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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Stanford University
Study ID
NCT06986382
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 30 Years
Healthy Volunteers
Not accepted

Interventions

  • CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System — DEVICE
    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.
  • Prednisone/Methylprenisolone — DRUG
    Administered as part of the HSCT conditioning regimen
  • Palifermin — DRUG
    Administered as part of the HSCT conditioning regimen
  • ATG — DRUG
    Administered as part of the HSCT conditioning regimen
  • Clofarabine — DRUG
    Administered as part of the HSCT conditioning regimen
  • Melphalan — DRUG
    Administered as part of the HSCT conditioning regimen
  • Total Body Irradiation — RADIATION
    200 cGy, administered as part of the HSCT conditioning regimen
  • Rituximab — DRUG
    Administered as part of the HSCT conditioning regimen

Study Details

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.

Key Dates

Start date
Jul 31, 2025
Status verified
May 2025
Primary completion
Jul 31, 2037
Completion
Jul 31, 2040

Study Design

Enrollment
14 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1b: Safety Lead-In
    An initial cohort of 4 patients will be enrolled as part of the initial Phase 1b safety run-in evaluation. Patients will undergo an αβdepleted hematopoietic stem cell transplant (HSCT) after receiving a conditioning regimen.
  • Experimental: Cohort 2a
    If the intervention is determined to be safe and non-futile, the study will continue to enroll 10 more patients under Phase 2a following the same treatment as Phase 1b.

Primary Outcome Measure

Number of patients with myeloid engraftment [ Time Frame: Day 42 post-HSCT ]

Locations (1)

FacilityCityStateZIPSite coordinators
Lucile Packard Children's HospitalPalo AltoCalifornia94305-

Find similar trials in Palo Alto, CA

By condition
Crohn's disease
By specialty
GastroenterologyInflammatory bowel diseaseAutoimmune disease
By research site
Lucile Packard Children's Hospital· Palo Alto, CA

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