NCT06986382

Phase 1b/2a Trial of Allogeneic HSCT From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset Medically Refractory Crohn Disease

Phase 1b/2a Trial of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) From an HLA-partially Matched Related or Unrelated Donor After TCRab+ T-cell/CD19+ B-cell Depletion for Patients With Monogenic and/or Early-onset (EO), Medically Refractory (MR) Crohn Disease (CD)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: Stanford University · Academic / Other
• Sex: All
• Age: 2 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

This research study is investigating whether alpha beta T-cell depleted hematopoietic stem cell transplant (HSCT) can be an immune system replacement for Crohn disease patients and whether this is safe and effective for patients with early onset, medically refractory Crohn disease.

Detailed description

This is a single center, non-randomized, non-controlled open-label Phase 1b/2a trial to study the safety and efficacy of performing TCRαβ+ T-cell/CD19+ B-cell depleted (TCRαβ-depleted) HSCT to induce immune tolerance in patients with monogenic or early onset, medically refractory Crohn disease to re-establish normal immune-intestinal homeostasis.

Conditions

• Crohn Disease

Interventions

Type	Name	Description
DEVICE	CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System	CliniMACS® TCRαβ-Biotin and CD19 Systems will be used to create the mobilized peripheral blood stem cells (PBSC) from allogeneic donors depleted of TCRαβ+ T cells and CD19+ B cells to be infused into the patient for the HSCT. The target dose for the number of CD34+ HSC infused is \> 10 x 10\^6 cells/Kg recipient weight. The minimum dose is 2 x 10\^6 cells/Kg. There is no upper limit to the dose of CD34+ HSC infused as long as no more than 1 x 10\^5 TCRαβ+ T-cells/Kg are infused. The target dose of TCRαβ+ T cells/Kg is \< 0.50 x 10\^5.
DRUG	Prednisone/Methylprenisolone	Administered as part of the HSCT conditioning regimen
DRUG	Palifermin	Administered as part of the HSCT conditioning regimen
DRUG	ATG	Administered as part of the HSCT conditioning regimen
DRUG	Clofarabine	Administered as part of the HSCT conditioning regimen
DRUG	Melphalan	Administered as part of the HSCT conditioning regimen
RADIATION	Total Body Irradiation	200 cGy, administered as part of the HSCT conditioning regimen
DRUG	Rituximab	Administered as part of the HSCT conditioning regimen

Timeline

• Start date: 2025-07-01
• Primary completion: 2037-07-01
• Completion: 2040-07-01
• First posted: 2025-05-23
• Last updated: 2025-05-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study