Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2INTERVENTIONAL

Autologous Stem Cell Transplant for Crohn's Disease

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

Autologous Stem Cell Transplant for Crohn's Disease (NCT03219359) is a Phase 2 interventional studying Crohn Disease, sponsored by Aaron Etra. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Crohn Disease and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 50 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Crohn Disease subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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.

Treatments Being Tested

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)

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Icahn School of Medicine at Mount Sinai

New York, New York, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT03219359), the sponsor (Aaron Etra), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT03219359 clinical trial studying?

Who can participate in NCT03219359?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT03219359?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT03219359. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT03219359. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.