Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects… (NCT07476183) | Clinical Trial Compass
RecruitingPhase 1
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
United States4 participantsStarted 2026-04-16
Plain-language 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.
Who can participate
Age range2 Years – 18 Years
SexALL
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Inclusion criteria
✓. Confirmed diagnosis of RPS19-deficient DBA.
✓. Signed informed consent by the subject or legally authorized representative.
✓. Bone marrow analysis demonstrating normal cytogenetics except for RPS19-deficient DBA.
✓. Subjects are between 2 and 18 years of age, inclusive.
✓. Eligible for allogeneic marrow or stem cell transplant for DBA (non-critical cardiac and hepatic iron overload).
✓. Corticosteroid resistance
✓. Transfusion-dependent anemia
✓. Willingness to return for long-term follow-up
Exclusion criteria
✕. Availability of a suitable, consenting HLA-identical sibling donor.
✕. Positive viral serology.
✕
What they're measuring
1
Incidence of protocol-defined dose limiting toxicities (DLTs) for APR-2020
Timeframe: 30 Days
2
Incidence and severity of treatment emergent adverse events, assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE)
Timeframe: 24 Months
3
Monitoring of laboratory parameters, frequency and severity of clinical adverse events (AEs), assessed by the NCI CTCAE
Timeframe: 24 Months
4
Fraction of reticulocytes greater than the baseline value where reticulocyte increases are sustained over 3 consecutive measurements within 4 weeks
Timeframe: 24 Months
5
Proportion of subjects with hemoglobin level of at least 8 g/dL starting 90 days after last RBC transfusion, sustained over 2 consecutive measurements that are approximately 1 month apart
. Clinically significant, active bacterial, viral, or fungal infection.
✕. Any prior or current malignancy, myeloproliferative disorder, or myelodysplastic syndrome, except where therapy was curative excision (ie, in situ squamous cell carcinoma).
✕. Any concerning molecular or cytogenetic abnormalities in hematopoietic cells.
✕. Previous receipt of an allogeneic transplant or gene therapy.
✕. Immediate family member with a known or suspected Familial Cancer Syndrome (including, but not limited to breast, colorectal, ovarian, prostate, and pancreatic cancers, excluding DBA).
✕. Diagnosis of significant psychiatric disorder that could impact the subject's ability to participate in the study, in the opinion of the Investigator.