Using stem cell transplants to treat Crohn's disease
Maintenance in Autologous Stem Cell Transplant for Crohn's Disease (MASCT - CD)
This study is testing whether using stem cell transplants along with immune-suppressing drugs can help people with Crohn's disease who haven't found relief from other treatments.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Icahn School of Medicine at Mount Sinai Academic / other |
| Drugs / interventions | infliximab, adalimumab, certolizumab, golimumab, natalizumab, vedolizumab, Ustekinumab, methotrexate, cyclophosphamide, chemotherapy |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT03219359 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the use of autologous stem cell transplantation combined with immunosuppressive drugs to treat patients with Crohn's disease who have not responded to conventional therapies. The study aims to achieve remission in patients suffering from active disease characterized by significant gastrointestinal inflammation. By utilizing hematopoietic stem cell transplantation, the researchers hope to reset the immune system and improve long-term outcomes for these patients. The trial will assess the effectiveness of this approach in achieving and maintaining remission.
Who should consider this trial
Good fit: Ideal candidates for this study are patients diagnosed with Crohn's disease who have active symptoms and have failed to respond to multiple standard therapies.
Not a fit: Patients who have not been diagnosed with Crohn's disease or those who have not experienced treatment failure with standard therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new, effective option for patients with refractory Crohn's disease, potentially leading to long-term remission.
How similar studies have performed: While the use of stem cell transplantation for Crohn's disease is a novel approach, there have been some promising results in similar studies exploring this treatment modality.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
New York, New York
- Icahn School of Medicine at Mount Sinai — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Aaron Etra, MD — Icahn School of Medicine at Mount Sinai
- Study coordinator: Louis Cohen, MD
- Email: louis.cohen@mssm.edu
- Phone: (212) 241-8100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.