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RecruitingPhase 2

Autologous Stem Cell Transplant for Crohn's Disease

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: CT scans

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Patients will have monthly study visits and a repeat colonoscopy and MR/CT scan at 6 months."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests a transplant using a person's own stem cells, followed by the drug vedolizumab, for Crohn's disease. It checks whether this treatment can help control the disease and keep it under control. It is for adults with active Crohn's disease whose medicines have not worked or cannot be used, and who cannot have or do not want surgery.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have a diagnosis of Crohn's disease.
  • Have active symptoms, with a Crohn's disease activity score (CDAI) above 250. For people with an ostomy, daily bag emptyings replace the count of liquid stools.
  • Have active disease seen with a scope inside the bowel, with an SES-CD score above 3 in at least one section.
  • Have tried without success, had bad reactions to, or declined a drug from each listed group: corticosteroids; azathioprine; 6-mercaptopurine or methotrexate; anti-TNF drugs; anti-integrin drugs; and ustekinumab.
  • If treatment has not worked, a digestive disease doctor must confirm ongoing swelling and irritation along with symptoms.
  • Have no surgery option because it could leave too little bowel, or choose not to have surgery.

Who can't join

  • Are younger than 18, pregnant, or breastfeeding.
  • Have had serious harmful effects from cyclophosphamide, thymoglobulin, or vedolizumab.
  • Have a Karnofsky score below 60, a measure of how well you can do daily tasks.
  • Have a suspected or confirmed infection that remains uncontrolled after at least one month of proper treatment, or have HIV.
  • Have symptoms from disease in the heart's blood vessels, or uncontrolled heart failure.
  • Have a heart pumping measure called ejection fraction below 30%, or need oxygen all the time.
  • Have a lung test score called DLCO below 35%, which measures how well lungs move gas into the blood, or need extra oxygen.
  • Have too few stem cells collected: fewer than 2 million per kilogram of body weight.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 sites (1)

NY