Original Query: diamond blackfan anemia
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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. Identifier: NCT02231710
Recruitment Status : Terminated (due to lack of enrollment and changes to the sponsor development portfolio)
First Posted : September 4, 2014
Last Update Posted : January 23, 2018
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 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 AP1903 Phase 1

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 an AP1903 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 AP1903 which can facilitate engraftment and prevent the occurrence of GVHD.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
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
Study Start Date : February 2015
Primary Completion Date : January 15, 2018
Study Completion Date : January 15, 2018

Arm Intervention/treatment
Experimental: BPX-501 and AP1903
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by AP1903 infusion on day 7
Biological: BPX-501 and AP1903
Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by AP1903 infusion on day 7

Primary Outcome Measures :
  1. GVHD [ Time Frame: day 100 ]
    The incidence and Grade of acute GVHD will be determined

Secondary Outcome Measures :
  1. Transplant related mortality [ Time Frame: 6 months ]
    Incidence of transplant related mortality

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. Males or females
  3. Age < 55 years old and > 4 months
  4. Diagnosis of a nonmalignant disorder considered treatable by HCT.
  5. Lack of suitable conventional donor

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

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 identifier (NCT number): NCT02231710

United States, Washington
Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, United States, 98109
Sponsors and Collaborators
Bellicum Pharmaceuticals
Principal Investigator: Lauri Burroughs, M.D. Fred Hutchinson Cancer Research Center

Responsible Party: Bellicum Pharmaceuticals Identifier: NCT02231710     History of Changes
Other Study ID Numbers: BP-003
First Posted: September 4, 2014    Key Record Dates
Last Update Posted: January 23, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Bellicum Pharmaceuticals:
Shwachman Diamond Syndrome
Diamond Blackfan Anemia
Fanconi Anemia
Severe Combined Immune Deficiency
Congenital T-cell Defect
Congenital T-cell Deficiency
Chronic Granulomatous Disease
Dyskeratosis Congenita
Sickle Cell Disease

Additional relevant MeSH terms:
Anemia, Aplastic
Anemia, Hemolytic
Immunologic Deficiency Syndromes
Metabolic Diseases
Lymphohistiocytosis, Hemophagocytic
Hemoglobinuria, Paroxysmal
Pathologic Processes
Immune System Diseases
Hematologic Diseases
Genetic Diseases, Inborn
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases
Bone Marrow Diseases
Myelodysplastic Syndromes