Ustekinumab for Crohn's disease | Ulcerative colitis

Inclusion criteria

• {"criterion_text":"- Ulcerative colitis or Crohn’s disease. Diagnosed, according to universally acknowledged criteria, a minimum of 3 months prior to inclusion"}
• {"criterion_text":"- Age ≥ 18"}
• {"criterion_text":"- Stable treatment with VDZ or UST for at least 3 months prior to inclusion"}
• {"criterion_text":"- Stable disease activity with no change in medical therapy within 3 months prior to inclusion. Mild disease activity, defined defined by fecal calprotectin ≤ 200, and a weighted PRO2 < 14 for CD or a PRO2 ≤3 for UC is allowed."}
• {"criterion_text":"- No change in medical therapy within 3 months prior to inclusion, as concomitant therapy with other immune suppressants is allowed (Azathioprine, 6-mercaptopurine, Methotrexate, 5-aminosaliclyic acid)"}
• {"criterion_text":"- The patient must be able to understand patient information material"}
• {"criterion_text":"- The patient must be able to give informed written consent"}

Exclusion criteria

• {"criterion_text":"- Having a diagnose of indeterminate colitis"}
• {"criterion_text":"- Patients who are pregnant or nursing at time of inclusion"}
• {"criterion_text":"- Having a stoma or pouch"}
• {"criterion_text":"- Fistulizing disease being the primary reason for treatment with VDZ or UST"}
• {"criterion_text":"- Expected eminent change of therapy"}
• {"criterion_text":"- Expected need for surgical intervention within the coming 3 months"}
• {"criterion_text":"- Contraindication against continuing treatment with VDZ or UST, including prior acute or delayed infusion reaction to VDZ or UST"}
• {"criterion_text":"- Any active infection requiring parenteral treatment, known infection with tuberculosis, human immunodeficiency virus (HIV) or hepatitis virus."}
• {"criterion_text":"- Any condition which the responsible physician finds incompatible with participation in the study"}
• {"criterion_text":"- Patients unable to participate in the collection of symptoms scores"}

Primary endpoints

• {"endpoint_text":"- The fraction of patients in steroid-free remission ((for CD defined by PRO2 ≤ 4, for UC defined by PRO2 = 0) AND a fecal calprotectin ≤200) at the end of the observation period (48 weeks).","definition_or_measurement_approach":"Steroid-free remission defined as: for Crohn's disease (CD) PRO2 ≤ 4, for ulcerative colitis (UC) PRO2 = 0, AND fecal calprotectin ≤ 200; assessed at end of observation period (48 weeks)."}

Secondary endpoints

• {"endpoint_text":"- The fraction of the observation period (48 weeks) where the disease is in steroid-free remission (for CD defined by PRO2 ≤ 4, for UC defined by PRO2 = 0) AND a fecal calprotectin ≤200.","definition_or_measurement_approach":"Proportion of 48-week observation period in steroid-free remission using PRO2 thresholds (CD ≤4; UC =0) and fecal calprotectin ≤200."}
• {"endpoint_text":"- The financial costs associated with the two treatment strategies over the observation period (48 weeks), including expenditure on medicines (both expenditure on the purchase of medicines and expenditure on the administration of medicines), expenses for doctor visits / visits to a nurse, possibly expenses for hospitalization, possibly expenses for surgery, possibly expenses for sick leave and possibly expenses for early retirement.","definition_or_measurement_approach":"Health economic assessment of all specified cost components over 48 weeks (drug acquisition and administration, outpatient visits, hospitalizations, surgeries, sick leave, early retirement)."}
• {"endpoint_text":"- Endoscopic healing of the mucosa assessed after 48 weeks (for CD assessed by Simple Endoscopic Score for Crohn's Disease (SES-CD) <3; and for UC assessed on the basis of the Ulcerative Colitis Endoscopic Index of Severity (UCEIS) ≤1. If disease is primarily located in the small intestine, MRI or capsule endoscopy will be used instead, and assessed by the simplified MaRIA score or Lewis score, respectively.","definition_or_measurement_approach":"Endoscopic healing at 48 weeks: SES-CD <3 for CD; UCEIS ≤1 for UC. If small bowel disease predominates, MRI (MaRIA) or capsule endoscopy (Lewis score) will be used."}
• {"endpoint_text":"- Quality of life assessed by SIBDQ at inclusion and end of treatment.","definition_or_measurement_approach":"SIBDQ administered at baseline and at end of treatment to measure QoL."}
• {"endpoint_text":"- Quality of life assessed by EQ-5D-5L and transferred to QALY. The price of QALYs will also be measured.","definition_or_measurement_approach":"EQ-5D-5L used to derive QALYs and cost per QALY over study period."}
• {"endpoint_text":"- The portion of the observational period wherein the patient’s disease is in clinical remission, assessed as the portion of the observational period in which the disease is in steroid-free remission according to Simplified Clinical Activity Index (SCCAI) for ulcerative colitis and Harvey-Bradshaw Index (HBI) for Crohn’s disease.","definition_or_measurement_approach":"Portion of 48-week period in steroid-free clinical remission using SCCAI (UC) and HBI (CD)."}
• {"endpoint_text":"- The inflammatory burden during the observational period, assessed by CRP, albumin, hemoglobin, leukocyte-measurements and fecal-calprotectin.","definition_or_measurement_approach":"Serial biochemical measures (CRP, albumin, hemoglobin, leukocytes, fecal calprotectin) over observation period."}
• {"endpoint_text":"- Drug concentration analysis expenses.","definition_or_measurement_approach":"Accounting of costs related to drug concentration testing."}
• {"endpoint_text":"- Drug expenses","definition_or_measurement_approach":"Accounting of drug-related costs during study period."}
• {"endpoint_text":"- The proportion of patients switching to another drug during the study period.","definition_or_measurement_approach":"Proportion of participants who switch therapy during the 48-week study period."}
• {"endpoint_text":"- Consumption of steroids, defined as redeemed prescriptions from the apothecary. Surgery related to IBD.","definition_or_measurement_approach":"Steroid consumption measured by redeemed prescriptions; record of IBD-related surgeries."}
• {"endpoint_text":"- Fatigue assessed by VAS-F difference between patients in the intervention and control group","definition_or_measurement_approach":"Difference in VAS-F fatigue scores between arms."}
• {"endpoint_text":"- The diseases impact on work productivity and performance of daily activities assessed by difference in WPAI scores and changes in scores between patients in the intervention and control group","definition_or_measurement_approach":"WPAI scores compared between intervention and control groups; change scores assessed."}

Denmark

Earliest CTIS Part Ii Submission Date

01-04-2025

Latest Decision Or Authorization Date

13-04-2025

Processing Time Days

12

Number Of Sites

6

Number Of Participants

166

Primary sponsor

Full Name

Odense University Hospital

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Denmark

Third parties

• {"country":"Denmark","full_name":"Odense University Hospital","duties_or_roles":"Codes: 1, 8","organisation_type":"Hospital/Clinic/Other health care facility"}