Autologous Stem Cell Transplant for Crohn's Disease
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ClinicalTrials.gov Identifier: NCT03219359 |
Recruitment Status :
Recruiting
First Posted : July 17, 2017
Last Update Posted : August 25, 2020
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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.
Condition or disease | Intervention/treatment | Phase |
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Crohn Disease | Procedure: Autologous stem cell transplant Drug: Cyclophosphamide Drug: Thymoglobulin Drug: Methylprednisolone Drug: Vedolizumab | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 50 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Maintenance in Autologous Stem Cell Transplant for Crohn's Disease (MASCT - CD) |
Actual Study Start Date : | July 12, 2017 |
Estimated Primary Completion Date : | August 2022 |
Estimated Study Completion Date : | August 2022 |

Arm | Intervention/treatment |
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Experimental: Experimental
Hematopoietic Stem Cell Transplant followed by maintenance Vedolizumab
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Procedure: Autologous stem cell transplant
Hematopoietic stem cell transplantation
Other Name: Bone Marrow Transplant 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 -5 to -2: 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)
Other Name: ATG Drug: Methylprednisolone Day -3 to -1: 1mg/kg 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) |
- 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.
- Change in endoscopic activity indices [ Time Frame: baseline and 1 year post transplant ]The change in endoscopic activity indices (SES CD) following autologous HCT and vedolizumab maintenance as compared to baseline.The maximal score for the SES-CD is 56. Each of 5 segments of intestine (rectum, colon - descending, colon - ascending, colon - transverse, ileum) is scored from 0-3 on four parameters - ulceration, extent of ulceration, extent of disease and stenosis. A maximal score of 45 is possible for the first three parameters and 11 for stenosis as a score of 3 implies a stricture that cannot be traversed prohibiting evaluation of later segments. An SES score of 0-2 would be defined as mucosal healing as the presence of a single ulcer in one bowel segment would necessitate a minimal score of 3. A decrease of SES of 50% from baseline is felt to represent an endoscopic response.
- Change in burden of intestinal disease [ Time Frame: baseline and 1 year post transplant ]The change in overall burden of intestinal disease (Magnetic resonance enterography ((MRE)+SES-CD) following autologous HCT and vedolizumab maintenance as compared to baseline.
- Change in Inflammatory Bowel Disease Questionnaire (IBDQ) [ Time Frame: baseline and 1 year post transplant ]The change in quality of life indices as evaluated via the IBDQ following autologous HCT and vedolizumab maintenance as compared to baseline. An increase in the IBD-Q of 16-32 points is generally considered meaningful.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
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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
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03219359
Contact: Artur Shchukin | 212-241-0497 | artur.shchukin@mssm.edu |
United States, New York | |
Icahn School of Medicine at Mount Sinai | Recruiting |
New York, New York, United States, 10029 | |
Contact: Alex Abrahams 212-241-8233 alex.abrahams@mssm.edu | |
Principal Investigator: Amir Steinberg, MD | |
Principal Investigator: Louis Cohen, MD |
Principal Investigator: | Amir Steinberg, MD | Icahn School of Medicine at Mount Sinai | |
Principal Investigator: | Louis Cohen, MD | Icahn School of Medicine at Mount Sinai |
Responsible Party: | Amir Steinberg, Assistant Professor, Icahn School of Medicine at Mount Sinai |
ClinicalTrials.gov Identifier: | NCT03219359 |
Other Study ID Numbers: |
GCO 17-0378 |
First Posted: | July 17, 2017 Key Record Dates |
Last Update Posted: | August 25, 2020 |
Last Verified: | August 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
IBD Inflammatory Bowel Diseases Gastroenteritis |
Crohn's disease Colitis Vedolizumab |
Crohn Disease Inflammatory Bowel Diseases Gastroenteritis Gastrointestinal Diseases Digestive System Diseases Intestinal Diseases Methylprednisolone Cyclophosphamide Vedolizumab Thymoglobulin Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antirheumatic Agents Antineoplastic Agents, Alkylating |
Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Anti-Inflammatory Agents Antiemetics Autonomic Agents Peripheral Nervous System Agents Gastrointestinal Agents Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Neuroprotective Agents Protective Agents |