ANZCTR - Registration

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]

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

Condition category

Condition code

Oral and Gastrointestinal

Inflammatory bowel disease

Intervention/exposure

Study type

Observational

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]

Diagnosis / Prognosis

Comparator / control treatment

n/a

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Cessation of ustekimumab determined by review of medical records and telephone survey or in person interview of participants

Timepoint [1]

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]

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]

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]

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]

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]

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]

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

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]

Concord Repatriation Hospital - Concord

Recruitment hospital [2]

Liverpool Hospital - Liverpool

Recruitment hospital [3]

Mater Adult Hospital - South Brisbane

Recruitment hospital [4]

The Alfred - Melbourne

Recruitment hospital [5]

Austin Health - Austin Hospital - Heidelberg

Recruitment hospital [6]

Monash University Public Health and Preventive Medicine - Melbourne

Recruitment hospital [7]

The Canberra Hospital - Garran

Recruitment postcode(s) [1]

2139 - Concord

Recruitment postcode(s) [2]

2170 - Liverpool

Recruitment postcode(s) [3]

4101 - South Brisbane

Recruitment postcode(s) [4]

3004 - Melbourne

Recruitment postcode(s) [5]

3084 - Heidelberg

Recruitment postcode(s) [6]

2605 - Garran

Funding & Sponsors

Funding source category [1]

Hospital

Name [1]

Concord Repatriation General Hospital Dept of Gastroenterology and Liver Services

Country [1]

Australia

Primary sponsor type

Hospital

Name

Concord Repatriation General Hospital

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Country [1]

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Sydney Local Health District HREC - Concord Repatriation General Hospital

Ethics committee address [1]

HREC, Concord Repatriation General Hospital, Hospital Rd, Concord NSW 2139

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

05/10/2021

Approval date [1]

24/11/2021

Ethics approval number [1]

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

Public notes

Contacts

Principal investigator

Name

Prof Rupert Leong

Address

Concord Hospital, Hospital Rd, Concord NSW 2139

Country

Australia

Phone

+61297676111

Email

[email protected]

Contact person for public queries

Name

Rupert Leong

Address

Concord Hospital, Hospital Rd, Concord NSW 2139

Country

Australia

Phone

+61 29767 5000

Email

[email protected]

Contact person for scientific queries

Name

Rupert Leong

Address

Concord Hospital, Hospital Rd, Concord NSW 2139

Country

Australia

Phone

+61 29767 5000

Email

[email protected]

Trial Review

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Documents added automatically