Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Public ClinicalTrials.gov record NCT07476183. 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, Open-label, Single-arm Study of APR 2020 in Transfusion Dependent, Steroid Resistant Pediatric and Adolescent Subjects With RPS19 Deficient Diamond Blackfan Anemia by Transplantation of Autologous CD34+ Stem Cells Transduced With CLIN LV EFS coRPS19 PRE* (APR-2020)
Study identification
- NCT ID
- NCT07476183
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Apriligen, Inc.
- Industry
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Interventions
- APR-2020 Biological
Biological
Eligibility (public fields only)
- Age range
- 2 Years to 18 Years
- 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
- Apr 15, 2026
- Primary completion
- Mar 31, 2027
- Completion
- Aug 31, 2028
- Last update posted
- May 14, 2026
2026 – 2028
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University - Stanford Children's Health | Stanford | California | 94304 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02215 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT07476183, 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 May 14, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT07476183 live on ClinicalTrials.gov.