Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT03219359

Autologous Stem Cell Transplant for Crohn's Disease

Led by Aaron Etra · Updated on 2025-11-05

50

Participants Needed

1

Research Sites

553 weeks

Total Duration

On this page

AI-Summary

What this Trial 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. 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

Official Title

Autologous Stem Cell Transplant for Crohn's Disease

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of Crohn's disease by standard criteria
  • Active disease with Crohn's Disease Activity Index (CDAI) greater than 250
  • Active disease confirmed by endoscopy with SES-CD score above 3 in at least one bowel segment
  • Failure, intolerance, adverse reaction, or refusal of treatment with corticosteroids
  • Failure, intolerance, adverse reaction, or refusal of treatment with azathioprine
  • Failure, intolerance, adverse reaction, or refusal of treatment with 6-mercaptopurine or methotrexate
  • Failure, intolerance, adverse reaction, or refusal of treatment with Anti-TNFb1 drugs (infliximab, adalimumab, certolizumab, golimumab)
  • Failure, intolerance, adverse reaction, or refusal of treatment with Anti-integrin agents (natalizumab, vedolizumab)
  • Failure, intolerance, adverse reaction, or refusal of treatment with Ustekinumab
  • No surgical treatment option due to risk of short bowel syndrome or patient refusal
Not Eligible

You will not qualify if you...

  • History of significant toxicity to cyclophosphamide, thymoglobulin, or vedolizumab
  • Pregnant or breastfeeding
  • Younger than 18 years old
  • Karnofsky Performance Score less than 60
  • Uncontrolled infection despite appropriate therapy for at least one month
  • Symptomatic coronary artery disease or uncontrolled congestive heart failure
  • HIV infection
  • Ejection fraction less than 30% or requiring continuous supplemental oxygen
  • DLCO less than 35% or requiring supplementary oxygen
  • Insufficient number of stem cells collected (less than 2 x 10^6/kg)

AI-Screening

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Trial Site Locations

Total: 1 location

1

Icahn School of Medicine at Mount Sinai

New York, New York, United States, 10029

Actively Recruiting

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Research Team

L

Louis Cohen, MD

CONTACT

A

Aaron Etra, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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