Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or

Andy,

Do you know anything about this study and what it entails?

https://clinicaltrials.gov/ct2/show/NCT00669305?term=thalassemia&rank=26

Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia
This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2015 by St. Jude Children's Research Hospital
Sponsor:
St. Jude Children's Research Hospital
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Assisi Foundation
Doris Duke Charitable Foundation
University of Tennessee
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00669305
First received: April 28, 2008
Last updated: December 3, 2015
Last verified: December 2015
History of Changes
Full Text View Tabular ViewNo Study Results PostedDisclaimerHow to Read a Study Record
  Purpose
Using sickle cell and thalassemia mouse models, researchers will evaluate the possibility of correcting these disorders by inserting healthy genetic material into the diseased blood cells or drug treatment. Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in the mouse models.

Condition   Intervention
Sickle Cell Anemia
Thalassemia
Genetic: Gene Therapy

Study Type:   Interventional
Study Design:   Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Basic Science
Official Title:   Experimental Evaluation of the Potential to Correct the Pathophysiology of Sickle Cell Anemia or Thalassemia by Retroviral Vector Mediated Globin Gene Transfer

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease
MedlinePlus related topics: Anemia Genes and Gene Therapy Sickle Cell Anemia Thalassemia
Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia Thalassemia
U.S. FDA Resources

Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
Percentage of successful achievement of therapeutic level in mouse models resulting from retroviral vector mediated gene transfer, gene editing or drug treatment. [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]
The specific hypothesis to be tested is that a gene therapy vector, gene editing strategy, or drug modality can be designed that achieves a therapeutic level of globin production in transduced cells in mouse models.


Estimated Enrollment:   28
Study Start Date:   July 2007
Estimated Study Completion Date:   July 2018
Estimated Primary Completion Date:   July 2017 (Final data collection date for primary outcome measure)
Arms   Assigned Interventions
1   Genetic: Gene Therapy
Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in the mouse models

Detailed Description:
These studies are designed to evaluate the potential of retroviral vector mediated gene transfer, gene editing, or drug treatment to correct the pathophysiology of sickle cell anemia and β-thalassemia. CD34+ cells purified from bone marrow of research participants with a sickle cell syndrome or a thalassemia syndrome will be subjected to genetic editing, drug treatment, or transduced with retroviral vectors containing γ-globin coding sequences under the control of the β-globin gene promoter and including various regulatory elements chosen to enhance gene expression and to insulate regulatory elements from cellular genes at or near the integration sites. The efficiency of gene transfer and the function of the globin transgene will be evaluated in erythroid cells derived from transduced progenitors and from the progenitors in the bone marrow of immunodeficient mice engrafted with transduced, primitive hematopoietic cells. The hypothesis to be tested in this research is that a gene therapy vector, gene editing strategy, or drug modality can be designed to achieve a potentially therapeutic level of globin gene expression in maturing erythroid cells.
  Eligibility

Genders Eligible for Study:     Both
Accepts Healthy Volunteers:     No
Criteria
Inclusion Criteria:

Patients with homozygous S/S disease or doubly heterozygous for S and β thalassemia who are 2 years or older are eligible. Patients with HbE- β- thalassemia or homozygous (severe) β-thalassemia are also eligible. Patients with thalassemia include those who are transfusion dependent (major) or severely anemic but relatively transfusion independent (intermedia). Diagnostic criteria include standard hematological parameters, red cell indices, hemoglobin electrophoresis and quantitative determination of HbF and HbA2.
Patients are eligible for participation in the protocol only if they are currently clinically stable and have been free of all acute disease manifestations for a minimum of 14 days.
Patients may participate while continuing their current therapeutic regimen including regular transfusion therapy or hydroxyurea administration.
In general, two categories of patients will be considered as research participants in this protocol.
Patients who are 18 years or older and therefore able to provide informed consent will be eligible. Such individuals will be recruited from among patients followed at SJCRH. In addition, individuals followed in an outside clinic who are recruited will be asked to come to the Hematology Clinic at SJCRH to enroll and have the procedure performed. Alternatively, if a patient who is 18 or older is to undergo a diagnostic or surgical procedure under general anesthesia, and they agree to participate in the study, the bone marrow aspirate will be obtained at that time.
Patients between the ages of 2 and 17 years who are scheduled for a diagnostic or surgical procedure at SJCRH or LeBonheur Children's Medical Center for which sedation or general anesthesia is indicated will be eligible for protocol enrollment. A bone marrow aspiration will be performed during the sedation or general anesthesia for the diagnostic or surgical procedure.
Exclusion Criteria:

Active, acute manifestations of sickle cell disease including painful crisis, acute chest syndrome, cerebrovascular events or active infection.
Pregnant women will not be eligible for study enrollment.
Inability or unwillingness of the research participant or legal guardian/representative to give written informed consent will preclude enrollment on this research protocol.
Platelet count < 150,000/mm^3
Neutrophil count < 2000/mm^3 (unless on hydroxyurea therapy)
Neutrophil count < 1000/mm^3 for patients on hydroxyurea therapy
Prothrombin Time > 17 seconds
Partial thromboplastin Time > 43 seconds
History of excessive bleeding in the context of previous procedures including surgery and dental extractions
   Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00669305

Contacts
Contact: Mitch Weiss, MD, PhD   1-866-278-5833   referralinfo@stjude.org   

Locations
United States, Tennessee
St. Jude Children's Research Hospital   Recruiting
Memphis, Tennessee, United States, 38105
Contact: Mitch Weiss, MD, PhD    866-278-5833    referralinfo@stjude.org  
Principal Investigator: Mitch Weiss, MD, PhD        
Sponsors and Collaborators
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)
Assisi Foundation
Doris Duke Charitable Foundation
University of Tennessee
Investigators
Principal Investigator:   Mitch Weiss, MD, PhD   St. Jude Children's Research Hospital   
  More Information

Additional Information:
St. Jude Children's Research Hospital   This link exits the ClinicalTrials.gov site
Clinical Trials Open at St. Jude   This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:   St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:   NCT00669305     History of Changes
Other Study ID Numbers:   EPSTRV, U54HL070590, P01HL053749, 201003
Study First Received:   April 28, 2008
Last Updated:   December 3, 2015
Health Authority:   United States: Institutional Review Board

Keywords provided by St. Jude Children's Research Hospital:
Genetics
Blood cells
Vector-mediated gene transfer
Bone marrow
CD34 cells

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

Thank you,

Sharmin

Thanks for the update. I do recall this was mentioned by someone last year as another option for thals. No specifics were discussed. Worth looking into it.

Kindly,

I've heard about this research at St Judes for years. It's good to see it's finally coming to trials.