Trial Profile

Phase 1/2a Study of 2-Hydroxypropyl-Beta-Cyclodextrin Therapy for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Status: Active, no longer recruiting

Phase of Trial: Phase I/II

Latest Information Update: 08 May 2025

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At a glance

Drugs Adrabetadex (Primary)
Indications Niemann-Pick disease type C
Focus Biomarker; Therapeutic Use

Most Recent Events

29 Apr 2025 Status changed from recruiting to active, no longer recruiting.
28 Mar 2025 Planned End Date changed from 1 Jun 2024 to 1 Oct 2025.
28 Mar 2025 Planned primary completion date changed from 1 Jun 2024 to 1 Oct 2025.

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