We are experiencing 4 week turn-around time in review of submissions and resubmissions. We recommend commencing this process concurrently with your ethics submission and allowing at least 8 weeks for registration to be completed from date of first submission. We currently do not have the capacity to expedite reviews.

Note also there are delays to review of updates. We appreciate your patience.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04206553
Ethics application status
Date submitted
18/12/2019
Date registered
20/12/2019
Date last updated
6/04/2021

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) - 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.
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 - 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.
Timepoint [4] 0 0
Baseline to week 36

Eligibility
Key inclusion criteria
Key

- Patients must have 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 histological and serological confirmation of
BP by 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
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Forms of pemphigoid other than classic BP (eg, Brunsting-Perry cicatrial 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 2 weeks before the baseline visit

- Treatment with topical corticosteroids of medium potency or higher, topical
calcineurin inhibitor, or topical crisaborole within 1 week 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: 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/Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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 hospital [3] 0 0
Regeneron Study Site - Parkville
Recruitment hospital [4] 0 0
Regeneron Study Site - East Melbourne
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment postcode(s) [4] 0 0
3002 - East Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Connecticut
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
France
State/province [8] 0 0
Bobigny
Country [9] 0 0
France
State/province [9] 0 0
Bordeaux
Country [10] 0 0
France
State/province [10] 0 0
Chambray Les Tours
Country [11] 0 0
France
State/province [11] 0 0
Le Mans Cedex 9
Country [12] 0 0
France
State/province [12] 0 0
Nice
Country [13] 0 0
France
State/province [13] 0 0
Rouen Cedex
Country [14] 0 0
France
State/province [14] 0 0
Toulouse
Country [15] 0 0
Germany
State/province [15] 0 0
North Rhine-Westphal
Country [16] 0 0
Germany
State/province [16] 0 0
Saxony
Country [17] 0 0
Germany
State/province [17] 0 0
Schleswig-Holstein
Country [18] 0 0
Germany
State/province [18] 0 0
Berlin
Country [19] 0 0
Germany
State/province [19] 0 0
Buxtehude
Country [20] 0 0
Germany
State/province [20] 0 0
Munich
Country [21] 0 0
Germany
State/province [21] 0 0
Stuttgart
Country [22] 0 0
Japan
State/province [22] 0 0
Osaka
Country [23] 0 0
Japan
State/province [23] 0 0
Sapporo
Country [24] 0 0
Japan
State/province [24] 0 0
Tokyo

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
The primary objective of the study is to demonstrate that dupilumab is superior to placebo in
achieving sustained remission off oral corticosteroids (OCS) in patients with bullous
pemphigoid (BP).

The secondary objectives of the study are:

- To evaluate the OCS-sparing effects of dupilumab in patients with BP

- To evaluate the effect of dupilumab on itch in patients with BP

- To evaluate the effects of dupilumab on health-related quality of life measures in
patients with BP

- To evaluate the effect of dupilumab in circulating BP180 and BP230 autoantibody titers

- To assess the safety and tolerability of dupilumab administered to patients with BP

- To characterize the trough concentrations of functional dupilumab over time following
administration of dupilumab in patients with BP

- To assess the immunogenicity of dupilumab in patients with BP over time
Trial website
https://clinicaltrials.gov/show/NCT04206553
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
Clinical Trials Administrator
Address 0 0
Country 0 0
Phone 0 0
844-734-6643
Fax 0 0
Email 0 0
clinicaltrials@regeneron.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04206553