# Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

> **NCT04713475** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **Gemma Biotherapeutics** · enrollment: 26 (estimated)

## Conditions studied

- GM1 Gangliosidosis
- GM1 Gangliosidosis, Type I
- GM1 Gangliosidosis, Type 2
- Beta-Galactosidase-1 (GLB1) Deficiency

## Interventions

- **BIOLOGICAL:** PBGM01

## Key facts

- **NCT ID:** NCT04713475
- **Lead sponsor:** Gemma Biotherapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2021-03-17
- **Primary completion:** 2026-02
- **Final completion:** 2029-02
- **Target enrollment:** 26 (ESTIMATED)
- **Last updated:** 2025-05-21


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04713475

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04713475, "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis". Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/clinical/NCT04713475. Licensed CC0.

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