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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04206553




Registration number
NCT04206553
Ethics application status
Date submitted
18/12/2019
Date registered
20/12/2019

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
Scientific title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
Secondary ID [1] 0 0
2019-003520-20
Secondary ID [2] 0 0
R668-BP-1902
Universal Trial Number (UTN)
Trial acronym
LIBERTY-BP
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Bullous Pemphigoid 0 0
Condition category
Condition code
Skin 0 0 0 0
Dermatological conditions
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - dupilumab
Treatment: Drugs - Matching Placebo
Treatment: Drugs - Oral corticosteroids (OCS)

Experimental: dupilumab -

Experimental: Matching placebo -


Treatment: Drugs: dupilumab
Loading dose administered subcutaneous (SC), followed by SC once every 2 weeks (Q2W) dosing.

Treatment: Drugs: Matching Placebo
Matching dupilumab without active substance

Treatment: Drugs: Oral corticosteroids (OCS)
Prednisone or prednisolone per standard of care to obtain control of disease activity.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients achieving sustained remission
Timepoint [1] 0 0
Week 36
Secondary outcome [1] 0 0
Total cumulative dose of oral corticosteroids (OCS)
Timepoint [1] 0 0
Baseline to week 36
Secondary outcome [2] 0 0
Percent change in weekly average of daily peak pruritus numerical rating score (NRS)
Timepoint [2] 0 0
Baseline to week 36
Secondary outcome [3] 0 0
Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS =4
Timepoint [3] 0 0
Baseline to week 36
Secondary outcome [4] 0 0
Percent change in Bullous Pemphigoid Disease Area Index Activity Score (BPDAI) activity score
Timepoint [4] 0 0
Baseline to week 36
Secondary outcome [5] 0 0
Time to first use of rescue medication
Timepoint [5] 0 0
Up to week 36

Eligibility
Key inclusion criteria
Key

* Patients must have characteristic clinical features of bullous pemphigoid (BP) (eg, urticarial or eczematous or erythematous plaques, bullae, pruritus) at the screening and baseline visits.
* Study participants are required to have a confirmed diagnosis of BP based on histopathology, immunopathology, and serology at the baseline visit, as defined in the protocol.
* Bullous Pemphigoid Disease Area Index (BPDAI) activity score =24 at baseline and screening visits.
* Baseline peak pruritus NRS score for maximum itch intensity =4
* Karnofsky performance status score =50% at the screening visit.

Key
Minimum age
18 Years
Maximum age
90 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Forms of pemphigoid other than classic BP (eg, Brunsting-Perry cicatricial pemphigoid, anti-p200 pemphigoid, epidermolysis bullosa acquisita, or BP with concomitant pemphigus vulgaris)
* Patients who are receiving treatments known to cause or exacerbate BP (eg, angiotensin converting enzyme inhibitors, penicillamine, furosemide, phenacetin, dipeptidyl peptidase 4 inhibitor) who have not been on a stable dose of these medications for at least 4 weeks prior to the screening visit
* Have ever received treatment with an IL-4 or IL-13 antagonist such as dupilumab, tralokinumab, or lebrikizumab.
* Treatment with systemic corticosteroids within 7 days before the baseline visit
* Treatment with topical corticosteroids of medium potency or higher, topical calcineurin inhibitor, or topical crisaborole within 7 days before the baseline visit
* Treatment with non-steroidal immunosuppressive/immunomodulating drug(s) (eg, mycophenolate mofetil, azathioprine, or methotrexate) within 4 weeks before the baseline visit.
* Treatment with BP-directed biologics as follows:
* Any cell-depleting agents including but not limited to rituximab: within 12 months before the baseline visit, or until lymphocyte and CD 19+ lymphocyte count returns to normal, whichever is longer
* Other biologics (such as IL-5 inhibitors benralizumab or mepolizumab): within 5 half-lives (if known) or 16 weeks prior to the baseline visit, whichever is longer
* Intravenous immunoglobulin within 16 weeks prior to the baseline visit

NOTE: Other Protocol Defined Inclusion/Exclusion Criteria Apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/10/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
5/01/2025
Sample size
Target
Accrual to date
Final
106
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Regeneron Study Site - Kogarah
Recruitment hospital [2] 0 0
Regeneron Study Site - Box Hill
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Oregon
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Rhode Island
Country [13] 0 0
United States of America
State/province [13] 0 0
Utah
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
France
State/province [15] 0 0
Bobigny
Country [16] 0 0
France
State/province [16] 0 0
Bordeaux
Country [17] 0 0
France
State/province [17] 0 0
Lille
Country [18] 0 0
France
State/province [18] 0 0
Nice
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
France
State/province [20] 0 0
Rouen Cedex
Country [21] 0 0
Germany
State/province [21] 0 0
North Rhine-Westphal
Country [22] 0 0
Germany
State/province [22] 0 0
Rheinland-Pfalz
Country [23] 0 0
Germany
State/province [23] 0 0
Saxony
Country [24] 0 0
Germany
State/province [24] 0 0
Schleswig-Holstein
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Buxtehude
Country [27] 0 0
Germany
State/province [27] 0 0
Erlangen
Country [28] 0 0
Germany
State/province [28] 0 0
Freiburg
Country [29] 0 0
Germany
State/province [29] 0 0
Magdeburg
Country [30] 0 0
Germany
State/province [30] 0 0
Marburg
Country [31] 0 0
Germany
State/province [31] 0 0
Munich
Country [32] 0 0
Germany
State/province [32] 0 0
Stuttgart
Country [33] 0 0
Israel
State/province [33] 0 0
Afula
Country [34] 0 0
Israel
State/province [34] 0 0
Petah Tikva
Country [35] 0 0
Israel
State/province [35] 0 0
Tel-Aviv
Country [36] 0 0
Japan
State/province [36] 0 0
Hukuoka
Country [37] 0 0
Japan
State/province [37] 0 0
Hirosaki
Country [38] 0 0
Japan
State/province [38] 0 0
Ichinomiya
Country [39] 0 0
Japan
State/province [39] 0 0
Osaka
Country [40] 0 0
Japan
State/province [40] 0 0
Sapporo
Country [41] 0 0
Japan
State/province [41] 0 0
Tokyo
Country [42] 0 0
Poland
State/province [42] 0 0
Malopolskie
Country [43] 0 0
Poland
State/province [43] 0 0
Ossy
Country [44] 0 0
Poland
State/province [44] 0 0
Wroclaw
Country [45] 0 0
Spain
State/province [45] 0 0
Barcelona
Country [46] 0 0
Spain
State/province [46] 0 0
Madrid
Country [47] 0 0
Spain
State/province [47] 0 0
Pamplona
Country [48] 0 0
Taiwan
State/province [48] 0 0
Zhongzheng District
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taoyuan City

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Regeneron Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Sanofi
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trial Management
Address 0 0
Regeneron Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04206553