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Trial record 1 of 1 for:    Gene Transfer for Patients with Sickle Cell Disease Cincinnati
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Gene Transfer for Patients With Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT02186418
Recruitment Status : Recruiting
First Posted : July 10, 2014
Last Update Posted : August 30, 2017
Sponsor:
Collaborator:
Doris Duke Charitable Foundation
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Brief Summary:
The purpose of this study is to determine whether transfer of a fetal hemoglobin gene using a lentivirus vector (gene transfer) into human blood making cells is safe and feasible in patients with sickle cell disease.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Genetic: Gene transfer Phase 1 Phase 2

Detailed Description:
The purpose is also to determine if this kind of gene transfer works in getting the body to make red blood cells that don't sickle in patients with sickle cell disease.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Gamma Globin Lentivirus Vector
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Gene Transfer for Patients With Sickle Cell Disease Using a Gamma Globin Lentivirus Vector: An Open Label Phase I/II Pilot Study
Study Start Date : July 2014
Estimated Primary Completion Date : August 2032
Estimated Study Completion Date : August 2033

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Gene transfer
gene transfer - getting the body to make red blood cells that don't sickle in patients with sickle cell disease.
Genetic: Gene transfer
Gene transfer using a gamma-globin lentivirus vector



Primary Outcome Measures :
  1. Evaluate Safety of Gene Transfer Therapy for Sickle Cell Disease [ Time Frame: from study entry to 15 years post infusion of gene modified cells ]
    Evaluate the safety of bone marrow collection, gene transfer and chemotherapy conditioning in subjects with SCD

  2. Evaluate the Feasibility of Gene Transfer Therapy for Sickle Cell Disease [ Time Frame: 1 year ]
    Evaluate the feasibility of obtaining sufficient autologous gene modified stem cells that can engraft the subject with SCD



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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed sickle cell disease that is severe
  • Failed hydroxyurea therapy, were unable to tolerate hydroxyurea therapy, or, if 18 years of age or older, have actively made the choice to not take the recommended daily hydroxyurea. Patients who are unable to take hydroxyurea due to financial or safety monitoring constraints are eligible.
  • Lansky/Karnovsky performance score≥ 60
  • Ability to undergo general or regional(spinal or epidural) anesthesia
  • Adequate kidney, liver and heart function as determined by clinical tests
  • Willing to reside within a 120 mile radius of Cincinnati Children's Hospital Medical Center for a period of 3-6 months

Exclusion Criteria:

  • Active malignant disease
  • Sero-positive for HIV
  • Taken an experimental drug in past 30 days
  • Abnormal pulmonary function tests that show severe obstruction, restriction or diffusion defects
  • Patients who are on chronic transfusions for:

    • clinical stroke
  • Pregnant or breast-feeding
  • Alpha thalassemia
  • Cirrhosis of liver, bridging hepatic fibrosis, or active hepatitis
  • Patients with matched sibling donors who have not declined the option of a hematopoietic stem cell transplant
  • Confirmed pulmonary hypertension
  • Any condition which in the opinion of the investigator makes participation ill advised

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02186418


Contacts
Contact: Amy Shova 513-636-6770 Sicklecell@cchmc.org

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Amy Shova    513-803-1917    Sicklecell@cchmc.org   
Principal Investigator: Punam Malik, MD         
Jamaica
Caribbean Institute for Health Research-Sickle Cell Unit, The University of the West Indies Recruiting
Kingston, Jamaica, 7
Contact: Monika Parshad-Asnani    876-977-6151-2      
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Doris Duke Charitable Foundation
Investigators
Principal Investigator: Punam Malik, MD Children's Hospital Medical Center, Cincinnati

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT02186418     History of Changes
Other Study ID Numbers: 2010-2588
2013122 ( Other Grant/Funding Number: Doris Duke Charitable Foundation )
First Posted: July 10, 2014    Key Record Dates
Last Update Posted: August 30, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Children's Hospital Medical Center, Cincinnati:
sickle cell disease
sickle cell anemia
hemoglobinopathies
hematopoietic stem cell
gene transfer
gene therapy

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn