MedPath

Feasibility Pilot Sequential Multiple Assignment Randomized Trial (SMART) for Acute Severe Ulcerative Colitis

Registration Number
NCT05867329
Lead Sponsor
Berinstein, Jeffrey
Brief Summary

The goal of this trial is to create personalized treatments for each patient admitted to the hospital with acute severe ulcerative colitis (ASUC). The study will test the feasibility and acceptability of these treatment strategies among patients and physicians so that the study team can later do a larger trial to test whether the medication treatment pathways help patients avoid colectomy while ensuring patient's are safe.

Detailed Description

It is anticipated that 62 participants will be enrolled on to the clinical trial and approximately 100 physicians caring for enrolled participants. There is also a third group that are eligible patients not enrolled in the trial (patients will only be interviewed and will not receive any active treatment) and will not be included in the enrollment numbers or outcome measures.

Recruitment & Eligibility

Status
RECRUITING
Sex
Female
Target Recruitment
162
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Methylprednisolone plus UpadacitinibUpadacitinib Extended Release Oral TabletMethylprednisolone IV 30mg BID plus Upadacitinib 45mg every day.
Methylprednisolone then CyclosporineCyclosporine Injection (IV)Methylprednisolone IV 30 mg BID Stage 1 and if determined to be a non-responder to Methylprednisolone, patient will receive rescue Cyclosporine (2milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) in addition to continuing Methylprednisolone for stage 2.
Oral Upadacitinib then Methylprednisolone plus cyclosporine infusionUpadacitinib Extended Release Oral TabletUpadacitinib 30 mg BID for stage 1. If a patient is a non-responder to Upadacitinib 30 mg, then the study team will stop Upadacitinib and initiate Methylprednisolone IV 30mg twice a day plus Cyclosporine (2 milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) for stage 2.
Methylprednisolone then UpadacitinibUpadacitinib Extended Release Oral TabletMethylprednisolone IV 30mg twice a day Stage 1 and if determined to be a non-responder to Methylprednisolone, patient will receive rescue Upadacitinib 30mg BID in addition to continuing Methylprednisolone for stage 2
Methylprednisolone plus Upadacitinib then increased UpadacitinibUpadacitinib Extended Release Oral TabletMethylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and if determined to be a non-responder to Methylprednisolone and 45 mg Oral Upadacitinib, patient will receive rescue Upadacitinib 30mg BID in addition to continuing Methylprednisolone for stage 2.
Oral Upadacitinib then MethylprednisoloneUpadacitinib Extended Release Oral TabletOral Upadacitinib 30mg BID for stage 1 then for patients that are non-responders, add rescue Methylprednisolone IV 30mg twice a day in addition to continuing Upadacitinib for stage 2.
Methylprednisolone plus Upadacitinib then cyclosporineCyclosporine Oral ProductMethylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and then Cyclosporine 2 mg/kg per day aiming for levels 200-400ng/mL for stage 2.
Methylprednisolone plus Upadacitinib then increased UpadacitinibPrednisone Oral ProductMethylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and if determined to be a non-responder to Methylprednisolone and 45 mg Oral Upadacitinib, patient will receive rescue Upadacitinib 30mg BID in addition to continuing Methylprednisolone for stage 2.
MethylprednisoloneIntravenous MethylprednisoloneMethylprednisolone Intravenous (IV) 30 milligram (mg) twice a day (BID)
Methylprednisolone plus UpadacitinibIntravenous MethylprednisoloneMethylprednisolone IV 30mg BID plus Upadacitinib 45mg every day.
MethylprednisolonePrednisone Oral ProductMethylprednisolone Intravenous (IV) 30 milligram (mg) twice a day (BID)
Oral UpadacitinibUpadacitinib Extended Release Oral TabletUpadacitinib 30 mg BID
Methylprednisolone then CyclosporineIntravenous MethylprednisoloneMethylprednisolone IV 30 mg BID Stage 1 and if determined to be a non-responder to Methylprednisolone, patient will receive rescue Cyclosporine (2milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) in addition to continuing Methylprednisolone for stage 2.
Methylprednisolone then CyclosporineCyclosporine Oral ProductMethylprednisolone IV 30 mg BID Stage 1 and if determined to be a non-responder to Methylprednisolone, patient will receive rescue Cyclosporine (2milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) in addition to continuing Methylprednisolone for stage 2.
Oral Upadacitinib then MethylprednisoloneIntravenous MethylprednisoloneOral Upadacitinib 30mg BID for stage 1 then for patients that are non-responders, add rescue Methylprednisolone IV 30mg twice a day in addition to continuing Upadacitinib for stage 2.
Methylprednisolone then UpadacitinibIntravenous MethylprednisoloneMethylprednisolone IV 30mg twice a day Stage 1 and if determined to be a non-responder to Methylprednisolone, patient will receive rescue Upadacitinib 30mg BID in addition to continuing Methylprednisolone for stage 2
Oral Upadacitinib then MethylprednisolonePrednisone Oral ProductOral Upadacitinib 30mg BID for stage 1 then for patients that are non-responders, add rescue Methylprednisolone IV 30mg twice a day in addition to continuing Upadacitinib for stage 2.
Oral Upadacitinib then Methylprednisolone plus cyclosporine infusionIntravenous MethylprednisoloneUpadacitinib 30 mg BID for stage 1. If a patient is a non-responder to Upadacitinib 30 mg, then the study team will stop Upadacitinib and initiate Methylprednisolone IV 30mg twice a day plus Cyclosporine (2 milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) for stage 2.
Oral Upadacitinib then Methylprednisolone plus cyclosporine infusionPrednisone Oral ProductUpadacitinib 30 mg BID for stage 1. If a patient is a non-responder to Upadacitinib 30 mg, then the study team will stop Upadacitinib and initiate Methylprednisolone IV 30mg twice a day plus Cyclosporine (2 milligram/kilogram (mg/kg) per day aiming for levels 200-400 nanograms per milliliter (ng/mL)) for stage 2.
Methylprednisolone plus Upadacitinib then cyclosporineCyclosporine Injection (IV)Methylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and then Cyclosporine 2 mg/kg per day aiming for levels 200-400ng/mL for stage 2.
Methylprednisolone plus Upadacitinib then cyclosporineIntravenous MethylprednisoloneMethylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and then Cyclosporine 2 mg/kg per day aiming for levels 200-400ng/mL for stage 2.
Methylprednisolone plus Upadacitinib then increased UpadacitinibIntravenous MethylprednisoloneMethylprednisolone IV 30mg BID and Oral Upadacitinib 45mg everyday stage 1 and if determined to be a non-responder to Methylprednisolone and 45 mg Oral Upadacitinib, patient will receive rescue Upadacitinib 30mg BID in addition to continuing Methylprednisolone for stage 2.
Primary Outcome Measures
NameTimeMethod
Proportion of randomly allocated participants to first stage of interventionApproximately 100 Days (intervention plus 90 days of follow-up)

The study team will assess the ability of the investigators to execute the SMART, as well as the ability to treat participants with each of three first stages of intervention.

Proportion of patients who complete the trial who successfully underwent the second randomization (non-responders) or continued current therapy (responders) of all enrolled patients.Up to 100 days (intervention plus follow-up)

The study team will assess the ability of the investigators to execute the SMART, as well as the ability to treat participants to complete one of the adaptive treatment strategies and complete the SMART study (including the intervention and follow-up period).

Proportion of inpatient physicians reporting trial design acceptableApproximately Day 10 (end of study treatment period)

This will be measured by semi-structured interviews with patients and clinicians at the end of study period. One question for this outcome will be asked the study was acceptable (yes or no).

Proportion of patients with completed CRP and daily bowel movements recorded on Day 2 or Day 3 prior to sequentially randomized allocationDay 2 or Day 3 (prior to sequentially randomized allocation)

To assess the feasibility of using bowel movements and CRP as the study primary tailoring variable to determine if these are too long or burdensome, and if these variables can be obtained in a timely and complete fashion.

Proportion of patients who successfully underwent the second randomization (or transition to the second stage among those not re-randomized)Up to 10 days

The study will assess the feasibility of using real-time sequentially randomized allocation performed successfully by the treatment team.

Proportion of patients reporting trial design acceptableApproximately Day 10 (end of study treatment period)

This will be measured by semi-structured interviews with patients and clinicians at the end of study period. One question for this outcome will be asked the study was acceptable (yes or no).

Proportion of randomly assigned patients who completed study period and 90-day follow-upUp to 100 days (intervention plus follow-up)

The study team will assess the ability of the investigators to execute the SMART, as well as the ability to treat participants to complete one of the adaptive treatment strategies and complete the SMART study (including the intervention and follow-up period).

Secondary Outcome Measures
NameTimeMethod
Proportion of patients in steroid-free remission at 90 daysAt 90-day follow-up

Patients will be assessed for on-going steroid use (any dose) at the 90-day follow-up and will be recorded as yes/no and compared between arms.

Proportion of patients undergoing colectomy within follow-upUp to 100 days (intervention plus follow-up)

Colectomy events within follow-up of index admission will be recorded as yes/no and compared between arms.

Proportion of patients without rescue therapy during intervention period or colectomy within 90 days per treatmentUp to 100 days (intervention plus follow-up)

Patients will be assessed for initiation non-study rescue therapy of at the 90-day follow-up and will be recorded as yes/no and compared between arms.

Incidence and severity of adverse eventsUp to 100 days (intervention plus follow-up)

Incidence and severity of adverse events will be reported. Shingles, acne, major cardiovascular events, venous thromboembolic events, cancers and other infections are adverse events of special interest (AESI). The study team will also record the incidence of serious infections and incidence and severity of laboratory abnormalities between first treatment stage and adaptive treatment strategies.

Adverse event will be graded as described in the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.

Proportion of patients undergoing same-admission colectomyApproximately 10 days (prior to discharge from index admission)

Colectomy events prior to discharge from index admission will be recorded as yes/no and compared between adaptive treatment strategies.

Proportion of patients in initial clinical response (C-reactive protein and number of bowel movements) without non-trial rescue therapy or colectomy at 120 hours (5 days) after initiating first stage therapy per to treatment and ATS.5 days from randomization

Patients assessed for initial clinical response, initiation of non-trial rescue therapy, or colectomy with 120 hours from randomization. Initial clinical response is defined by a reduction in bowel movements per 24 hours by ≥ 60% from enrollment or less than 4 bowel movements per day AND a C-reactive protein (CRP) \< 1 mg/dL

Trial Locations

Locations (1)

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

© Copyright 2025. All Rights Reserved by MedPath