Safety Study of Gene Modified Donor T-cells Following TCR + Alpha Beta Depleted Stem Cell Transplant

Description

This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially matched family donor. The study will assess whether immune cells, called T cells, from the family donor, that are specially grown in the laboratory and given back to the patient along with the stem cell transplant can help the immune system recover faster after transplant. As a safety measure these T cells have been programmed with a self-destruct switch so that they can be destroyed if they start to react against tissues (Graft versus host disease).

Study Start Date

April, 01 2014

Estimated Completion Date

December 2019

Interventions

  • Biological: BPX-501 T cells and AP1903
  • Drug: AP1903

Study ID

Bellicum Pharmaceuticals -- BP-U-004

Status

Unknown

Trial ID

NCT03301168

Study Type

Interventional

Trial Phase

Phase 1/Phase 2

Enrollment Quota

120

Sponsor

Bellicum Pharmaceuticals

Inclusion Criteria

    1. Age < 18 years and > 1 month (< 1 month upon approval by Sponsor) 2. Life expectancy > 10 weeks 3. Patients deemed clinically eligible for allogeneic stem cell transplantation. 4. Patients may have failed prior allograft 5. Patients with life-threatening acute leukemia (high-risk ALL in 1st CR, ALL in 2nd CR, high-risk AML in 1st CR, AML in 2nd CR.) or myelodysplastic syndromes. Morphological CR must be documented and minimal residual disease measurement before transplantation is recommended. 6. Non-malignant disorders deemed curable by allogeneic transplantation: a. primary immune deficiencies, b. severe aplastic anemia not responding to immune suppressive therapy, c. osteopetrosis d. selected cases of erythroid disorders such as ?0 ?0 thalassemia major, sickle cell disease, Diamond-Blackfan anemia. e. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML). Note: Subjects will be eligible if they meet either item 5 OR item 6. 7. Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative or 10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor 8. A minimum genotypic identical match of 5/10 is required. 9. The donor and recipient must be identical, as determined by high resolution typing, on at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA- DRB1 and HLA-DQB1. 10. Lansky/Karnofsky score > 50 11. Signed informed consent by the patient or the patient's parent or guardian for patients who are minors

Exclusion Criteria

    1. Greater than active Grade II acute GvHD or chronic extensive GvHD due to a previous allograft at the time of screening 2. Patient receiving an immunosuppressive treatment for GvHD treatment due to a previous allograft at the time of screening 3. Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance <30ml/min/1.73m2) 4. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%) 5. Current clinically active infectious disease (including positive HIV serology or viral RNA) 6. Serious concurrent uncontrolled medical disorder 7. Pregnant or breast feeding female patient 8. Lack of parents'/guardian's informed consent for children who are minors.

Gender

All

Ages

1 Month to 18 Years

Accepts Healthy Volunteers

No

Study Locations and Contact Information (39)

Study Location Distance Name Phone Email
DanaFarber Boston Childrens Cancer and Blood Disorders Center - Boston, Massachusetts 2.4 miles Colleen Dansereau 617-919-7008 colleen.dansereau@childrens.harvard.edu
DanaFarber Boston Childrens Cancer and Blood Disorders Center - Boston, Massachusetts 2.4 miles Colleen Dansereau 617-919-7008 colleen.dansereau@childrens.harvard.edu
DanaFarber Boston Childrens Cancer and Blood Disorders Center - Boston, Massachusetts 2.4 miles Colleen Dansereau 617-919-7008 colleen.dansereau@childrens.harvard.edu
DanaFarber Boston Childrens Cancer and Blood Disorders Center - Boston, Massachusetts 2.4 miles Colleen Dansereau 617-919-7008 colleen.dansereau@childrens.harvard.edu
Childrens Hospital at Montefiore - The Bronx, New York 176.6 miles Ruth Santizo 718-920-6506 rsantizo@montefiore.org
Childrens Hospital at Montefiore - The Bronx, New York 176.6 miles Ruth Santizo 718-920-6506 rsantizo@montefiore.org
Childrens Hospital at Montefiore - The Bronx, New York 176.6 miles Ruth Santizo 718-920-6506 rsantizo@montefiore.org
Childrens Hospital at Montefiore - The Bronx, New York 176.6 miles Ruth Santizo 718-920-6506 rsantizo@montefiore.org
Childrens National Medical Center - Washington, D.C., District of Columbia 391.7 miles Divyesh Kukadiya 202-476-6850 Dkukadiy@childrensnational.org
Childrens National Medical Center - Washington, D.C., District of Columbia 391.7 miles Divyesh Kukadiya 202-476-6850 Dkukadiy@childrensnational.org
Childrens National Medical Center - Washington, D.C., District of Columbia 391.7 miles Divyesh Kukadiya 202-476-6850 Dkukadiy@childrensnational.org
Childrens National Medical Center - Washington, D.C., District of Columbia 391.7 miles Divyesh Kukadiya 202-476-6850 Dkukadiy@childrensnational.org
Childrens Healthcare of Atlanta - Atlanta, Georgia 932.0 miles Ashley Griffin 404-785-0653 ashley.griffin@choa.org
Childrens Healthcare of Atlanta - Atlanta, Georgia 932.0 miles Ashley Griffin 404-785-0653 ashley.griffin@choa.org
Childrens Healthcare of Atlanta - Atlanta, Georgia 932.0 miles Ashley Griffin 404-785-0653 ashley.griffin@choa.org
Childrens Healthcare of Atlanta - Atlanta, Georgia 932.0 miles Ashley Griffin 404-785-0653 ashley.griffin@choa.org
University of Texas SouthwesternChildrens Medical Center - Dallas, Texas 1,585.2 miles Gevel Jackson CCRP 214-456-1430 Gevel.Jackson@childrens.com
University of Texas SouthwesternChildrens Medical Center - Dallas, Texas 1,585.2 miles Gevel Jackson CCRP 214-456-1430 Gevel.Jackson@childrens.com
University of Texas SouthwesternChildrens Medical Center - Dallas, Texas 1,585.2 miles Gevel Jackson CCRP 214-456-1430 Gevel.Jackson@childrens.com
University of Texas SouthwesternChildrens Medical Center - Dallas, Texas 1,585.2 miles Gevel Jackson CCRP 214-456-1430 Gevel.Jackson@childrens.com
Baylor College of Medicine Texas Childrens Hospital - Houston, Texas 1,609.9 miles Marlen Dinu 832-824-4881 mxdinu@txch.org
Baylor College of Medicine Texas Childrens Hospital - Houston, Texas 1,609.9 miles Marlen Dinu 832-824-4881 mxdinu@txch.org
Baylor College of Medicine Texas Childrens Hospital - Houston, Texas 1,609.9 miles Marlen Dinu 832-824-4881 mxdinu@txch.org
Baylor College of Medicine Texas Childrens Hospital - Houston, Texas 1,609.9 miles Marlen Dinu 832-824-4881 mxdinu@txch.org
Fred Hutchinson Cancer Research Center - Seattle, Washington 2,490.2 miles Michelle Bouvier 206-667-6993 meb@fhcrc.org
Fred Hutchinson Cancer Research Center - Seattle, Washington 2,490.2 miles Michelle Bouvier 206-667-6993 meb@fhcrc.org
Fred Hutchinson Cancer Research Center - Seattle, Washington 2,490.2 miles Michelle Bouvier 206-667-6993 meb@fhcrc.org
Oregon Health Sciences University Doernbecher Childrens Hospital - Portland, Oregon 2,538.3 miles Jennifer Williamson 503-494-3639 willjenn@ohsu.edu
Oregon Health Sciences University Doernbecher Childrens Hospital - Portland, Oregon 2,538.3 miles Jennifer Williamson 503-494-3639 willjenn@ohsu.edu
Oregon Health Sciences University Doernbecher Childrens Hospital - Portland, Oregon 2,538.3 miles Jennifer Williamson 503-494-3639 willjenn@ohsu.edu
Oregon Health Sciences University Doernbecher Childrens Hospital - Portland, Oregon 2,538.3 miles Jennifer Williamson 503-494-3639 willjenn@ohsu.edu
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles Renna Killen RN 323-361-2217 rkillen@chla.usc.edu
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles Renna Killen RN 323-361-2217 rkillen@chla.usc.edu
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles Renna Killen RN 323-361-2217 rkillen@chla.usc.edu
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles Renna Killen RN 323-361-2217 rkillen@chla.usc.edu
Stanford University Division of Pediatric Stem Cell Transplant Regenerative Medicine - Palo Alto, California 2,694.6 miles Prianka Kumar 650-721-8637 None
Stanford University Division of Pediatric Stem Cell Transplant Regenerative Medicine - Palo Alto, California 2,694.6 miles Prianka Kumar 650-721-8637 None
Stanford University Division of Pediatric Stem Cell Transplant Regenerative Medicine - Palo Alto, California 2,694.6 miles Prianka Kumar 650-721-8637 None
Stanford University Division of Pediatric Stem Cell Transplant Regenerative Medicine - Palo Alto, California 2,694.6 miles Prianka Kumar 650-721-8637 None

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