DB-3Q bmMSC-EVs in Patients With Perianal Fistulizing Crohn's Disease
A Phase 2a Study of Allogeneic Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicle Isolate Product (DB-3Q) in Patients With Perianal Fistulizing 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
- 18 Years to 75 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 the safety of increasing doses of DB-3Q and how well it works compared with a placebo, a treatment with no active drug. It is for adults with Crohn’s disease who have draining fistulas near the anus. Fistulas are abnormal tunnels between body parts.
This is a double-blind, randomized, placebo-controlled, sequential cohort, ascending dose clinical trial to evaluate the safety and determine the efficacy of ascending doses of DB-3Q for the treatment of Perianal Fistulizing Crohn's Disease.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Give written consent to join the study.
- Be 18–75 years old and have had Crohn’s disease for at least 6 months before treatment starts.
- Have 1 or 2 fistulas near the anus, with 1 or 2 outside openings, that are actively draining.
- Have tried at least one medicine for this condition in the past year that did not work.
- Have had surgery that did not work, including a seton (a drain placed in the fistula) at least 2 weeks before screening, or be unable or unwilling to have surgery.
- Have no changes in Crohn’s medicines for at least 2 months before treatment starts. Any antibiotics for Crohn’s must be unchanged for at least 2 weeks.
- If taking steroid pills, take no more than 20 mg of prednisone daily, or an equal dose of another steroid, with no changes for at least 4 weeks.
- Have a pelvic MRI scan approved by the study’s imaging doctor. The fistulas must meet the study’s rules for location, type, length of at least 2.5 cm, fluid, healing tissue, and scarring.
Who can't join
- Be pregnant or breastfeeding, lack a negative pregnancy test if able to become pregnant, or be unwilling to use birth control accepted by the study.
- Have an active pocket of pus or infection near the anus, or confirmed HIV, hepatitis B, or hepatitis C.
- Have had a major health problem in the past 6 months that could make the study unsafe, or be unable to safely have MRI scans.
- Have had cancer in the past year, except localized skin cancer that is not melanoma, or cancer of the colon or rectum in the past 2 years.
- Have a blood-clotting disorder or currently take blood-thinning medicine.
- Have a fistula connecting the anus or rectum to the vagina, an internal pouch made from the small intestine, past surgery to remove the rectum, or severe swelling in the rectum.
- Have screening blood tests outside the study’s allowed limits, or have used an experimental treatment within 30 days before study treatment starts.
- Have fistulas that do not meet the study’s MRI rules for location, type, activity, or amount of scarring.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Inclusion Criteria: 1. Written informed consent from participant 2. Men and women 18-75 years of age with a diagnosis of Crohn's Disease for at least six months duration prior to Day 1 3. One or two perianal fistula(s) with 1 or two external openings, that are actively draining 4. Failed at least one medical therapy within the last year including, but not limited to, antibiotics, immunomodulators (6-MP, methotrexate, azathioprine), monoclonal antibodies (adalimumab, certolizumab, golimumab, guselkumab, infliximab, risankizumab, ustekinumab, vedolizumab), or small molecule inhibitors (tofacitinib, upadacitinib) 5. Previous failed surgical intervention, including seton placement at least two weeks prior to screening, or are not candidates for surgical intervention or are not willing to undergo surgical intervention for the management of their fistula 6. Medical therapy for CD stable for at least 2 months prior to Day 1 (Changes in dosing or dosing intervals related to serum drug levels are not permitted) 7. Antibiotics for the treatment of CD (e.g., ciprofloxacin, metronidazole, or rifaximin) must have been stable for at least 2 weeks prior to Day 1 8. Oral corticosteroids ≤ 20 mg/day prednisone or its equivalent and must have been stable for at least 4 weeks prior to Day 1 9. Approval of a screening pelvic MRI by review of a central radiologist. The definition of an acceptable screening pelvic MRI is provided in the study imaging manual. 10. Fulfillment of radiologic inclusion by a central radiologist. Radiologic inclusion on MRI is presence of 1 or 2 qualifying perianal fistula(s). Radiologic inclusion assesses anatomic criteria of a perianal fistula and study-defined imaging endpoint criteria. a. Anatomically, a qualifying perianal fistula on MRI fulfills the following from central radiologist assessment: i. has a communication with the internal sphincter of the anal canal ii. is classified as a Parks intersphincteric, transsphincteric, or suprasphincteric perianal fistula. b. For study imaging endpoints, a central radiologist confirms that a fistula has a minimum score of 7 (range 0 to 25) on MAGNIFI-CD and a fistula fibrosis -to-fluid ratio of less than 80%.25, 37 Within components of MAGNIFI-CD, the fistula has a length of 2.5 cm or greater and has a composition of either 'predominantly fluid/pus' or 'predominantly granulation tissue'. c. Within components of fistula fibrosis-to-fluid ratio, the ratio of hypointense scarring or fibrosis on MRI T2 weighted sequences to hyperintense fluid on MRI T2 weighted sequences is below 80%.25 The fistula is categorized into one of following ranges of fibrosis-to- fluid ratio: i) 0%, ii) 1-19%, iii) 20-39%, iv) 40-59%, or v) 60-79% Exclusion Criteria 1. Lack of informed consent 2. Pregnant woman, woman of childbearing potential without a documented negative urine or serum pregnancy test, or woman who is breast feeding 3. A participant who is unwilling to use medically acceptable contraception methods during participation in study 4. Active perianal abscess or infection at screening or Day 1 5. Clinically significant medical conditions within six months before Day 1 that would, in the opinion of the Investigator, compromise the safety of the participant 6. Confirmed HIV, Hepatitis B, or Hepatitis C infections 7. History of cancer including melanoma (with the exception of localized non-melanoma skin cancers) within one year of screening 8. History of colorectal cancer within 2 years of screening 9. History of coagulation disorders and/or are currently on anti- coagulant therapy 10. Presence of a recto/ano-vaginal fistula 11. Presence of an ileal anal pouch and/or history of proctectomy 12. Presence of severe proctitis (SES CD \>6) 13. Any condition which, in the opinion of the Investigator, would make it unsafe or unsuitable for participant to undergo MRI evaluations (i.e., presence of implantable or external MRI unsafe device that cannot be removed, body weight exceeding limitations, claustrophobia, etc.) 14. The following out of range laboratory results at screening * WBC \> 13 x 109 /L * Hemoglobin \< 8 g/dL * Platelet count \< 100,000/mL * AST/ALT \> 3 times the upper limit of normal * Creatinine \> 2 μmol/L * PT/INR: 2 times the upper limit of normal * Hemoglobin A1c \> 6.5 mg/dL 15. Use of investigational therapy or treatment within 30 days prior to Day 1 16. Does not meet the radiologic anatomic inclusion criteria: i) has a primary communication with the rectum and not the anal canal; ii) is classified as a Parks extrasphincteric fistula 17. Does not meet the minimum thresholds for the radiologic imaging endpoint inclusion criteria: i) has a MAGNIFI-CD score of \<7 (range 0-6) on screening pelvic MRI; ii) has a fibrosis-to-fluid ratio of 80% or more (range 80-100%) on screening pelvic MRI
Study sites (5)
CO
- Direct Biologics Investigational Site · Aurora, CO 80045More trials in Aurora
MO
- Direct Biologics Investigational Site · St Louis, MO 63130More trials in St Louis
NY
- Columbia University Irving Medical Center/NYPH · New York, NY 10032Claudia Musat · Study contact · cm2065@cumc.columbia.eduMore trials in New York
PA
- Direct Biologics Investigational Site · Philadelphia, PA 19104More trials in Philadelphia
TN
- Direct Biologics Investigational Site · Nashville, TN 37232More trials in Nashville