# Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

> **NCT04771416** · PHASE1,PHASE2 · SUSPENDED · sponsor: **Gemma Biotherapeutics** · enrollment: 24 (estimated)

## Conditions studied

- Leukodystrophy, Globoid Cell

## Interventions

- **BIOLOGICAL:** PBKR03

## Key facts

- **NCT ID:** NCT04771416
- **Lead sponsor:** Gemma Biotherapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** SUSPENDED
- **Start date:** 2022-02-24
- **Primary completion:** 2027-01
- **Final completion:** 2030-01
- **Target enrollment:** 24 (ESTIMATED)
- **Why stopped:** The study was stopped due to changes in the company strategy.
- **Last updated:** 2025-11-13


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04771416

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04771416, "Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT04771416. Licensed CC0.

---

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