NCT02065869

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRαβ+ T Cells in Paediatric Patients Affected by Haematological Disorders

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 187 (actual)
• Sponsor: Bellicum Pharmaceuticals · Industry
• Sex: All
• Age: 1 Month – 18 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

This is a Phase I/II study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT) in pediatric patients. The purpose of this clinical trial is to determine whether BPX-501 infusion can enhance immune reconstitution in those patients with hematologic disorders, with the potential for reducing the severity and duration severe acute graft versus host disease (GvHD). The trial will also evaluate the treatment of GvHD by the infusion of dimerizer drug (AP1903/rimiducid) in those subjects who present with GVHD who progress or do not respond to standard of care treatment.

Conditions

• Acute Lymphoblastic Leukemia
• Leukemia, Acute Myeloid (AML), Child
• Lymphoma, Non-Hodgkin
• Myelodysplastic Syndrome
• Primary Immunodeficiency
• Anemia, Aplastic
• Osteopetrosis
• Hemoglobinopathies
• Cytopenia
• Fanconi Anemia
• Diamond Blackfan Anemia
• Thalassemia
• Anemia, Sickle Cell

Interventions

Type	Name	Description
BIOLOGICAL	BPX-501 T cells	1x10E6 cells/kg infused on Day 0
DRUG	Rimiducid	0.4mg/kg administered IV to treat GVHD

Timeline

• Start date: 2014-04-01
• Primary completion: 2020-06-01
• Completion: 2021-09-07
• First posted: 2014-02-19
• Last updated: 2023-09-29
• Results posted: 2023-01-04

Locations

4 sites across 2 countries: Italy, United Kingdom

Regulatory

• FDA-regulated drug study