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 range
2 Years – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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
. Availability of a suitable, consenting HLA-identical sibling donor.
. Positive viral serology.
. 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.