hucMSCs Exosomes for the Treatment of Active Ulcerative Colitis
ConditionUlcerative Colitis (UC)
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorShanghai East Hospital
About this trial
To evaluate the safety and efficacy of hUC-MSCs-Exos in the treatment of ulcerative colitis.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Subjects have had UC for at least 3 months (since symptom onset). The diagnosis should be confirmed by clinical and endoscopic evidence and confirmed by histopathological reports (note: if no previous reports are available, endoscopy and histopathology may be performed at the time of screening).
Subjects had active UC, defined as four-component Mayo score of 6-12 (inclusive), endoscopy score ≥2, rectal bleeding score ≥1, and bowel frequency score ≥1.
18 to 75 years old, weight ≥40 kg
Meet at least one of the following a/b/c criteria: a. inadequate or non-response to one or more of the following treatments: i) oral prednisone ≥40mg/ day (or equivalent) or budesonide ≥9mg/ day or equivalent or beclomethasone ≥5mg/ day for at least 2 weeks. ii) At least 8 weeks of immunomodulators (AZA≥2 mg/kg/ day or 6-MP≥1.0mg/kg/ day [or lower doses, but 6-thioguanine nucleotides with therapeutic concentrations recorded]). iii) Oral administration of aminosalicylate (e.g. Mesalazine, salazine sulfopyridine, oxalazine, balsalazine) in accordance with the dosage and duration of the applicable local instructions. iv) The frontier therapy for UC has completed at least the induction dosing regimen, At doses greater than or equal to the approved instructions: anti-TNF anti-integrins (e.g., Vederizumab), JAK inhibitors (e.g., Tofaciib, Upatinib, or filgotinib), anti-IL-23 or anti-IL-12/23 drugs for the treatment of UC (e.g., ulinumab), S1PR modulators (e.g., ozamod) b. Corticosteroid dependence: failure to taper successfully to <10mg/ day of prednisone or equivalent or <6mg/ day of budesonide or <5mg/ day of beclometasone within 3 months of starting treatment (i.e., disease onset), or relapse occurs within 3 months of stopping corticosteroids. c. Intolerance to 1 or 2 of the following treatments (e.g., inability to reach the therapeutic dose or duration of treatment due to dose-limiting adverse reactions) i) corticosteroids: Adverse reactions associated with dose-limiting therapy may include, but are not limited to, infections, hyperglycemia, osteoporosis, insomnia, or psychiatric disorders. ii) Immunomodulators: Adverse reactions associated with dose-restricted therapeutic administration may include, but are not limited to, infection, nausea/vomiting, fatigue, myelosuppression, or liver toxicity.
Disqualifiers
Diagnosis of CD or undefined colitis (IBD- undefined) or other types of colitis or enteritis that may confuse assessment of effectiveness.
The current diagnosis is explosive colitis and/or toxic megacolon.
Had received fecal microbial transplantation within 4 weeks prior to randomization.
Had been hospitalized for UC within 2 weeks prior to screening.
Trial design
Design model
Parallel
Treatments tested in this trial
exosomes derived from human umbilical cord mesenchymal stem cells
DrugThe corresponding exosome content of 60×10\^6 umbilical cord mesenchymal stem cells was given
saline +5% albumin
DrugEqual amount of saline +5% albumin was given
Treatment groups
40 Participants
are divided into 2 treatment groupsGroup A: Exos Localized Treatment GroupExperimental treatment 1 intervention
Group B: Placebo Localized Treatment GroupPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Mayo Score
The complete Mayo Score consists of four assessments, each scored on a scale of 0 to 3: stool frequency, rectal bleeding, endoscopy, and the physician's global assessment (PGA) of disease activity. The endoscopy subscore of the complete Mayo Score is derived from the central reading of endoscopy results by a qualified central laboratory. Patient-reported stool frequency and rectal bleeding, as well as the PGA reported by the clinician, will be collected in an electronic diary.
Time frame
Baseline, 1 week, 4 weeks, 8 weeks, and 12 weeks after treatment.Secondary outcomes
1
Geboes Score
It is the most commonly used histological scoring system for colonic mucosa in the field of ulcerative colitis (UC). It is divided into six grades: 1) Structural changes (Grade 0); 2) Chronic inflammatory infiltration (Grade 1); 3) Neutrophils and eosinophils in the lamina propria (Grade 2); 4) Neutrophils in the epithelium (Grade 3); 5) Crypt destruction (Grade 4); 6) Erosion or ulceration (Grade 5). Each grade is further divided into four subcategories. The Geboes score ranges from 0 to 5.4, with higher scores indicating more severe inflammation. Typically, active histological inflammation in UC is defined as a Geboes score of ≥2B.1.
Time frame
Baseline, at 12 weeks after treatment.2
Inflammatory Bowel Disease Questionnaire (IBDQ)
The IBDQ is a well-established assessment tool for measuring disease-specific health-related quality of life. It consists of 32 items : bowel symptoms (10 items), systemic symptoms (5 items), emotional functioning (12 items), and social functioning (5 items). The questionnaire is scored using a 7-point Likert scale, ranging from worst health (1) to best health (7). Domain scores are calculated by summing the item responses, with higher values indicating better quality of life.The total score range of the IBDQ is 32-224, which can be obtained by adding up the scores of individual domains. According to the total score, a meaningful change in the IBDQ score (also defined as an IBDQ response) is defined as a change of ≥ 16 points, and this threshold has been applied in studies involving subjects with Crohn's disease (CD) or ulcerative colitis (UC). In addition, IBDQ remission can be defined as a total score of ≥ 170 points.
Time frame
Baseline, at 1 week, 4 weeks, 8 weeks, and 12 weeks after treatment.3
The item of urgency to defecate
Subjects are required to complete a single item that assesses how frequently they needed to empty their bowels immediately to avoid an accident in the past 7 days. The response scale ranges from 0 (never) to 4 (more than once a day).
Time frame
Baseline, at 1 week, 4 weeks, 8 weeks, and 12 weeks after treatment.4
EQ-5D-5L
The EQ-5D-5L is a brief self-report measure of health status and function. The descriptive system of this instrument includes 5 items for measuring problems (none, mild, moderate, severe, or extreme), covering mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. In total, the responses to these items describe 5\^5 = 3,125 health states. The responses of the subjects to the 5 items can be weighted and aggregated to generate a utility index score that measures the social value of their current health. These scores range from 1 (perfect health) to 0 (death), and negative values indicate a health state worse than death. Additionally, the EQ-5D-5L includes a Visual Analogue Scale (VAS), which allows subjects to rate their current health on a scale from 0 (worst imaginable health) to 100 (best imaginable health). Among patients receiving treatment for IBD, a meaningful change in the EQ-5D-5L VAS score is defined as a change of ≥ 10 points.
Time frame
Baseline, at 1 week, 4 weeks, 8 weeks, and 12 weeks after treatment.Sponsors and contacts
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Source ClinicalTrials.gov
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