Overview

An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Trehalose and Miglustat for the Treatment of CLN3 Disease

Status:
Not yet recruiting

Trial end date:
2023-07-01

Target enrollment:

Participant gender:

Summary

This is an open label study in approximately 6 subjects in 2 centers to assess the safety, PK, and efficacy of the maximum tolerable dose (MTD) of intravenous (IV) trehalose (0.25, 0.50, or 0.75 g/kg administered once-weekly) and the MTD of oral miglustat (100 mg once daily [QD] to 200 mg 3 times daily [TID]) in subjects ≥ 17 years of age with CLN3 disease over a period of 104 weeks.

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

Beyond Batten Disease Foundation

Collaborator:

Theranexus

Treatments:

Miglustat