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

> **NCT05174039** · PHASE1,PHASE2 · COMPLETED · sponsor: **Beyond Batten Disease Foundation** · enrollment: 6 (actual)

## Conditions studied

- Batten Disease

## Interventions

- **DRUG:** Miglustat 100 milligrams (mg) Oral Capsule

## Key facts

- **NCT ID:** NCT05174039
- **Lead sponsor:** Beyond Batten Disease Foundation
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2022-03-10
- **Primary completion:** 2024-05-30
- **Final completion:** 2024-05-30
- **Target enrollment:** 6 (ACTUAL)
- **Last updated:** 2025-09-09

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05174039

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT05174039, "An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease". Retrieved via AI Analytics 2026-09-28 from https://api.ai-analytics.org/clinical/NCT05174039. Licensed CC0.

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