Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
Public ClinicalTrials.gov record NCT04771416. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)
Study identification
- NCT ID
- NCT04771416
- Recruitment status
- Suspended
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Enrollment
- 24 participants
Conditions and interventions
Conditions
Interventions
- PBKR03 Biological
Biological
Eligibility (public fields only)
- Age range
- 1 Month to 9 Months
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 23, 2022
- Primary completion
- Dec 31, 2026
- Completion
- Dec 31, 2029
- Last update posted
- Nov 12, 2025
2022 – 2030
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ann & Robert Lurie | Chicago | Illinois | 60611 | — |
| New York-Presbyterian | New York | New York | 10065 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Utah School of Medicine | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04771416, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 12, 2025 · Synced May 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04771416 live on ClinicalTrials.gov.