Stem Cell Transplant for Crohn's Disease

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Stanford University

Disqualifiers: Ulcerative colitis, Severe cardiovascular, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

14 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Research Team

Jessie Alexander, MD

Principal Investigator

Stanford University

Eligibility Criteria

This trial is for Crohn's disease patients aged 2-30 with specific genetic causes or severe cases not responding to treatment. Participants must have a minimum Lansky/Karnofsky score of 50, agree to birth control if applicable, and have a donor match at certain genetic points. It excludes those with treatable extraintestinal symptoms, certain mutations, active HLH, organ dysfunction, HIV/HBV/HCV infections, prior radiation therapy or HSCT, recent cancer (with exceptions), pregnancy/breastfeeding, or inability to consent.

Inclusion Criteria

I am between 2 and 30 years old.

My donor and I are a genetic match at specific points.

My IBD is caused by a single gene or my Crohn's disease doesn't respond to treatment.

Exclusion Criteria

HIV-infected patients or patients with chronic HBV or HCV infection

I have ulcerative colitis.

I have been diagnosed with active HLH.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Regimen

Participants receive a conditioning regimen prior to hematopoietic stem cell transplantation (HSCT)

1-2 weeks

Hematopoietic Stem Cell Transplantation (HSCT)

Participants undergo TCRαβ+ T-cell/CD19+ B-cell depleted HSCT to induce immune tolerance

Immediate post-conditioning

Post-Transplant Monitoring

Participants are monitored for myeloid engraftment and acute graft vs host disease (GVHD)

6 months

Regular visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment, including remission of Crohn disease and immune function

2 years

Treatment Details

Interventions

• Allogeneic HSCT

Trial Overview The study tests whether alpha beta T-cell depleted HSCT from partially matched donors can replace the immune system in early-onset Crohn's disease. The process involves using drugs like Palifermin and Clofarabine along with TCRα/β+ T-cell/CD19+ B-cell depletion kits and other treatments such as ATG and Rituximab.

Participant Groups

Group I: Cohort 2a Experimental Treatment 8 Interventions

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.

Group II: Cohort 1b: Safety Lead-In Experimental Treatment 8 Interventions

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.

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+