NCT03219359 Autologous Stem Cell Transplant for Crohn's Disease
| NCT ID | NCT03219359 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Aaron Etra |
| Condition | Crohn Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 50 participants |
| Start Date | 2018-02-22 |
| Primary Completion | 2028-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 50 participants in total. It began in 2018-02-22 with a primary completion date of 2028-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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.
Contact & Investigator
Aaron Etra, MD
PRINCIPAL INVESTIGATOR
Icahn School of Medicine at Mount Sinai
Frequently Asked Questions
Who can join the NCT03219359 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Crohn Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03219359 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT03219359 currently recruiting?
Yes, NCT03219359 is actively recruiting participants. Contact the research team at louis.cohen@mssm.edu for enrollment information.
Where is the NCT03219359 trial being conducted?
This trial is being conducted at New York, United States.
Who is sponsoring the NCT03219359 clinical trial?
NCT03219359 is sponsored by Aaron Etra. The principal investigator is Aaron Etra, MD at Icahn School of Medicine at Mount Sinai. The trial plans to enroll 50 participants.