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Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

NCT07476183 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2026-05-15

No results posted yet for this study

Summary

Brief summary

The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The main questions it aims to answer are:

* Is APR-2020 safe and well tolerated?
* Does APR-2020 modify or correct an underlying genetic condition which causes DBA?
* Does APR-2020 reduce or eliminate the need for blood transfusions and/or restore certain blood counts affected by DBA?

Participants will:

* Take the drug one time as an infusion.
* Undergo two rounds of a cellular harvest procedure in which their own cells will be used in the manufacturing of their own participant-specific product.
* Initially return to the clinic for two years of follow up at increasingly sparse intervals.

Conditions

  • RPS19 Deficient Diamond-Blackfan Anemia

Interventions

BIOLOGICAL

APR-2020

The drug product (DP) is designated as APR-2020 and is composed of autologous human CD34+ cells derived from each clinical patient with RPS19-deficient DBA that have been transduced ex vivo with the drug substance.

Sponsors & Collaborators

  • Apriligen, Inc.

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-16
Primary Completion
2027-04-30
Completion
2028-09-30
FDA Drug
Yes

Countries

  • United States

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 NCT07476183 on ClinicalTrials.gov