OPEN-LABEL EXTENSION STUDY TO ASSESS THE SAFETY AND LONG-TERM EFFECTIVENESS OF ORALLY ADMINISTERED EFAVIRENZ IN PATIENTS DIAGNOSED WITH ADULT OR LATE-ONSET JUVENILE TYPE C NIEMANN-PICK DISEASE WITH COGNITIVE IMPAIRMENT

Phase 1

• Conditions: Type C Niemann-Pick Disease; MedDRA version: 20.0Level: PTClassification code: 10029403Term: Niemann-Pick disease Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: CTIS2023-505832-36-01
• Lead Sponsor: Bellvitge University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-29
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

Patients (>18 years of age) included in the previous Niemann-Pick trial (EudraCT: 2019-004498-18), Patients who sign the written informed consent

Exclusion Criteria

Patients who (at the principal investigator's discretion) would experience an overdemanding effort (financially and in terms of time) to participate in the extension study.

Study & Design

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