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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
Active, not recruiting
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
21/01/2025
Actual
Sample size
Target
Accrual to date
Final
98
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
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Iowa
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United States of America
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Massachusetts
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United States of America
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Michigan
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United States of America
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North Carolina
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United States of America
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Oregon
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United States of America
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Pennsylvania
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United States of America
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Rhode Island
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United States of America
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Utah
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United States of America
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Virginia
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France
State/province [15] 0 0
Bobigny
Country [16] 0 0
France
State/province [16] 0 0
Bordeaux
Country [17] 0 0
France
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Lille
Country [18] 0 0
France
State/province [18] 0 0
Nice
Country [19] 0 0
France
State/province [19] 0 0
Paris
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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
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Ossy
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Poland
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Wroclaw
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Spain
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Barcelona
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Spain
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Madrid
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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
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
All individual patient data (IPD) that underlie publicly available results will be considered for sharing

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR), Analytic code
When will data be available (start and end dates)?
Individual anonymized participant data will be considered for sharing once the indication has been approved by a regulatory body, if there is legal authority to share the data and there is not a reasonable likelihood of participant re-identification.
Available to whom?
Qualified researchers may request access to anonymized patient level data or aggregate study data when Regeneron has received marketing authorization from major health authorities (eg, FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc) for the product and indication, has the legal authority to share the data, and has made the study results publicly available (eg, scientific publication, scientific conference, clinical trial registry).
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/


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