Stem Cell Transplantation in Crohn's Disease
Autologous Hematopoietic Stem Cell Transplantation for Refractory Crohn's Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 13 Years to 28 Years
- 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
Not stated in the study description — ask the study team about visits and procedures.
- 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 an experimental treatment for severe Crohn's disease in people ages 13–28 when medicines have not worked well. It removes overactive immune cells, then gives back blood stem cells collected from the patient's own body. The goal is to reset the immune system.
Unfortunately, some patients with Crohn's disease (CD) fail to respond to the best clinical treatments and some only experience temporary benefit. For severe Crohn's disease, there is an experimental treatment called "high dose immunoablation" followed by autologous hematopoietic stem cell transplantation (HSCT). This study removes over active lymphocytes (immunoablation) and replaces them using blood stem cells that have been taken from the patient's own body. The aim of the study is to reset or reprogram the patient's immune system to its state prior to diagnosis.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- You are 13–28 years old.
- Tests confirmed Crohn's disease at least 6 months before screening.
- Your disease is active, with a Crohn's activity score above 30 and at least 2 signs: a high inflammation blood test, inflammation seen with a scope and tissue sample, or active small bowel disease on CT or MRI.
- Your disease has not improved enough after steroids and 3 medicines that lower immune activity. You have at least 1 flare a year despite ongoing treatment, or these medicines cause harmful side effects.
- Surgery is not a good option, or you risk having too little small bowel left to absorb enough nutrients.
- Most members of the study's bowel disease care team agree you are a good candidate.
- You give informed consent, agree to extra study visits and procedures, and receive detailed counseling about the risks.
Who can't join
- You are pregnant or unwilling to use suitable birth control during the study.
- You have severe kidney, heart, or lung disease; another long-term illness causing major organ failure; or blood pressure of 140/90 or higher despite at least 2 medicines.
- You have a mental health disorder, including current drug or alcohol abuse, or current or recent cancer other than non-melanoma skin cancer.
- You have HIV, HTLV-1 or HTLV-2, a hepatitis virus, or another infection the study team considers unsafe. You have a significant active infection or an abscess, a pocket of infection.
- You have a fistula near the anus—an abnormal tunnel—that does not drain freely. A fistula may be allowed if it drains naturally or through a special stitch.
- You have had tuberculosis, are at increased risk for it, have tests suggesting active tuberculosis, or have a chest X-ray suggesting active infection or a tumor.
- You have serious poor nutrition, listed as a body mass index (BMI) of 18 or lower and blood albumin, a protein, below 20 g/L.
- You have a history of not following treatment instructions, or you are in another study using an experimental drug or blood cell growth medicine, including within 4 weeks before study entry.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Aged 13-28 years are eligible 2. Confirmed diagnosis of active Crohn's disease: 1. Diagnosis of Crohn's disease based on typical radiological appearances and / or typical histology at least 6 months prior to screening. 2. Active disease at the time of registration to the trial, defined as i) PCDAI \> 30, and ii) Two of the following: 1. elevated CRP 2. endoscopic evidence of active disease confirmed by histology 3. clear evidence of active small bowel Crohn's disease on CT or MR enterography. 3. Unsatisfactory course despite 3 immunosuppressive agents (usually azathioprine, methotrexate and infliximab, adalimumab and/or certolizumab) in addition to corticosteroids. Patients should have relapsing disease (i.e. 1 exacerbation/year) despite thiopurines, methotrexate and/or infliximab/adalimumab/certolizumab maintenance therapy or clear demonstration of intolerance / toxicity to these drugs. 4. Current problems unsuitable for surgery or patient at risk for developing short bowel syndrome. 5. Accepted by a majority of the members of the combined IBD Center as an appropriate candidate (see Selection description below). 6. Informed consent 1. Prepared to undergo additional study procedures as per trial schedule 2. Patient has undergone intensive counseling about risks Exclusion Criteria: 1. Pregnancy or unwillingness to use adequate contraception during the study, in women of childbearing age. Unwillingness of using appropriate contraceptive measures in males. 2. Concomitant severe disease 1. renal: creatinine clearance \< 30 mL/min (measured or estimated) 2. cardiac: clinical evidence of refractory congestive heart failure; left ventricular ejection fraction \< 40% by cardiac echo; chronic atrial fibrillation necessitating oral anticoagulation; uncontrolled ventricular arrhythmia; pericardial effusion with hemodynamic consequences as evaluated by an experienced echo cardiographer 3. pulmonary: diffusion capacity \<40% 4. psychiatric disorders including active drug or alcohol abuse 5. concurrent or recent history of malignant disease (excluding non-melanoma skin cancer) 6. uncontrolled hypertension, defined as resting systolic blood pressure ≥ 140 and/or resting diastolic pressure ≥ 90 despite at least 2 anti-hypertensive agents. 7. any infection with HIV, HTLV-1 or 2, hepatitis viruses, or any other infection the investigators consider a contraindication to participation. 8. other chronic disease causing significant organ failure. 3. Infection or risk thereof: 1. Current clinical relevant abscess or significant active infection. 2. Perianal fistula without free drainage. Perianal fistulas is not an exclusion provided there is natural free drainage or a seton suture(s) have been placed. 3. History of tuberculosis or at current increased risk of tuberculosis 4. Quantiferon Gold test result or other investigations that the investigators regard as evidence of active tuberculosis. 5. Abnormal chest X-ray (CXR) consistent with active infection or neoplasm. 6\) Significant malnutrition: Body Mass Index (BMI) ≤ 18, serum albumin \< 20 g/l. 7\) Previous poor compliance. 8) Concurrent enrollment in any other protocol using an investigational drug or hematopoietic growth factor up to four weeks before study entry.
Study sites (1)
CA
- Cedars-Sinai Medical Center · Los Angeles, CA 90048David Ziring, MD · Study contact · 310-423-7100 · David.Ziring@cshs.orgYvette Gonzales, MBA · Study contact · 310-423-4072 · Yvette.Gonzales@cshs.orgDavid Ziring, MD · Principal investigatorShervin Rabizadeh, MD · Sub-investigatorMore trials in Los Angeles