Gene Transfer for Patients with Sickle Cell Disease

Why are we doing this research?

Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, because you have sickle cell disease that is affecting your life in a bad way. The current ways to treat the disease just help with the symptoms of the disease. This study is looking at using a new treatment called “gene transfer” to try and get your body to permanently make red blood cells that don’t sickle and to see if this is safe and effective.

Who can participate?

 Patients must: 

  • Have Sickle β° thalassemia or Sickle β+ thalassemia followed by Hb SS 
  • Be 18-35 years old 
  • Live or willing to live within a 120 mile drive of Cincinnati Children’s Hospital Medical Center for 3-6 months.

 

Conditions

  • Hematology - Sickle Cell and Hemoglobin

What will happen in the study?

You, as a participant or as a parent/guardian, will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.

What is involved:  

    • Screening procedures will be done to determine if you are eligible to participate in this study.
    • A surgical procedure, called a bone marrow harvest, will be performed to collect blood making cells.
    • A chemotherapy drug called melphalan will be given to get you ready to receive the “gene transfer” blood-making cells.
    • The “gene transfer” blood-making cells that were collected from your bone marrow harvest will be given back to you via a vein in your arm.
    • You will be followed for approximately 15 years.

 

What are the good things that can happen from this research?

Being in this study may or may not help you right now. When we finish the study, we hope that we will know more about using gene transfer in people with sickle cell disease. The possible benefit is that the gene transfer may reduce your current sickle cell symptoms and stop further damage, or that it may decrease the amount of supportive care you have been receiving, such as transfusions and pain medications.

What are the bad things that can happen from this research?

Possible risks and side effects will be provided to and discussed with those participants, parents or guardians interested in knowing more about this study. You may already be familiar with many of the tests and procedures done during this study. Many of these tests are regularly used to monitor your sickle cell disease.

Will you/your child be paid to be in this research study?

You will be paid for time/travel to participate in this study. All research procedures will be covered by the study.

Contact

Contact Us.Amy Shova
513-803-1917
Amy.Shova@cchmc.org

Courtney Little RN, BSN
513-803-0226
Courtney.Little@cchmc.org

Cincinnati Children's Hospital Medical Center
3333 Burnet Avenue, MLC 7015
Cincinnati, OH 45229-3039

Study Doctor

Contact Us.Punam Malik, MD
Cincinnati Children’s Hospital Medical Center
Director, Comprehensive Sickle Cell Program

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