# N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

> **NCT03759665** · PHASE2 · COMPLETED · sponsor: **IntraBio Inc** · enrollment: 30 (actual)

## Conditions studied

- GM2 Gangliosidosis
- Tay-Sachs Disease
- Sandhoff Disease

## Interventions

- **DRUG:** IB1001

## Key facts

- **NCT ID:** NCT03759665
- **Lead sponsor:** IntraBio Inc
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2019-06-07
- **Primary completion:** 2023-01-09
- **Final completion:** 2023-01-09
- **Target enrollment:** 30 (ACTUAL)
- **Last updated:** 2024-03-12


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT03759665

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT03759665, "N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)". Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/clinical/NCT03759665. Licensed CC0.

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