RecruitingPhase 2NCT03219359

Autologous Stem Cell Transplant for Crohn's Disease

Maintenance in Autologous Stem Cell Transplant for Crohn's Disease (MASCT - CD)


Sponsor

Aaron Etra

Enrollment

50 participants

Start Date

Feb 22, 2018

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This trial is testing whether a stem cell transplant using the patient's own stem cells (autologous transplant) can help treat severe Crohn's disease that has not responded to multiple medications. **You may be eligible if...** - You have a confirmed diagnosis of Crohn's disease - Your disease is currently active based on symptoms (high disease activity score) - Your disease is confirmed as active on a colonoscopy - You have already tried and failed (or cannot tolerate) multiple types of Crohn's medications, including biologic drugs **You may NOT be eligible if...** - Your Crohn's disease is currently in remission - You have not yet tried the required range of medications - You have other conditions that make a stem cell transplant unsafe (such as certain infections or organ problems) Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

PROCEDUREAutologous stem cell transplant

Hematopoietic stem cell transplantation

DRUGCyclophosphamide

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

DRUGCyclophosphamide

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

DRUGThymoglobulin

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

DRUGMethylprednisolone

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

DRUGVedolizumab

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)


Locations(1)

Icahn School of Medicine at Mount Sinai

New York, New York, United States

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NCT03219359


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