A Phase IIa, Open Label, Single-centre Study to Assess the Initial Antifibrotic Efficacy, Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Profile of MBF-118 in Crohn's Disease Patients with Stenosis
Overview
- Phase
- Phase 2
- Intervention
- MBF-118 oral capsules
- Conditions
- Crohn Disease
- Sponsor
- Medibiofarma S.L.
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Number of Adverse events
- Status
- Completed
- Last Updated
- last year
Overview
Brief Summary
This is a Phase IIa (proof of concept), single center clinical trial to evaluate the safety and efficacy of daily MBF-118 oral treatment during 28 days in Crohn's disease patients on top of standard of care.
Detailed Description
This is a Phase 2a study to evaluate the safety, tolerability and exploratory efficacy of daily oral treatment with either 100 mg or 200 mg MBF-118 during 28 days in Crohn's disease patients diagnosed with stenosis, in order to provide reliable safety, tolerability, and PK data that will guide drug dose and therapeutic regimen choices in subsequent clinical studies. The study design is a single-center, open-label group consisting of two cohorts, one dosed with 100 mg MBF-118 daily for 28 days and one dosed with 200 mg MBF-118 daily for 28 days, in approximately 5 subjects per cohort without placebo control. No randomisation is required. The first five patients to start initiate the trial will be dosed with 100 mg. The subsequent five patients initiating the trial will be dosed with 200 mg. For each subject, the study duration will last a total of approximately 14-16 weeks consisting of a screening period of 2-4 weeks, a treatment period of 4 weeks and a follow-up period of 8 weeks. The subjects who have previously completed 28 days treatment with 100 mg MBF-118 and did not show any serious adverse effects will have the option to participate to an open-label 90-day extension phase. The extension phase will last approximately 19-21 weeks consisting of up to 21 days screening, 90 treatment days and 42 follow-up days.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Participants must meet each of the following criteria for enrolment into the study:
- •Male or nonpregnant, nonlactating females, age 18-
- •Males and females of childbearing potential must agree to use adequate birth control measures during the study. Females of childbearing potential must have a negative serum pregnancy test prior to Visit 2 and either be sexually abstinent or must use a hormonal (oral, implantable, or injectable) or double barrier method of birth control throughout the study, and until 60 days after the last dose of study drug. Females unable to bear children must have documentation of such in the source records (i.e., tubal ligation, hysterectomy, or postmenopausal \[defined as a minimum of 1 year since the last menstrual period\]).
- •Diagnosis of CD based on clinical, endoscopic, and histologic evidence established at least 3 months prior to Screening.
- •Has mild to severe ileal and/or colonic CD.
- •Participant has no more than 4 naïve or anastomotic small bowel strictures by MRE or IU in the terminal ileum at Screening. A stricture is defined as:
- •localized luminal narrowing (luminal ≤ 50% relative to normal adjacent bowel); AND
- •wall thickening (≥ 125% relative to adjacent bowel); AND
- •length \< 20 cm
- •If participants are using a treatment for CD, they should be on a stable dose for at least 3 months prior to study commencement. Acceptable treatments include non-steroidal anti-inflammatory drugs (NSAIDs) and anti-inflammatory biologics.
Exclusion Criteria
- •Participants who exhibit any of the following conditions are to be excluded from the study:
- •CD-related complications:
- •Previous ileorectal anastomosis, or a proctocolectomy. Patients who have received colonic resection are allowed in this study.
- •Short bowel syndrome
- •Ileostomy, colostomy, small bowel stoma, or ileoanal pouch
- •Fistulae in or adjacent to an ileal stenosis. Participants with perianal fistulae could be included if not septic. Participants with internally penetrating fistulae are excluded.
- •Suspected or diagnosed active intra-abdominal or perianal abscess that has not been appropriately treated
- •Toxic megacolon
- •Use of corticosteroid treatment for symptoms of inflammatory bowel disease within the last 2 weeks. Corticosteroids should not be taken during the screening, treatment or follow-up periods of the trial.
- •History or current diagnosis of ulcerative colitis, indeterminate colitis, ischemic colitis, nonsteroidal anti-inflammatory drug (NSAID)-induced colitis, idiopathic colitis (i.e., colitis not consistent with CD), radiation colitis, microscopic colitis, colonic mucosal dysplasia, or untreated bile acid malabsorption.
Arms & Interventions
MBF-118 100mg oral multiple dose
Drug: MBF-118 100mg oral capsules. Single daily dose. One hard gelatin capsules during 28 days. Subjects who have previously completed 28 days treatment with 100 mg MBF-118 and did not show any serious adverse effects will have the option to participate to a 90-day treatment.
Intervention: MBF-118 oral capsules
MBF-118 200mg oral multiple dose
Drug: MBF-118 100mg oral capsules. Single daily dose. Two hard gelatin capsules during 28 days
Intervention: MBF-118 oral capsules
Outcomes
Primary Outcomes
Number of Adverse events
Time Frame: 12 weeks
Evaluate the safety of MBF-118 administered for 28 days in subjects with Crohn's disease by assessing the number, severity, and type of adverse events.