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

StatusRecruiting
PhasePhase 1
Started
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary endpoint reworded 3 of 5 changed
See other at-risk trials from Apriligen, Inc.

Record History

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  1. Primary endpoint(s) modified
    • Eligibility criteria expanded, about 21 words
    • Secondary endpoints revised (7)
    v5
  2. Site details revised at 1 of 2 sitesv4
  3. Recruitment opened
    • Primary completion pushed from Mar 2027 to Apr 2027
    • Start date changed from estimate of Mar 2026 to Apr 16, 2026
    • Site details revised at 2 of 2 sites
    v3
  4. Trial status: Recruiting to Not Yet Recruiting
    • Site details revised at 2 of 2 sites
    v2
  5. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT07476183
Lead Sponsor Apriligen, Inc.
Conditions
(from sponsor)		 RPS19 Deficient Diamond-Blackfan Anemia
Related terms Anemia, Diamond-Blackfan
Enrollment 4 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov