ExoFlo for ulcerative colitis: A Phase I study of ExoFlo, an ex vivo culture-expanded adult allogeneic bone marrow mesenchymal stem cell derived extracellular vesicle isolate product, for the treatment of medically refractory ulcerative colitis
To evaluate the usefulness, safety, and effectiveness of intravenous ExoFlo in patients with ulcerative colitis who are not responding to conventional therapies.
- Adults (18-75 years)
- Patient has been diagnosed with ulcerative colitis for at least 6 months who has failed to have improvement after receiving at least one monoclonal antibody for 8 weeks prior to enrollment
- Patients who do not tolerate conventional therapy
- The patient may have to stop certain medications for a specific period of time before enrolling in the trial
- No colon cancer or abnormal tissues which may develop into cancer diagnosed by colonoscopy within 30 days of enrollment
- Ability to follow the protocol and provide informed consent
- Willing to comply with all study procedures and participate for the length of the study
- Willing to use adequate birth control measures throughout the study
- Heart, liver, kidney, or other diseases which may make it unsafe for the patient to participate in the trial
- History of cancer within 5 years of enrollment except for nonmelanoma skin cancers
- Severe inflammation, infection, perforation, or cancer of the colon
- Multiple surgeries removing sections of the small bowel or colon, or surgeries which involved creating a stoma
- Women who are pregnant or breastfeeding or trying to become pregnant
Study description/explanation of participation:
The patient will be evaluated to determine if they meet the qualifications for the study. If the patient is enrolled in the study, the patient will be evaluated by the physician and receive an intravenous dose of bone marrow mesenchymal stem cell derived extracellular vesicles (ExoFlo) each visit. The patient will receive ExoFlo intravenously a total of 15 doses over 46 weeks. There will be a total of 17 visits, with the last visit during week 58.
Description of treatment or intervention (mechanism of action):
ExoFlo may help decrease inflammation and promotes wound healing and tissue regeneration. The patient may benefit from ExoFlo and be able to avoid surgical removal of a portion of the intestine and side effects from immunosuppressive therapies currently used for ulcerative colitis.
The patient will be evaluated to determine whether the study enrollment criteria are met. This evaluation includes a thorough physician evaluation including physical assessment, medical history, blood work, stool samples, radiology studies, coloscopy, heart studies, and questionnaires focused on symptoms and quality of life. The patient will have a special radiology study of the intestinal tract and a colonoscopy with biopsy during the screening process. A dose of bone marrow mesenchymal stem cell derived extracellular vesicles (ExoFlo) through an IV over sixty minutes every other day for a total of 3 doses. Two weeks after that, the patient will receive the 4th dose, followed by a dose every month for 11 months. Additional assessments and testing will occur with each physician visit during the trial. At times, the patient will have a special radiology study of the intestinal tract during the study or a special scope of the colon or sigmoid with a biopsy. The patient will be asked to complete questionnaires during each visit to evaluate symptoms associated with ulcerative colitis.
Possible risks and side effects:
Over 300 patients have been treated with ExoFlo intravenously and there are no known side effects caused by ExoFlo to date.