RPS19 Deficient Diamond-Blackfan Anemia study: APR-2020
Official title Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
ClinicalTrials.gov ID: NCT07476183
What this study is testing
What is APR-2020?
This is an investigational cell therapy being studied as a potential treatment for Diamond-Blackfan Anemia. It is designed to use your own blood cells, which are changed in a lab to fix a specific gene problem, and then put back into your body.
Also referred to as Cell Therapy.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study checks if the treatment is safe and if it can fix the genetic cause of the disease. It also looks to see if it can reduce or stop the need for blood transfusions.
- Who it's for
- This study is for children and teenagers who have Diamond-Blackfan Anemia caused by a specific gene problem (RPS19).
- What's involved
- Participants will receive the drug once through an IV. They will also have two procedures to collect their own cells, which are used to make their specific treatment.
- Time commitment
- The study involves two cell collection procedures and a one-time infusion, followed by two years of check-up visits that become less frequent over time.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 25
You may be able to join if
- Confirmed diagnosis of RPS19-deficient DBA.
- Signed informed consent by the subject or legally authorized representative.
- Bone marrow analysis demonstrates normal cytogenetics except for RPS19-deficient DBA.
- Between 2 and 25 years of age, inclusive.
- Eligible for allogeneic marrow or stem cell transplant for DBA (noncritical cardiac and hepatic iron overload).
You likely can't join if
- 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...
- Any concerning cytogenetic abnormalities in hematopoietic cells.
- Previous receipt of an allogeneic transplant or gene therapy.
See the full eligibility criteria
- Confirmed diagnosis of RPS19-deficient DBA.
- Signed informed consent by the subject or legally authorized representative.
- Bone marrow analysis demonstrates normal cytogenetics except for RPS19-deficient DBA.
- Between 2 and 25 years of age, inclusive.
- Eligible for allogeneic marrow or stem cell transplant for DBA (noncritical cardiac and hepatic iron overload).
- Corticosteroid resistance
- Transfusion-dependent anemia
- Willingness to return for long-term follow-up
- Adequate renal and pulmonary function
- Able to undergo hematopoietic stem cell transplant (HSCT) mobilization and apheresis procedures. Key
- 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 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.
- History of complex allo-immunization, as determined by the Investigator.
- Female people who are lactating/breast feeding or who plan to breastfeed within 6 months following APR-2020 infusion.
- Male and female people of childbearing potential who are unwilling to practice highly effective methods of birth control from screening until ≥ 6 months after APR-2020 infusion.
- Female people with a positive serum pregnancy test at Screening or who are planning to become pregnant during the study period.
- Liver disease, as evidenced by critical iron overload with magnetic resonance imaging (MRI)
- Heart disease or Type 1 diabetes.
- Evidence of significant pulmonary hypertension, per Investigator assessment.
- Any other condition that would render the subject ineligible for HSCT, as determined by the Investigator.
- Contraindication to stem cell or bone marrow aspiration, mobilization or collection including allergies to filgrastim or plerixafor.
- Currently enrolled in another investigational drug study or received an investigational study drug or procedure within 90 days of study enrollment.
- A physical, functional, or emotional status that would prevent giving informed consent, protocol compliance, or adequate follow-up.
- An assessment by the Investigator that the subject or parents of the subject will not comply with the study procedures outlined in the study protocol.
- Taking prohibited medications.
- Has insufficient personal history of RBC transfusions over the 13 weeks prior to the end of screening.
The study team makes the final eligibility decision.
Where it is enrolling
- Stanford, California, United States
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Stanford, California, United States; Boston, Massachusetts, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.