Recruiting
Phase 2

Autologous Stem Cell Transplant

Sponsor:

Aaron Etra

Code:

NCT03219359

Conditions

Crohn Disease

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Autologous stem cell transplant

Cyclophosphamide

Cyclophosphamide

Thymoglobulin

Methylprednisolone

Study Details

Brief summary:

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.

Conditions

Crohn Disease

Study ID

NCT03219359

Start date

Feb 22, 2018

Status verified date

Jul, 2026

Completion date

Oct, 2028

Anticipated

Primary completion date

Oct, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

50 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Experimental

Hematopoietic Stem Cell Transplant followed by maintenance Vedolizumab

Interventions

Autologous stem cell transplant

Hematopoietic stem cell transplantation

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

Cyclophosphamide

Day -6 to -3: Cyclophosphamide 50 mg/kg/day (200 mg/kg total dose)

Thymoglobulin

Day -3 to -1: 2.5 mg/kg/day (7.5 mg/kg total dose)

Methylprednisolone

Day -3 to -1: 1 gram prior to each ATG dose

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 measure

  • Change in Crohn's Disease Activity Index (CDAI) [ Time Frame: baseline and 1 year post transplant ]

Central Contacts and Locations

Central contacts

Louis Cohen, MD

(212) 241-8100louis.cohen@mssm.edu

Aaron Etra, MD

aaron.etra@mountsinai.org

Locations

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Louis Cohen, MD

(212) 241-8100louis.cohen@mssm.edu

Principal Investigator:

Aaron Etra, MD

More Information

Sponsor

Aaron Etra

Last update posted

Jul 24, 2026

Last verified

Jul, 2026

Keywords

  • IBD
  • Inflammatory Bowel Diseases
  • Gastroenteritis
  • Crohn's disease
  • Colitis
  • Vedolizumab

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Aaron Etra on 2026-07-24.