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

Conditions

• RPS19 Deficient Diamond-Blackfan Anemia

Eligibility Criteria

Sex

ALL

Age

2 Years - 18 Years

Healthy Volunteers

Not accepted

Interventions

• APR-2020 — BIOLOGICAL
  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.

Study Details

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.

Key Dates

Start date

Apr 16, 2026

Status verified

May 2026

Primary completion

Apr 30, 2027

Completion

Sep 30, 2028

Study Design

Enrollment

4 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: APR-2020

Primary Outcome Measure

Incidence of protocol-defined dose limiting toxicities (DLTs) for APR-2020 [ Time Frame: 30 Days ]

Central Contacts

• Nova Silver
  617-306-3020
  [email protected]

Locations (2)

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