Actively Recruiting
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Led by Apriligen, Inc. · Updated on 2026-04-20
4
Participants Needed
2
Research Sites
124 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of RPS19-deficient Diamond-Blackfan Anemia.
- Signed informed consent by the subject or legally authorized representative.
- Bone marrow analysis showing normal cytogenetics except for RPS19-deficient DBA.
- Age between 2 and 18 years, inclusive.
- Eligible for allogeneic marrow or stem cell transplant for DBA with non-critical cardiac and hepatic iron overload.
- Resistant to corticosteroid treatment.
- Dependent on blood transfusions.
- Willing to return for long-term follow-up.
- Adequate kidney and lung function.
- Able to undergo hematopoietic stem cell transplant mobilization and apheresis procedures.
You will not qualify if you...
- 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 curatively excised in situ squamous cell carcinoma.
- Concerning molecular or cytogenetic abnormalities in hematopoietic cells.
- Previous allogeneic transplant or gene therapy.
- Immediate family member with known or suspected familial cancer syndrome (excluding DBA).
- Significant psychiatric disorder affecting study participation.
- History of complex allo-immunization.
- Women who are breastfeeding or plan to breastfeed within 6 months after APR-2020 infusion.
- Men and females of childbearing potential unwilling to use highly effective birth control during the study.
- Females with positive pregnancy test at screening or planning pregnancy during the study.
- Liver disease with critical iron overload.
- Heart disease or Type 1 diabetes.
- Significant pulmonary hypertension.
- Any condition making the subject ineligible for stem cell transplant.
- Contraindications to stem cell or bone marrow collection including allergies to filgrastim or plerixafor.
- Currently enrolled in another investigational drug study or received investigational therapy within 90 days.
- Physical or emotional status preventing informed consent, protocol compliance, or follow-up.
- Investigator assessment of likely non-compliance with study procedures.
- Taking prohibited medications.
AI-Screening
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Trial Site Locations
Total: 2 locations
1
Stanford University - Stanford Children's Health
Stanford, California, United States, 94304
Not Yet Recruiting
2
Boston Children's Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
N
Nova Silver
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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