Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622001332718
Ethics application status
Approved
Date submitted
23/07/2021
Date registered
17/10/2022
Date last updated
17/10/2022
Date data sharing statement initially provided
17/10/2022
Type of registration
Retrospectively registered

Titles & IDs
Public title
UNIFI-ED ustekinumab in patients with ulcerative colitis long term on extended follow up
Scientific title
Ustekinumab as Induction and Maintenance Therapy for Ulcerative Colitis Extension Discovery on their clinical outcomes
Secondary ID [1] 304777 0
None
Universal Trial Number (UTN)
Trial acronym
UNIFI-ED
Linked study record
This is a subgroup analysis of certain patients who were enrolled in NCT02407236, ie this study is an extended follow-up of Australian participants enrolled into the UNIFI study (NCT02407236).

Health condition
Health condition(s) or problem(s) studied:
ulcerative colitis 322839 0
Condition category
Condition code
Oral and Gastrointestinal 320419 320419 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Non-interventional questionnaire on the long term outcomes of ulcerative colitis patients on ustekimumab therapy. Patients are not contacted. Instead questionnaires to be distributed to the clinical trials site to determine how many patients are on the compassionate access for ustekinumab and their clinical well being.
The questionnaire will be undertaken by a site trial coordinator from the clinical trials unit from the relevant institution (hospital). These will be taken either as face-to-face or telephone consultations at the election of the site trial coordinator. The interview itself is expected to take up to 20 minutes, and will be corroborated from the clinical notes. This will be at a single time-point, and the date of this interview will be collected to determine the follow up time since enrolment in the UNIFI trial (and thus the duration they have been receiving the medication, ustekinumab).
All information will be collected retrospectively, though some of the clinical information from hospital records will have been entered prospectively. The information collected will be: demographics, baseline clinical and endoscopic severity, clinical and endoscopic severity at the end of the induction and the maintenance study, clinical relapse data, biochemical (C-reactive protein and faecal calprotectin)/endoscopic severity (total and partial Mayo score) /radiologic (computerised tomography, magnetic resonance imaging, ultrasound) since enrollment in the UNIFI trial. Furthermore all possible adverse events will be reported via interview with the patient, but also with review of the clinical notes. The above information is necessary to determine: the types of patients described, the baseline severity and initial response to ustekinumab in the cohort, as well as the subsequent efficacy and adverse event rate in the cohort on this medication.
This will be assessed at time that the trial coordinators are able to contact the consented patient, between July to December 2022
Intervention code [1] 321158 0
Diagnosis / Prognosis
Comparator / control treatment
n/a
Control group
Uncontrolled

Outcomes
Primary outcome [1] 328257 0
Cessation of ustekimumab determined by review of medical records and telephone survey or in person interview of participants
Timepoint [1] 328257 0
Between July to December 2022 (since these patients have enrolled many years ago in UNIFI), they will be interviewed opportunistically when the clinical trial coordinators at the relevant sites are able to arrange a convenient day/time. The interview date will be recorded to determine the time since UNIFI commencement and the dates they first received ustekinumab within the trial to determine the time since trial commencement.
Secondary outcome [1] 398263 0
Clinical relapse, and time to clinical disease relapse.
Clinical relapse is defined as a recurrence of colitis symptoms with commensurate biochemical, endoscopic, histological, and/or radiological evidence of disease activity that led to commencement or dose escalation of medical therapy. Such symptoms were persistent watery diarrhoea and/or abdominal pain and/or rectal bleeding. These symptoms were then confirmed by an objective measure of activity (faecal calprotectin greater than 250ug/g from baseline, endoscopic activity with Mayo score 1, radiographic evidence of colitis via CT, MRI or USS). The exclusion of infection was required for the definition of a disease relapse (via stool culture and clinical assessment.
Timepoint [1] 398263 0
Between July to December 2022 (since these patients have enrolled many years ago in UNIFI), they will be interviewed opportunistically when the clinical trial coordinators at the relevant sites are able to arrange a convenient day/time. The interview date will be recorded to determine the time since UNIFI commencement and the dates they first received ustekinumab within the trial to determine the time since trial commencement.
Secondary outcome [2] 398264 0
Adverse outcome related to ustekimumab therapy. Telephone or face-to-face survey of participants. Examples include: injection site reactions, allergy to medication, infections, malignancies, need for abdominal surgery including colectomy
Timepoint [2] 398264 0
Between July to December 2022 (since these patients have enrolled many years ago in UNIFI), they will be interviewed opportunistically when the clinical trial coordinators at the relevant sites are able to arrange a convenient day/time. The interview date will be recorded to determine the time since UNIFI commencement and the dates they first received ustekinumab within the trial to determine the time since trial commencement.
Secondary outcome [3] 398265 0
Dose intensification of ustekinumab or escalation of co-therapy (dose-intensification or addition) for disease control. Telephone or face-to-face survey of participants, data-linkage to medical records.
Timepoint [3] 398265 0
Between July to December 2022 (since these patients have enrolled many years ago in UNIFI), they will be interviewed opportunistically when the clinical trial coordinators at the relevant sites are able to arrange a convenient day/time. The interview date will be recorded to determine the time since UNIFI commencement and the dates they first received ustekinumab within the trial to determine the time since trial commencement.

Eligibility
Key inclusion criteria
Patients who participated in the UNIFI study (NCT02407236 , DOI: 10.1056/NEJMoa1900750) from Australian sites, and who persisted on ustekimumab therapy as maintenance treatment for their ulcerative colitis
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients with no relevant data

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Retrospective
Statistical methods / analysis
Data will be expressed as percentages with numbers, and median or mean (depending on data distribution) with standard deviations and interquartile ranges. Chi-square test will be used to compare categorical outcome variables.
Comparisons of continuous outcome variables will be analysed using Students t test and Mann-Whitney U tests for parametric and non-parametric variables respectively. Kaplan-Meier analysis will be used to describe cessation of ustekimumab. Univariate analysis will be performed first to identify risk factors associated with cessation of ustekimumab using a proportional Cox model, with subsequent multivariate analysis using a stepwise progression. Statistical significance was taken as P<0.05.

This study is retrospective in nature and thus size and power calculations are not applicable

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
5/02/2022
Date of last participant enrolment
Anticipated
1/12/2022
Actual
Date of last data collection
Anticipated
1/12/2022
Actual
Sample size
Target
20
Accrual to date
15
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,QLD,VIC
Recruitment hospital [1] 22936 0
Concord Repatriation Hospital - Concord
Recruitment hospital [2] 22937 0
Liverpool Hospital - Liverpool
Recruitment hospital [3] 22938 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [4] 22939 0
The Alfred - Melbourne
Recruitment hospital [5] 22940 0
Austin Health - Austin Hospital - Heidelberg
Recruitment hospital [6] 22941 0
Monash University Public Health and Preventive Medicine - Melbourne
Recruitment hospital [7] 22942 0
The Canberra Hospital - Garran
Recruitment postcode(s) [1] 38242 0
2139 - Concord
Recruitment postcode(s) [2] 38243 0
2170 - Liverpool
Recruitment postcode(s) [3] 38244 0
4101 - South Brisbane
Recruitment postcode(s) [4] 38245 0
3004 - Melbourne
Recruitment postcode(s) [5] 38246 0
3084 - Heidelberg
Recruitment postcode(s) [6] 38247 0
2605 - Garran

Funding & Sponsors
Funding source category [1] 309151 0
Hospital
Name [1] 309151 0
Concord Repatriation General Hospital Dept of Gastroenterology and Liver Services
Country [1] 309151 0
Australia
Primary sponsor type
Hospital
Name
Concord Repatriation General Hospital
Address
Concord Repatriation General Hospital, Hospital Rd, Concord NSW 2139
Country
Australia
Secondary sponsor category [1] 310104 0
None
Name [1] 310104 0
Address [1] 310104 0
Country [1] 310104 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 309016 0
Sydney Local Health District HREC - Concord Repatriation General Hospital
Ethics committee address [1] 309016 0
HREC, Concord Repatriation General Hospital, Hospital Rd, Concord NSW 2139
Ethics committee country [1] 309016 0
Australia
Date submitted for ethics approval [1] 309016 0
05/10/2021
Approval date [1] 309016 0
24/11/2021
Ethics approval number [1] 309016 0
2021/PID02762

Summary
Brief summary
Observational multicentre cohort study on the long term outcomes of ulcerative colitis patients on ustekimumab therapy (post UNIFI study, NCT02407236), including time to flare, need for escalation and co-therapy for disease control. Time on treatment will be assessed from the first dose of ustekinumab, and 'survival' analysis performed with time on medication (with resutls censored at any loss to follow up. The primary outcome is proportion of patients who relapsed on ustekinumab, defined as a recurrence of flare/colitis symptoms with commensurate biochemical, endoscopic, histological, and/or radiological evidence of disease activity that led to commencement or dose escalation of medical therapy
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 112654 0
Prof Rupert Leong
Address 112654 0
Concord Hospital, Hospital Rd, Concord NSW 2139
Country 112654 0
Australia
Phone 112654 0
+61297676111
Fax 112654 0
Email 112654 0
[email protected]
Contact person for public queries
Name 112655 0
Prof Rupert Leong
Address 112655 0
Concord Hospital, Hospital Rd, Concord NSW 2139
Country 112655 0
Australia
Phone 112655 0
+61 29767 5000
Fax 112655 0
Email 112655 0
[email protected]
Contact person for scientific queries
Name 112656 0
Prof Rupert Leong
Address 112656 0
Concord Hospital, Hospital Rd, Concord NSW 2139
Country 112656 0
Australia
Phone 112656 0
+61 29767 5000
Fax 112656 0
Email 112656 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Not applicable


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseUstekinumab as induction and maintenance therapy for ulcerative colitis-national extended follow-up and a review of the literature.2023https://dx.doi.org/10.1080/14740338.2023.2278686
N.B. These documents automatically identified may not have been verified by the study sponsor.