Posted: 3 April 2012

Recruitment & Eligibility

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

Inclusion Criteria

Diagnosis of NPC confirmed by biochemical tests (filipin staining and LDL-cholesterol esterification) and/or molecular analysis
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 unable to take the drug Bad compliance to study procedures Patients with renal failure Fertile women that do not consent to oral contraceptives during the study and until three months after discontinuation of treatment

Study & Design

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