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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04206553
Registration number
NCT04206553
Ethics application status
Date submitted
18/12/2019
Date registered
20/12/2019
Date last updated
1/05/2024
Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid
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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
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Secondary ID [1]
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2019-003520-20
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Secondary ID [2]
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R668-BP-1902
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Universal Trial Number (UTN)
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Trial acronym
LIBERTY-BP
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Bullous Pemphigoid
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Condition category
Condition code
Skin
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Dermatological conditions
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional(has expanded access)
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Proportion of patients achieving sustained remission
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Assessment method [1]
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Timepoint [1]
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Week 36
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Secondary outcome [1]
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Total cumulative dose of oral corticosteroids (OCS)
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Assessment method [1]
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Timepoint [1]
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Baseline to week 36
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Secondary outcome [2]
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Percent change in weekly average of daily peak pruritus numerical rating score (NRS)
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Assessment method [2]
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Individual NRS used to rate the intensity of pruritus using an 11-point scale (0 to 10) in which 0 indicates no itch while 10 indicates worst itch possible.
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Timepoint [2]
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Baseline to week 36
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Secondary outcome [3]
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Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS =4
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Assessment method [3]
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Timepoint [3]
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Baseline to week 36
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Secondary outcome [4]
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Percent change in Bullous Pemphigoid Disease Area Index Activity Score (BPDAI) activity score
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Assessment method [4]
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BPDAI activity score is the arithmetic sum of 3 subcomponents: cutaneous blisters/erosions, cutaneous urticaria/erythema, and mucosal blisters/erosions. Scores can range from 0 to 360 for BPDAI total activity (maximum 240 for total skin activity and 120 for mucosal activity), with higher scores indicating greater disease activity.
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Timepoint [4]
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Baseline to week 36
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Secondary outcome [5]
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Time to first use of rescue medication
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Assessment method [5]
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Timepoint [5]
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Up to week 36
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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.
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Minimum age
18
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Maximum age
90
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2/Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
28/10/2020
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
21/01/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
98
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Regeneron Study Site - Kogarah
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Recruitment hospital [2]
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Regeneron Study Site - Box Hill
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Recruitment postcode(s) [1]
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2217 - Kogarah
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Recruitment postcode(s) [2]
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3128 - Box Hill
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Arizona
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California
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Connecticut
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Florida
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Iowa
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Massachusetts
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Michigan
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Virginia
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France
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Bobigny
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France
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Bordeaux
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France
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Lille
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France
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Nice
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France
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Paris
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Stuttgart
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Israel
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Afula
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Israel
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Petah Tikva
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Israel
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Hukuoka
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Hirosaki
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Ichinomiya
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Osaka
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Japan
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Sapporo
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Japan
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Tokyo
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Poland
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Malopolskie
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Poland
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Ossy
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Wroclaw
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Barcelona
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Madrid
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Pamplona
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Taiwan
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Taoyuan City
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Regeneron Pharmaceuticals
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Address
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Sanofi
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Address [1]
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Ethics approval
Ethics application status
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Summary
Brief summary
The main purpose of this study is to investigate whether dupilumab is effective and safe for
the treatment of bullous pemphigoid. Dupilumab is a type of drug called a "monoclonal
antibody". An antibody is a special kind of protein that the immune (defense) system normally
makes to fight bacteria and viruses. Bullous pemphigoid is an autoimmune subepidermal
blistering disease, predominately affecting the elderly (typical onset after age 60).
The study is looking at several other research questions, including:
- Side effects that may be experienced by people taking dupilumab
- How dupilumab works in the body and affects the body
- How dupilumab affects quality of life
- How much dupilumab is present in the blood
- To see if dupilumab works to wean the patient off oral corticosteroids
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04206553
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Trial Management
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Address
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Regeneron Pharmaceuticals
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04206553
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