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

NCT02231710 | Terminated Phase 1 Results FDA Drug

• 1 other identifier: BP-003
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary Immune Deficiency Disorders; Hemophagocytic Lymphohistiocytosis; Inherited Bone Marrow Failure Syndrome; Hemoglobinopathies; Metabolic Disorders

Keywords

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; Thalassemia; Mucopolysaccharidosis; Sphingolipidoses

Outcome Measures

Primary Outcomes (2)

• Adverse Events

  To determine the safety (as defined by non-responsive Grade III-IV GVHD to rimiducid) of HCT with HLA-haploidentical CD34+ selected peripheral blood stem cell (PBSC) grafts and BPX 501 T cells followed by scheduled rimiducid infusion on Day 7. this outcome measure is reported as number of patients who experienced the AE of Grade III-IV GVHD that was not non-responsive to rimiducid (safety switch) administration.

  24 months

• Engraftment

  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. NOTE: only one patient was enrolled who received the dose of 5x 10\^6cell/kg dose of BPX-501

  Day 28

Secondary Outcomes (6)

• GvHD

  Month 24

• Immune Reconstitution

  Month 24

• Infection Rates

  Day 200

• Graft Rejection

  Month 24

• Rimiducid Activity

  Month 24

Study Arms (1)

BPX-501 and Rimiducid

EXPERIMENTAL

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

Interventions

BPX-501 and Rimiducid BIOLOGICAL

Single administration of BPX-501 T cells post partially-mismatched, related T cell depleted HCT followed by Rimiducid infusion on day 7

BPX-501 and Rimiducid

Eligibility Criteria

• Age: 4 Months - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient must meet eligibility criteria for allogeneic transplantation
• 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
• Males or females
• Age \< 55 years old and \> 4 months
• Diagnosis of a nonmalignant disorder considered treatable by HCT.
• 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
• If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment.
• Informed consent signed by patient (if ≥18 years old) or parent/guardian (if \<18 years old).
• Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:
• 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.
• Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure
• Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage.

You may not qualify if:

• Serious organ dysfunction
• Pregnant or breast-feeding
• Evidence of HIV infection
• Bovine product allergy
• Patients with an active infectious disease
• Patients with Fanconi anemia with AML/MDS.

Sponsors & Collaborators

• Bellicum Pharmaceuticals: lead

Study Sites (1)

Fred Hutchinson Cancer ResearchCenter

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases; Lymphohistiocytosis, Hemophagocytic; Congenital Bone Marrow Failure Syndromes; Hemoglobinopathies; Metabolic Diseases; Severe Combined Immunodeficiency; Granulomatous Disease, Chronic; Shwachman-Diamond Syndrome; Anemia, Diamond-Blackfan; Dyskeratosis Congenita; Fanconi Anemia; Anemia, Sickle Cell; Thalassemia; Mucopolysaccharidoses; Sphingolipidoses

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases; Hematologic Diseases; Infant, Newborn, Diseases; Nutritional and Metabolic Diseases; DNA Repair-Deficiency Disorders; Phagocyte Bactericidal Dysfunction; Leukocyte Disorders; Genetic Diseases, X-Linked; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Exocrine Pancreatic Insufficiency; Pancreatic Diseases; Digestive System Diseases; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Lipomatosis; Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Red-Cell Aplasia, Pure; Skin Abnormalities; Congenital Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Lipidoses

Results Point of Contact

• Title: Rivogenlecleucel Study Team
• Organization: Bellicum Pharmaceuticals

clinicaltrials@bellicum.com

(832) 384-1100

Study Officials

• Bellicum Pharmaceuticals Senior Director

  Clinical Development

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: August 28, 2014
• First Posted: September 4, 2014
• Study Start: February 1, 2015
• Primary Completion: September 1, 2015
• Study Completion: January 1, 2018
• Last Updated: March 25, 2024
• Results First Posted: March 25, 2024

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)