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

NCT04771416 · Status: SUSPENDED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2026-06-30

No results posted yet for this study

Summary

PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended to deliver a functional copy of the GALC gene to the brain and peripheral tissues. This study will evaluate the safety, tolerability and efficacy of this treatment by first evaluating two different doses in two different age groups, then confirming the optimal dose to be used for confirmation of safety and efficacy.

Conditions

  • Leukodystrophy, Globoid Cell

Interventions

BIOLOGICAL

PBKR03

Single dose of PBKR03, via intra cisterna magna

Sponsors & Collaborators

  • Gemma Biotherapeutics

    lead INDUSTRY

Principal Investigators

  • May Orfali, MD · Gemma Biotherapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Month
Max Age
9 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-24
Primary Completion
2027-01-31
Completion
2030-01-31
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Israel
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04771416 on ClinicalTrials.gov