Stem Cell Transplantation for Crohn's Disease in Young People
This study is exploring an experimental treatment for severe Crohn's disease (CD) in young people aged 13-28 who haven't responded to other treatments. It involves removing overactive immune cells (immunoablation) and then replacing them with your own blood stem cells. This process uses medications like Cyclophosphamide and Filgrastim to prepare your body and help collect your stem cells. The goal is to "reset" your immune system to how it was before you developed Crohn's. Researchers will measure changes in mucosal healing (how well your gut lining heals), inflammation (using blood tests like SED rate), and a marker in your stool (fecal calprotectin) to see if the treatment is successful. The study aims to enroll 15 participants, but its current status is unclear.
- Study design
- This is an open-label, non-randomized study with 15 planned participants. It is not blinded, meaning both you and the researchers will know what treatment you are receiving.
- What's involved
- You will undergo procedures like apheresis catheter placement and leukapheresis to collect stem cells. You will also receive intravenous infusions of medications like Cyclophosphamide, Mesna, and Filgrastim.
- Compensation
- Not stated in the trial record.
- Follow-up
- Researchers will follow up on your mucosal healing at 6 and 12 months. They will also track your SED rate and fecal calprotectin for up to 24 months after the stem cell transplant.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Stem Cell Transplantation in Crohn's Disease
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- David Ziring, MD · PRINCIPAL_INVESTIGATOR · Cedars-Sinai Medical Center
Who to contact
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What this trial measures
- Change in mucosal healingChange from pre-HSCT (baseline) to 6 months and 12 months post HSCT
Change mucosal healing as determined by the simple endoscopic score for crohn's disease (SES-CD). The SES-CD assesses the size of mucosal ulcers, the ulcerated surface, the endoscopic extension and the presence of stenosis. Each are measured on a scale of 0-3 and are summed to create a total score. For total score, 0-2 indicates remission, 3-6 indicates mild endoscopic activity, 7-15 indicates moderate endoscopic activity, and \> 15 indicates severe endoscopic activity.
- Change in erythrocyte sedimentation rate (SED rate)Change from pre-HSCT (baseline) to 2, 4, 6, 12, and 24 months post HSCT
Change in SED rate (mm/hour)
- Change in fecal calprotectin concentrationChange from pre-HSCT (baseline) to 2, 4, 6, 12, and 24 months post HSCT
Change in fecal calprotectin concentration
- Change in C reactive protein (CRP)Change from pre-HSCT (baseline) to 2, 4, 6, 12, and 24 months post HSCT
Change in C reactive protein (CRP)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]Up to 24 months post HSCT
Number of treatment-emergent adverse events (including death (transplant related mortality, TRM) and severe toxicity (≥ grade 3 toxicity; NCI Toxicity Criteria version 4.0)
- Incidence of HSCT Related ComplicationsUp to 24 months post HSCT
The incidence of HSCT related complications, i.e. viral reactivations (CMV, Adenovirus, EBV, BK virus) or fungal infections.
- Change in clinical measures of sustained remissionUp to 24 months post HSCT
Change in CDAI score (Crohn's Disease Activity Index). The CDAI measure the signs, symptoms, and history of Crohn's Disease based on the past 7 days. The index measures abdominal pain, stools per day, general wellbeing, HCT, ESR, Albumin, height, weight, abdominal exam, perirectal disease, and extra-intestinal manifestations each scaled between 0-10. The sum of these measures creates a total score between 0-100 with the higher score representative of more disease activity.