Autologous Stem Cell Transplant for Crohn's Disease
Phase 2 · Testing Autologous stem cell transplant, Cyclophosphamide and Thymoglobulin · 1 site · Aaron Etra
Official record & contacts on ClinicalTrials.gov ↗
In plain English
What this trial is testing
This study is testing Autologous stem cell transplant (a procedure), Cyclophosphamide (a drug), Cyclophosphamide (a drug) and Thymoglobulin (a drug) as a possible treatment. Condition(s) listed: Crohn Disease.
It is open-label: participants know what they are receiving.
Phase 2 studies test whether a treatment shows signs of working, and keep checking safety, in a larger group of people with the condition.
Who can join
- Ages 18 and older
- Any sex
Some of the main requirements, copied from the record:
- Diagnosis of Crohn's disease by standard criteria
- Active disease based on clinical symptoms, defined as CDAI >250. In patients with an ostomy, the number of liquid stools score in the CDAI will be replaced by the number of times that the ostomy bag is emptied daily.
- Active disease based on endoscopic evaluation, defined as SES-CD score > 3 in at least one bowel segment
Plus 9 more requirement(s). There are also 10 reasons someone might not be able to join (exclusion criteria). Only the trial team can confirm whether you qualify.
What's involved
- About 50 participants are expected to take part.
- Start date: 2018-02-22.
- Main results expected around 2028-10 (estimate from the record).
- 1 study site listed (1 marked recruiting) in United States.
Main thing being measured
- Change in Crohn's Disease Activity Index (CDAI) (baseline and 1 year post transplant)
In the researchers’ words
Crohn's Disease (CD) is an inflammatory bowel disease. It can lead to significant complications and discomfort in the stomach and intestines.
This is a short automatic summary built from the official record. A fuller plain-English card is coming; until then, please read the official record.
Questions to ask your doctor and the trial team
- Am I likely to qualify, given my health history?
- What would I have to do, and how often would I need to visit?
- Could I get a placebo? What happens to my current treatment?
- What are the possible risks and benefits?
- Are travel or other costs covered?
- What happens after the trial ends?
Official details
| Status | Recruiting |
|---|---|
| Phase | Phase 2 |
| Study type | Interventional |
| Conditions | Crohn Disease |
| Lead sponsor | Aaron Etra |
| Enrollment | 50 (estimated) |
| Start date | 2018-02-22 |
| Primary completion (est.) | 2028-10 |
| First posted | 2017-07-17 |
| Last updated | 2026-07-24 |
| Official title | Maintenance in Autologous Stem Cell Transplant for Crohn's Disease (MASCT - CD) |
| ClinicalTrials.gov ID | NCT03219359 |
Interventions
- Autologous stem cell transplant (Procedure)
Hematopoietic stem cell transplantation - Cyclophosphamide (Drug)
Days 1 and 2: Cyclophosphamide 2gm/m2/day x 2 days (total dose 4gm/m2) Day 3 until leukapheresis: G-CSF 10μg/kg/day to CD34+ >20x104/ml then leukapheresis daily to collection goal - Cyclophosphamide (Drug)
Day -6 to -3: Cyclophosphamide 50 mg/kg/day (200 mg/kg total dose) - Thymoglobulin (Drug)
Day -3 to -1: 2.5 mg/kg/day (7.5 mg/kg total dose) - Methylprednisolone (Drug)
Day -3 to -1: 1 gram prior to each ATG dose - Vedolizumab (Drug)
Starting first day after discharge from transplant admission, then 2 weeks after 1st infusion, 4 weeks after 2nd infusion, followed by every 8 weeks for 52 weeks (8 doses)
Full eligibility criteria (from the record)
Inclusion Criteria: * Diagnosis of Crohn's disease by standard criteria * Active disease based on clinical symptoms, defined as CDAI >250. In patients with an ostomy, the number of liquid stools score in the CDAI will be replaced by the number of times that the ostomy bag is emptied daily. * Active disease based on endoscopic evaluation, defined as SES-CD score > 3 in at least one bowel segment * Failure to respond to (or intolerant/adverse reaction to or declines) a member of each of the class of drugs listed below: 1. corticosteroids 2. azathioprine, 3. 6-mercaptopurine, methotrexate 4. Anti-TNFα (infliximab, adalimumab, certolizumab, golimumab) 5. Anti-integrin agents (natalizumab, vedolizumab) 6. Ustekinumab * Failure to respond refers to ongoing objective inflammation with symptoms and, as is traditional, is defined by the gastroenterologist evaluating the patient. * No surgical therapeutic option secondary to risk of short bowel syndrome or patient refusal Exclusion Criteria: * History of significant toxicity to any medications used in trial (cyclophosphamide, thymoglobulin, vedolizumab) * Pregnant or breastfeeding * Age <18 * Karnofsky Performance Score <60 * Patients who have an uncontrolled infection (presumed or documented) despite appropriate therapy for at least one month * Patients with symptomatic coronary artery disease or uncontrolled congestive heart failure. * HIV infected * Ejection fraction <30% or requiring supplemental continuous oxygen. * DLCO <35% or requiring supplementary oxygen. * Patients for whom an insufficient number of stem cells (<2 X 10^6/kg) have been collected.
Locations (1)
| Site | City | Country | Site status |
|---|---|---|---|
| Icahn School of Medicine at Mount Sinai | New York, New York | United States | Recruiting |
For contacts at each site, use the official record.
Information only, not medical advice. Talk to your doctor and the trial team before making any decision about a clinical trial.