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

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.

Conditions

• Crohn Disease

Interventions

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

Sponsors & Collaborators

• Stanford University

  lead OTHER

Principal Investigators

• Jessie Alexander, MD · Stanford University

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

2 Years

Max Age

30 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-07-31

Primary Completion

2037-07-31

Completion

2040-07-31

FDA Device

Yes

Countries

• United States

Study Locations