NCT03047746

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Unrelated And Partially Matched Related Donor Peripheral Blood Stem Cell Transplantation (PSCT) With TCR αβ + T Cell And B Cell Depletion For Patients With Acquired And Inherited Bone Marrow Failure

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

Summary

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.

Detailed description

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes. Previously established, disease-specific transplant preparative regimens will be administered based on the specific underlying BMF condition. Mobilized PBSC will be processed using the CliniMACS system for TCR alpha/beta+ T cell depletion plus cluster of differentiation 19+ (CD19+) B cell depletion. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one year overall and event-free survival.

Conditions

• Acquired Aplastic Anemia
• Paroxysmal Nocturnal Hemoglobinuria
• Inherited Bone Marrow Failure Syndromes

Interventions

Type	Name	Description
DEVICE	CliniMACs	Peripheral blood stem cells from closely matched unrelated or partially matched related donors will be processed using the CliniMACS device to remove TCRalpha/beta T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique

Timeline

• Start date: 2017-02-01
• Primary completion: 2028-05-01
• Completion: 2029-05-01
• First posted: 2017-02-09
• Last updated: 2025-07-16

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study