New Hyde Park, NYNCT07186179Now EnrollingIRB Ready

Diamond Blackfan Anemia Clinical Trial in New Hyde Park, NY

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

Sponsored by Northwell Health

Quick Self-Assessment

See if you qualify for this New Hyde Park location

Preparing your pre-screening questions…

Expert Care in New Hyde Park

Access diamond blackfan anemia specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related diamond blackfan anemia treatment provided free

Apply for This New Hyde Park Location

Check if you qualify for this diamond blackfan anemia clinical trial in New Hyde Park, NY

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to New Hyde Park

    Convenient for NY 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 New Hyde Park site if eligible
  4. 4Begin participation

About This Diamond Blackfan Anemia Study in New Hyde Park

Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS. This involves inserting the corrected RPS19 gene into the cells, leading to correction of the anemia. The application of gene therapy requires sufficient numbers of stem cells on which the correction can be performed. Stem cells must be mobilized (stimulated to move) from the bone marrow to the peripheral blood and then collected (also called 'harvested'). It is not known if patients with DBAS can mobilize enough stem cells into the peripheral blood to allow for the harvesting of sufficient numbers to permit genetic manipulation. It is important to demonstrate the ability to harvest an adequate number of stem cells before gene therapy can be tried in patients with DBAS. The purpose of this study is to determine if mobilization of stem cells from the bone marrow in patients with DBAS is enough to obtain the numbers of peripheral blood stem cells necessary for effective gene therapy. An actual harvest will not be done.

Sponsor: Northwell Health

Who Can Participate

Inclusion Criteria

Diamond Blackfan anemia syndrome as defined by the known criteria with a known gene mutation
Male or female patients of all ethnic background, greater than or equal to 3 years of age and weighing at least 10 kg, and less than or equal to 30 years of age
Enrolled in Diamond Blackfan Anemia Registry of North America (DBAR)
Chronically red blood cell transfusion dependent for at least 6 months
Performance scale (Lansky Play-performance Scale for Pediatric Functional Status for age \<16 years; Karnofsky Performance Scale for age ≥16 years) ≥ 70
Must sign informed consent

Exclusion Criteria

Receiving prednisone therapy for treatment of DBAS (this does not include patients receiving physiologic steroid replacement for adrenal insufficiency)
Known history of myelodysplasia or presence of a hematopoietic clone
Current malignancy or previous treatment for malignancy
Pregnancy or breast-feeding mother
Known history of severe iron overload as defined by a liver iron concentration (LIC) \> 15 mg Fe/ g dry liver weight
Significant cytopenias, defined as:
Platelet count \<100,000/mcL
Absolute neutrophil count \<750/mCL
Any GCSF use in the 3 months prior to enrollment
Liver dysfunction: aspartate aminotransferase (AST), alanine aminotransferase (ALT), or direct bilirubin values \>3 x the upper limit of normal (ULN)
Kidney dysfunction: baseline estimated glomerular filtration rate (GFR) \<70 mL/min/1.73 m2

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 New Hyde Park?

Yes, this clinical trial (NCT07186179) has an active research site in New Hyde Park, NY 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.

Diamond Blackfan Anemia Treatment Options in New Hyde Park, NY

If you're searching for diamond blackfan anemia treatment options in New Hyde Park, NY, this clinical trial (NCT07186179) 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 New Hyde Park research site is actively enrolling participants for this clinical trial. You'll receive care from experienced 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 diamond blackfan anemia clinical trials near you to find additional studies recruiting in your area.

More Heart Attack Trials in New Hyde Park, NY

See all heart attack clinical trials recruiting in New Hyde Park — not just this study.

Browse Heart Attack Trials in New Hyde Park

Ready to Join in New Hyde Park?

Take the first step toward participating in this groundbreaking clinical trial

Secure · Expert Care · New Hyde Park, NY