Boston, MANCT07476183Now EnrollingIRB Ready

RPS19 Deficient Diamond-Blackfan Anemia Clinical Trial in Boston, MA

Access cutting-edge rps19 deficient diamond-blackfan anemia treatment through this clinical trial at a research site in Boston. Study-provided care at no cost to qualified participants.

Sponsored by Apriligen, Inc.

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Expert Care in Boston

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IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related rps19 deficient diamond-blackfan anemia treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Boston

    Convenient for MA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Boston site if eligible
  4. 4Begin participation

About This RPS19 Deficient Diamond-Blackfan Anemia Study in Boston

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.

Sponsor: Apriligen, Inc.

Who Can Participate

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
Adequate renal and pulmonary function
Able to undergo hematopoietic stem cell transplant (HSCT) mobilization and apheresis procedures. Key

Exclusion Criteria

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.
History of complex allo-immunization, as determined by the Investigator.
Women who are lactating/breast feeding or who plan to breastfeed within 6 months following APR-2020 infusion.
Men and females of childbearing potential who are unwilling to practice highly effective methods of birth control through the duration of the study.
Females 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 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 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.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Boston?

Yes, this clinical trial (NCT07476183) has an active research site in Boston, MA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

RPS19 Deficient Diamond-Blackfan Anemia Treatment Options in Boston, MA

If you're searching for rps19 deficient diamond-blackfan anemia treatment options in Boston, MA, this clinical trial (NCT07476183) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Boston research site is actively enrolling participants for this clinical trial. You'll receive care from experienced rps19 deficient diamond-blackfan anemia specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all rps19 deficient diamond-blackfan anemia clinical trials near you to find additional studies recruiting in your area.

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