NCT02860559

Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Taiga Biotechnologies, Inc. · Industry
• Sex: All
• Age: 1 Month – 4 Years
• Healthy volunteers: Not accepted

Summary

This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID). The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability of the donor cells to make blood and immune cells after transplant. Exposure of the donor cells to this protein does not modify the genes in the cells in any way. This study has two goals. The first goal is to find out if transplant with TBX-1400 is safe. The second goal is to find out what effects TBX-1400 stem cells have on time to engraftment in pediatric subjects with SCID. The study hypothesis is that TBX-1400 cells will shorten the time to immune reconstitution after transplant.

Conditions

• Severe Combined Immunodeficiency

Interventions

Type	Name	Description
BIOLOGICAL	TBX-1400	Hematopoietic stem cells transplantation

Timeline

• Start date: 2021-08-01
• Primary completion: 2024-02-01
• Completion: 2024-03-01
• First posted: 2016-08-09
• Last updated: 2020-10-08

Locations

2 sites across 1 country: Israel

Regulatory

• FDA-regulated drug study