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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04548583
Other study ID # 20-845
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date November 4, 2020
Est. completion date October 2023

Study information

Verified date April 2022
Source The Cleveland Clinic
Contact Alex VanDenBossche, BSN
Phone 216-3790307
Email ibdstemcelltherapy@ccf.org
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Crohn's disease has several phenotypes (inflammatory, stricturing, fistulizing) and location (small bowel, ileocecal, colon, and perianal). Approximately one third of patients have inflammation limited to the colon. Up to two thirds will become medically refractory and require a total abdominal colectomy for symptom control. The purpose of this study is to determine the safety and efficacy of using allogeneic bone marrow derived mesenchymal stem cells (MSCs) delivered by targeted endoscopic delivery to treat people for medically refractory Crohn's colitis.


Description:

Participants with medically refractory Crohn's colitis will be treated by targeted endoscopic delivery of remestemcel-L, an ex vivo culture expanded allogeneic bone marrow derived mesenchymal stem cell product at a dose of 150 or 300 million. This will be injected into the submucosal layer of the colon and rectal wall. Patients will receive a second dose of remestemcel-L at a dose of 150 or 300 million MSCs (same dose as initial). If at 3 months post injection of remestemcel-L there is clinical remission, escalation of medical management and/or surgery will be delayed and patients observed. If there is worsening or no improvement in treated patients, then patients will proceed with escalation of medical management or colectomy as per standard of care. Control patients without improvement will cross over to receive remestemcel-L at 3 months and may be retreated at 6 months. All patients will be followed for two years post initial treatment. There will be a total of 4 cohorts of 3 patients (2 treatment:1 control) receiving the 150 million MSC dose of study drug and a total of 4 cohorts of 3 patients (2 treatment:1 control) receiving 300 million MSCs dose of study drug. This study plans to enroll a total of 24 participants. The primary endpoint of this study is to determine the safety and feasibility of endoscopic injection of remestemcel-L, an ex vivo culture expanded allogeneic bone marrow derived mesenchymal stem cell product for treatment of medically refractory Crohn's colitis.


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Remestemcel-L
adult allogeneic bone marrow derived mesenchymal stem cell product for the treatment of medically refractory Crohn's colitis
Remestemcel-L
adult allogeneic bone marrow derived mesenchymal stem cell product for the treatment of medically refractory Crohn's colitis
Other:
Placebo
Normal saline

Locations

Country Name City State
United States Cleveland Clinic Cleveland Ohio

Sponsors (2)

Lead Sponsor Collaborator
The Cleveland Clinic Mesoblast, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Treatment related adverse events The primary endpoint of this study is to determine the safety and feasibility of endoscopic injection of remestemcel-L, an ex vivo expanded allogeneic bone marrow-derived mesenchymal stem cell product, for treatment of medically refractory Crohn's colitis. Month 3
Secondary Complete clinical healing Number of participants with complete clinical healing post-injection of 150 or 300 million bone marrow allogeneic derived mesenchymal stem cells for the treatment of medically refractory Crohn's colitis.
Complete healing is defined as: Clinical and endoscopic remission
Clinical Healing: Normalization of CRP to <2.87 mg per liter, CDAI drops to <150
Radiographic Healing: MR enterography with improvement of inflammation
Endoscopic healing: Absence of mucosal ulceration and SES-CD score of 0-5
Month 3, Month 12
Secondary Clinical response Number of participants with clinical response post-injection of 150 or 300 million allogeneic bone marrow derived mesenchymal stem cell product for the treatment of medically refractory Crohn's colitis.
Clinical response is defined as:
Clinical Healing: Normalization of CRP to <2.87 mg per liter, CDAI drops to <150
Radiographic Healing: MR enterography with improvement of inflammation
Endoscopic healing: Absence of mucosal ulceration and SES-CD score of 0-5
Month 3, Month 12
Secondary Partial clinical response Number of participants with partial clinical response post-injection of 150 or 300 million allogeneic bone marrow derived mesenchymal stem cell product for the treatment of medically refractory Crohn's colitis.
Partial clinical response is defined as:
Clinical Healing: >25% reduction of CRP, decrease in CDAI by <100 points
Radiographic Healing: MR enterography with improvement in inflammation
Endoscopic healing: Decreased SES-CD by >25% but < 50% or to score of 10-15
Month 3, Month 12
Secondary Lack of response Number of participants with lack of response post-injection of 150 or 300 million allogeneic bone marrow derived mesenchymal stem cell product for the treatment of medically refractory Crohn's colitis.
Lack of response is defined as:
Clinical Healing: No improvement
Radiographic Healing: MR enterography without resolution of inflammation
Endoscopic healing: No improvement in SES-CD
Month 3, Month 12
Secondary Crohn's disease activity index Crohn's disease activity index will be used to measure quality of life in participants.
*Remission of Crohn's disease is defined as CDAI below 150. Severe disease is defined as a value of greater than 450.
Month 1 through Month 24
Secondary Inflammatory bowel disease questionnaire Inflammatory bowel disease questionnaire will be used to measure quality of life in participants.
*Score ranges from 32 (best health) to 224 (worst health)
Month 1 through Month 24
Secondary EuroQol 5 Dimensions survey EuroQol 5 Dimensions survey will be used to measure quality of life in participants.
*Score ranges from 5 (full health) to 25 (worst health).
Month 1 through Month 24
Secondary Inflammatory bowel disease patient reported treatment impact survey IBD-patient reported treatment impact survey will be used to measure quality of life in participants.
*Score ranges from 3 (most satisfied) to 15 (least satisfied)
Month 1 through Month 24
Secondary Short Form 36 health survey Short Form 36 health survey will be used to measure quality of life in participants.
*Score ranges from 0 (least favorable health state) to 3600 (most favorable health state)
Month 1 through Month 24
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