# Gene Therapy for Children With CLN3 Batten Disease

> **NCT03770572** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Alcyone Therapeutics, Inc** · enrollment: 7 (estimated)

## Conditions studied

- CLN3
- Batten Disease

## Interventions

- **GENETIC:** Low dose CLN-301
- **GENETIC:** High dose CLN-301

## Key facts

- **NCT ID:** NCT03770572
- **Lead sponsor:** Alcyone Therapeutics, Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2018-11-13
- **Primary completion:** 2029-09-30
- **Final completion:** 2029-09-30
- **Target enrollment:** 7 (ESTIMATED)
- **Last updated:** 2025-08-07


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03770572

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03770572, "Gene Therapy for Children With CLN3 Batten Disease". Retrieved via AI Analytics 2026-06-25 from https://api.ai-analytics.org/clinical/NCT03770572. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*