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

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ClinicalTrials.gov Identifier: NCT02231710
Recruitment Status : Recruiting
First Posted : September 4, 2014
Last Update Posted : December 7, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
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 Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 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
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2019


Arms and Interventions

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


Outcome Measures

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


Eligibility Criteria

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
Criteria

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
Contacts and Locations

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


Locations
United States, Washington
Fred Hutchinson Cancer ResearchCenter Recruiting
Seattle, Washington, United States, 98109
Contact: Michele Bouvier    206-667-6993      
Principal Investigator: Lauri Burroughs, M.D.         
Principal Investigator: Ann Woolfrey, M.D.         
Sponsors and Collaborators
Bellicum Pharmaceuticals
Investigators
Principal Investigator: Lauri Burroughs, M.D. Fred Hutchinson Cancer Research Center
More Information

Responsible Party: Bellicum Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02231710     History of Changes
Other Study ID Numbers: BP-003
First Posted: September 4, 2014    Key Record Dates
Last Update Posted: December 7, 2017
Last Verified: December 2017
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
Thalassemia
Mucopolysaccharidosis
Sphingolipidoses

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