APR-2020 for RPS19 Deficient Diamond-Blackfan Anemia
This study is testing APR-2020 to see if it is safe and effective for children and adolescents with RPS19-deficient Diamond-Blackfan Anemia (DBA). DBA is a rare blood disorder where the bone marrow doesn't make enough red blood cells. APR-2020 is made from your own cells that are specially treated. Researchers want to know if APR-2020 is well-tolerated, can help correct the genetic problem causing DBA, and reduce or eliminate the need for blood transfusions. The study plans to enroll 4 participants between 2 and 25 years old who have a confirmed diagnosis of RPS19-deficient DBA and are eligible for a bone marrow transplant.
- Study design
- This is an open-label, single-arm study, meaning all 4 participants will receive APR-2020, and everyone involved will know what treatment is being given.
- What's involved
- You will receive APR-2020 as a one-time infusion. You will also undergo two procedures to collect your cells, which will be used to create your personalized treatment. You will have clinic visits for two years after treatment, with visits becoming less frequent over time.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for 24 months to monitor for side effects and changes in laboratory parameters.
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Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
At a glance
Conditions
Where it's being run
2 sites across 2 statesWho to contact
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What this trial measures
- Incidence of protocol-defined dose limiting toxicities (DLTs) for APR-202030 Days
- Incidence and severity of treatment emergent adverse events (TEAEs), assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE)24 Months
- Monitoring laboratory parameters, frequency and severity of TEAEs, assessed by the NCI CTCAE24 Months
- Fraction of reticulocytes greater than the baseline value where reticulocyte increases are sustained over 3 consecutive measurements within 4 weeks24 Months
Baseline is defined as the average of 3 measurements prior to APR-2020 infusion
- Proportion of subjects with a hemoglobin level of ≥ 8 g/dL starting 90 days after the last RBC transfusion, sustained over 2 consecutive measurements that are approximately 1 month apart24 Months