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Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02231710
Recruitment Status : Active, not recruiting
First Posted : September 4, 2014
Last Update Posted : July 12, 2022
Information provided by (Responsible Party):
Bellicum Pharmaceuticals

Brief Summary:
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.

Condition or disease Intervention/treatment Phase
Primary Immune Deficiency Disorders Hemophagocytic Lymphohistiocytosis Inherited Bone Marrow Failure Syndrome Hemoglobinopathies Metabolic Disorders Biological: BPX-501 and Rimiducid Phase 1

Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Detailed Description:
This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg followed by a Rimiducid infusion on day 7 after a partially mismatched, related, T cell-depleted hematopoietic cell transplantation (HCT) in patients with non-malignant diseases. The purpose of this clinical trial is to determine the dose of BPX 501 T cell infusion with subsequent planned infusion of Rimiducid which can facilitate engraftment and prevent the occurrence of GVHD.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Study Evaluating BPX-501 T Cells and AP1903 for Prevention of Graft Versus Host Disease (GVHD) After Haploidentical, Related, T Cell-Depleted Hematopoietic Cell Transplantation for Non-Malignant Diseases
Actual Study Start Date : February 2015
Actual Primary Completion Date : July 2017
Estimated Study Completion Date : July 2030

Arm Intervention/treatment
Experimental: BPX-501 and Rimiducid
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7
Biological: BPX-501 and Rimiducid
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7

Primary Outcome Measures :
  1. Adverse Events [ Time Frame: Month 24 ]
    Determine the safety of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled AP1903 infusion

  2. Engraftment [ Time Frame: Day 28 ]
    Determine the engraftment rate (defined as >50% donor CD3 chimerism) on day 28 after HCT with HLA-haploidentical CD34+ selected PBSC grafts per dose cohort of BPX 501 T cells followed by Rimiducid infusion on Day 7.

Secondary Outcome Measures :
  1. GvHD [ Time Frame: Month 24 ]
    To determine the incidence and severity of acute and chronic GVHD

  2. Immune Reconstitution [ Time Frame: Month 24 ]
    Measure immune reconstitution

  3. Infection rates [ Time Frame: Day 200 ]
    Determine the risk for severe infections

  4. Graft rejection [ Time Frame: Month 24 ]
    Incidence of graft rejection

  5. Rimiducid Activity [ Time Frame: Month 24 ]
    Time to resolution of acute and chronic GvHD following administration of Rimiducid

  6. High grade toxicity [ Time Frame: Month 24 ]
    Rate of high grade toxicity

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Months to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patient must meet eligibility criteria for allogeneic transplantation
  2. Lack of suitable conventional donor (10/10 allele matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
  3. Males or females
  4. Age < 55 years old and > 4 months
  5. Diagnosis of a nonmalignant disorder considered treatable by HCT.
  6. HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, DRBl, and DQB1 loci.

    i. A minimum match of 5/10 is required. ii. The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following

  7. If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment.
  8. Informed consent signed by patient (if ≥18 years old) or parent/guardian (if <18 years old).
  9. Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:

    1. Any patient with Fanconi anemia and bone marrow failure involving 2 of the following 3 lineages: granulocyte count <0.5 x 109/L, platelet count <20 x 109/L, or hemoglobin <8 g/dL.
    2. Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure
    3. Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage.

Exclusion Criteria:

  1. Serious organ dysfunction
  2. Pregnant or breast-feeding
  3. Evidence of HIV infection
  4. Bovine product allergy
  5. Patients with an active infectious disease
  6. Patients with Fanconi anemia with AML/MDS.

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): NCT02231710

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United States, Washington
Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Bellicum Pharmaceuticals
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Study Director: Bellicum Pharmaceuticals Senior Director Clinical Development
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Responsible Party: Bellicum Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02231710    
Other Study ID Numbers: BP-003
First Posted: September 4, 2014    Key Record Dates
Last Update Posted: July 12, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bellicum Pharmaceuticals:
Severe Combined Immune Deficiency
Congenital T-cell Defect
Congenital T-cell Deficiency
Chronic Granulomatous Disease
Shwachman Diamond Syndrome
Diamond Blackfan Anemia
Dyskeratosis Congenita
Fanconi Anemia
Sickle Cell Disease
Additional relevant MeSH terms:
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Bone Marrow Failure Disorders
Lymphohistiocytosis, Hemophagocytic
Congenital Bone Marrow Failure Syndromes
Primary Immunodeficiency Diseases
Metabolic Diseases
Immunologic Deficiency Syndromes
Pathologic Processes
Immune System Diseases
Bone Marrow Diseases
Hematologic Diseases
Genetic Diseases, Inborn
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases
Infant, Newborn, Diseases