NCT03219359 · Aaron Etra
Autologous Stem Cell Transplant for Crohn's Disease
What this study is about
Crohn's Disease (CD) is an inflammatory bowel disease. It can lead to significant complications and discomfort in the stomach and intestines. Crohn's disease is a debilitating, incurable disease of immune cells; it affects almost 1 million people in the United States.
View original scientific description
Crohn's Disease (CD) is an inflammatory bowel disease. It can lead to significant complications and discomfort in the stomach and intestines. Crohn's disease is a debilitating, incurable disease of immune cells; it affects almost 1 million people in the United States. CD is characterized by inflammation of the stomach and intestine as well as organs outside of the intestines such as the skin, eyes, and joints. Current therapies to treat CD aim to suppress the patient's immune cells but these therapies become ineffective for the majority of patients and lead to complications including the requirement for surgical bowel resection, impaired quality of life, and lifelong disability. Hematopoietic stem cell transplantation (HCT) is a procedure used to treat a number of medical conditions including Crohn's disease. To improve success of HCT in CD doctors considered combining transplant with other drugs to improve the chances of achieving remission and also maintaining the remission. The Investigators' plan in this study is to incorporate the drug Vedolizumab after transplant to test if this drug will improve remission and make patients healthier. Patients may qualify to take part in this research study because Crohn's disease is active, because surgery is not a treatment option and because there is evidence that the disease has failed to respond to treatments for Crohn's disease including the following: * corticosteroids * azathioprine, 6-mercaptopurine, methotrexate * Anti-TNFα (infliximab, adalimumab, certolizumab, golimumab) * Anti-integrin agents (natalizumab, Vedolizumab) If patients meet entry criteria will undergo a baseline endoscopy, colonoscopy and MR or CT enterography. If documentation of active mucosal disease patients will then be tapered off of current medications and undergo stem cell mobilization. Mobilization will involve low dose chemotherapy, growth factors and require 1-2 week hospitalization. Patients will then undergo stem cell transplant which will involve high dose chemotherapy and require a 2-4 week hospitalization. After restoration of the immune system patients will be placed on vedolizumab per standard dosing (0,2,6 then 8 every weeks) for a total of 8 doses. Patients will have monthly study visits and a repeat colonoscopy and MR/CT scan at 6 months.
Interventions
PROCEDURE
Autologous stem cell transplant
Hematopoietic stem cell transplantation
DRUG
Cyclophosphamide
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
DRUG
Cyclophosphamide
Day -6 to -3: Cyclophosphamide 50 mg/kg/day (200 mg/kg total dose)
DRUG
Thymoglobulin
Day -3 to -1: 2.5 mg/kg/day (7.5 mg/kg total dose)
DRUG
Methylprednisolone
Day -3 to -1: 1 gram prior to each ATG dose
DRUG
Vedolizumab
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)
Primary outcome measures
Change in Crohn's Disease Activity Index (CDAI)
Time frame: baseline and 1 year post transplant
The proportion of patients in clinical remission, defined as a CDAI \< 150, one year after autologous HCT from 45% to 65% as compared to baseline.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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:
- corticosteroids
- azathioprine,
- 6-mercaptopurine, methotrexate
- Anti-TNFα (infliximab, adalimumab, certolizumab, golimumab)
- Anti-integrin agents (natalizumab, vedolizumab)
- 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
- 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.
Where
- New York, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Nov 5, 2025 · Source of record for eligibility and locations