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 |
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Recruitment Status :
Recruiting
First Posted : December 20, 2019
Last Update Posted : May 24, 2023
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Sponsor:
Regeneron Pharmaceuticals
Collaborator:
Sanofi
Information provided by (Responsible Party):
Regeneron Pharmaceuticals
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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 |
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
| 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 : | October 16, 2024 |
| Estimated Study Completion Date : | January 7, 2025 |
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:
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 :
- Proportion of patients achieving sustained remission [ Time Frame: Week 36 ]
Secondary Outcome Measures :
- Total cumulative dose of oral corticosteroids (OCS) [ Time Frame: Baseline to week 36 ]
- 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.
- Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 36 ]
- 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:
- Duration of complete remission while not requiring OCS [ Time Frame: Baseline to week 36 ]
- 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
- Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75%, and 90% [ Time Frame: Baseline to week 36 ]
- 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.
- Change in percent body surface area (BSA) of BP involvement [ Time Frame: Baseline to week 36 ]
- Change in BP180 autoantibody (IgG) titers [ Time Frame: Baseline to week 36 ]
- Change in BP230 autoantibody (IgG) titers [ Time Frame: Baseline to week 36 ]
- Proportion of patients with sustained remission [ Time Frame: Week 52 ]
- Total cumulative dose of OCS [ Time Frame: Baseline to week 52 ]
- Duration of complete remission while not requiring OCS [ Time Frame: Up to week 52 ]
- 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 ]
- Percent change in weekly average of daily peak pruritus NRS [ Time Frame: Baseline to week 52 ]
- Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 52 ]
- Percent change in BPDAI activity score [ Time Frame: Baseline to week 52 ]
- Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75% and 90% [ Time Frame: Baseline to week 52 ]
- Change in ABQOL [ Time Frame: Baseline to week 52 ]
- Change in percent BSA of BP involvement [ Time Frame: Baseline to week 52 ]
- Change in BP180 autoantibody (IgG) titers [ Time Frame: Baseline to week 52 ]
- Change in BP230 autoantibody (IgG) titers [ Time Frame: Baseline to week 52 ]
- Proportion of patients in complete remission and off OCS [ Time Frame: Week 16 ]
- Percent change in BPDAI activity score [ Time Frame: Baseline to week 16 ]
- Proportion of patients who achieve a reduction in BPDAI activity score of at least 50%, 75%, and 90% [ Time Frame: Baseline to week 16 ]
- Percent change in weekly average of daily peak pruritus NRS [ Time Frame: Baseline to week 16 ]
- Proportion of patients with improvement (reduction) of weekly average of daily peak pruritus NRS ≥4 [ Time Frame: Baseline to week 16 ]
- Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Baseline to week 64 ]
- Incidence of treatment-emergent serious adverse events (SAEs) [ Time Frame: Baseline to week 64 ]
- Incidence of adverse events of special interest (AESIs) [ Time Frame: Baseline to week 64 ]
- Concentrations of functional dupilumab in serum [ Time Frame: Baseline to week 64 ]
- 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 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 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
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
| Contact: Clinical Trials Administrator | 844-734-6643 | clinicaltrials@regeneron.com |
Locations
Show 56 study locations
Sponsors and Collaborators
Regeneron Pharmaceuticals
Sanofi
Investigators
| Study Director: | Clinical Trial Management | Regeneron Pharmaceuticals |
| 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: | May 24, 2023 |
| Last Verified: | April 2023 |
| 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/ |
| 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 |