Study of Stem Cell Transplant for Crohn's Disease
This study is looking at a special type of stem cell transplant (HSCT) for children and young adults (ages 2-30) with Crohn's disease that started early in life or is caused by a specific genetic problem, and hasn't responded to other treatments. The transplant uses stem cells from a partially matched donor, which are specially prepared using the CliniMACS® TCR α/β Reagent Kit and CliniMACS® CD19 System. You would also receive medicines like Prednisone/Methylprenisolone, Palifermin, ATG, and Clofarabine. Researchers want to see if this treatment is safe and effective in helping your body's immune system work better to control Crohn's disease. They will measure success by checking for new healthy blood cells at 42 days, serious transplant-related complications at 90 and 180 days, and if your Crohn's disease is in remission after 2 years. The study plans to enroll 14 participants.
- Study design
- This is a Phase 1b/2a study, meaning it's an early-stage trial looking at safety and effectiveness. It's a single-center, open-label study, so everyone knows what treatment is being given, and it will include 14 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Researchers will follow participants to check for new healthy blood cells at 42 days, serious transplant-related complications at 90 and 180 days, and if Crohn's disease is in remission after 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
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
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Jessie Alexander, MD · PRINCIPAL_INVESTIGATOR · Stanford University
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
What this trial measures
- Number of patients with myeloid engraftmentDay 42 post-HSCT
Cumulative incidence of donor myeloid engraftment by Day +42 post-HSCT. Myeloid engraftment is defined as ANC of \> 0.5 x 109/L for three consecutive laboratory values obtained on different days. Date of myeloid engraftment is the first date of the three lab values taken.
- Number of patients with Grade III-IV acute graft vs host disease (GVHD)Day 90 and 180 post-HSCT
Cumulative incidence of acute GvHD (graded as II-IV and III-IV using the Magic criteria) Cumulative incidence of acute GvHD (graded as II-IV and III-IV using the Magic criteria) Cumulative incidence of acute GvHD (graded as II-IV and III-IV using the Magic criteria) Cumulative incidence of acute GvHD (graded using the Magic criteria)
- Number of patients in remission of Crohn disease (Calprotectin)2 years post-HSCT
Cumulative incidence of patient who achieve remission defined by fecal calprotectin values less than 250 mg/g
- Number of patients in remission of Crohn disease (wPCDAI or CDAI)2 years post-HSCT
Cumulative incidence of remission demonstrated by weighted Pediatric Crohn's Disease Activity Index (wPCDAI) \<12.5 or Crohn's Disease Activity Index (CDAI) of \<150 if adult