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A Study to Evaluate the Efficacy and Safety of Dupilumab in Adult Patients With Bullous Pemphigoid (LIBERTY-BP)

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ClinicalTrials.gov Identifier: NCT04206553
Recruitment Status : Recruiting
First Posted : December 20, 2019
Last Update Posted : February 23, 2021
Sponsor:
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

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

Condition or disease Intervention/treatment Phase
Bullous Pemphigoid Drug: dupilumab Drug: Matching Placebo Drug: Oral corticosteroids (OCS) Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 98 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 28, 2020
Estimated Primary Completion Date : February 27, 2023
Estimated Study Completion Date : September 15, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus
Drug Information available for: Dupilumab

Arm Intervention/treatment
Experimental: dupilumab Drug: dupilumab
Loading dose administered subcutaneous (SC), followed by SC once every 2 weeks (Q2W) dosing.
Other Names:
  • Dupixent®
  • REGN668
  • SAR231893

Drug: Oral corticosteroids (OCS)
Prednisone or prednisolone per standard of care to obtain control of disease activity.

Experimental: Matching placebo Drug: Matching Placebo
Matching dupilumab without active substance

Drug: Oral corticosteroids (OCS)
Prednisone or prednisolone per standard of care to obtain control of disease activity.




Primary Outcome Measures :
  1. Proportion of patients achieving sustained remission [ Time Frame: Week 36 ]

Secondary Outcome Measures :
  1. Total cumulative dose of oral corticosteroids (OCS) [ Time Frame: Baseline to week 36 ]
  2. Percent change in weekly average of daily peak pruritus numerical rating score (NRS) [ Time Frame: Baseline to week 36 ]
    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.

  3. Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 36 ]
  4. Percent change in Bullous Pemphigoid Disease Area Index Activity Score (BPDAI) activity score [ Time Frame: Baseline to week 36 ]
    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.


Other Outcome Measures:
  1. Duration of complete remission while not requiring OCS [ Time Frame: Baseline to week 36 ]
  2. Proportion of patients who do not achieve control of disease activity or who relapse after achieving control of disease activity [ Time Frame: Baseline to week 36 ]
    Note: control of disease activity is defined when new lesions cease to form and existing lesions begin to heal

  3. Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75%, and 90% [ Time Frame: Baseline to week 36 ]
  4. Change in autoimmune bullous disease quality of life (ABQOL) [ Time Frame: Baseline to week 36 ]
    ABQOL questionnaire consists of 17 items, which encompass physical burden of the disease, psychiatric effects, and effects on daily life functioning. Each question ranges from 0 to 3 points, with higher scores indicating poorer quality of life. The maximum ABQOL score is 51.

  5. Change in percent body surface area (BSA) of BP involvement [ Time Frame: Baseline to week 36 ]
  6. Change in BP180 autoantibody (IgG) titers [ Time Frame: Baseline to week 36 ]
  7. Change in BP230 autoantibody (IgG) titers [ Time Frame: Baseline to week 36 ]
  8. Proportion of patients with sustained remission [ Time Frame: Week 52 ]
  9. Total cumulative dose of OCS [ Time Frame: Baseline to week 52 ]
  10. Duration of complete remission while not requiring OCS [ Time Frame: Up to week 52 ]
  11. Proportion of patients who do not achieve control of disease activity or who relapse after achieving control of disease activity [ Time Frame: Up to week 52 ]
  12. Percent change in weekly average of daily peak pruritus NRS [ Time Frame: Baseline to week 52 ]
  13. Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 52 ]
  14. Percent change in BPDAI activity score [ Time Frame: Baseline to week 52 ]
  15. Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75% and 90% [ Time Frame: Baseline to week 52 ]
  16. Change in ABQOL [ Time Frame: Baseline to week 52 ]
  17. Change in percent BSA of BP involvement [ Time Frame: Baseline to week 52 ]
  18. Change in BP180 autoantibody (IgG) titers [ Time Frame: Baseline to week 52 ]
  19. Change in BP230 autoantibody (IgG) titers [ Time Frame: Baseline to week 52 ]
  20. Proportion of patients in complete remission and off OCS [ Time Frame: Week 16 ]
  21. Percent change in BPDAI activity score [ Time Frame: Baseline to week 16 ]
  22. Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75%, and 90% [ Time Frame: Baseline to week 16 ]
  23. Percent change in weekly average of daily peak pruritus NRS [ Time Frame: Baseline to week 16 ]
  24. Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 16 ]
  25. Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Baseline to week 64 ]
  26. Incidence of treatment-emergent serious adverse events (SAEs) [ Time Frame: Baseline to week 64 ]
  27. Incidence of adverse events of special interest (AESIs) [ Time Frame: Baseline to week 64 ]
  28. Concentrations of functional dupilumab in serum [ Time Frame: Baseline to week 64 ]
  29. Incidence of treatment-emergent anti-drug antibody (ADA) responses and titer [ Time Frame: Baseline to week 64 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • 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 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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04206553


Contacts
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Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

Locations
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United States, Connecticut
Regeneron Study Site Recruiting
Farmington, Connecticut, United States, 06030
United States, Florida
Regeneron Study Site Recruiting
Miami, Florida, United States, 33135
United States, Georgia
Regeneron Study Site Recruiting
Macon, Georgia, United States, 31217
United States, Maryland
Regeneron Study Site Recruiting
Rockville, Maryland, United States, 20850
United States, Massachusetts
Regeneron Study Site Recruiting
Boston, Massachusetts, United States, 02116
United States, Oregon
Regeneron Study Site Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Regeneron Study Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Regeneron Study Site Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Australia, New South Wales
Regeneron Study Site Recruiting
Kogarah, New South Wales, Australia, 2217
Australia, Victoria
Regeneron Study Site Recruiting
Box Hill, Victoria, Australia, 3128
Regeneron Study Site Recruiting
Parkville, Victoria, Australia, 3050
Australia
Regeneron Study Site Recruiting
East Melbourne, Australia, 3002
France
Regeneron Study Site Recruiting
Bobigny, France, 93009
Regeneron Study Site Recruiting
Bordeaux, France, 33000
Regeneron Study Site Recruiting
Chambray Les Tours, France, 37171
Regeneron Study Site Recruiting
Le Mans Cedex 9, France, 72037
Regeneron Study Site Recruiting
Nice, France, 06200
Regeneron Study Site Recruiting
Rouen Cedex, France, 76031
Regeneron Study Site Recruiting
Toulouse, France, 31000
Germany
Regeneron Study Site Recruiting
Muenster, North Rhine-Westphal, Germany, 48149
Regeneron Study Site Recruiting
Dresden, Saxony, Germany, 01307
Regeneron Study Site Recruiting
Luebeck, Schleswig-Holstein, Germany, 23538
Regeneron Study Site Recruiting
Berlin, Germany, 10117
Regeneron Study Site Recruiting
Berlin, Germany, 13055
Regeneron Study Site Recruiting
Buxtehude, Germany, 21614
Regeneron Study Site Recruiting
Munich, Germany, 80337
Regeneron Study Site Recruiting
Stuttgart, Germany, 70178
Japan
Regeneron Study Site Recruiting
Osaka, Japan, 545-8586
Regeneron Study Site Recruiting
Sapporo, Japan, 060-8648
Regeneron Study Site Recruiting
Tokyo, Japan, 160-8582
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
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Study Director: Clinical Trial Management Regeneron Pharmaceuticals
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Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04206553    
Other Study ID Numbers: R668-BP-1902
2019-003520-20 ( EudraCT Number )
First Posted: December 20, 2019    Key Record Dates
Last Update Posted: February 23, 2021
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: All individual patient data (IPD) that underlie publicly available results will be considered for sharing
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Analytic Code
Time Frame: 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.
Access Criteria: 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).
URL: https://vivli.org/

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases