NCT05174039View on ClinicalTrials.gov

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

March 10, 2022

Primary Completion Date

May 30, 2024

Study Completion Date

May 30, 2024

Conditions
Batten Disease
Interventions
DRUG

Miglustat 100 milligrams (mg) Oral Capsule

Subjects will initiate miglustat at Week 1 and dosing will be escalated until 600mg/d. If a subject has not reached the maximum dose (600 mg/d) by Week 8, the Week 8 dose will be subject's MTD.

Trial Locations (1)

77030

Texas Children Hospital, Houston

Sponsors

Collaborators (1)

Theranexus
All Listed Sponsors
collaborator

Theranexus

INDUSTRY

lead

Beyond Batten Disease Foundation

OTHER

NCT05174039 - An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease | Biotech Hunter | Biotech Hunter