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A Phase 2/3 Study of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD)

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ClinicalTrials.gov Identifier: NCT05267600
Recruitment Status : Recruiting
First Posted : March 4, 2022
Last Update Posted : May 12, 2023
Sponsor:
Information provided by (Responsible Party):
argenx

Study Description
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Brief Summary:

ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global, multicenter, randomized, double-blinded, placebo-controlled study to investigate the efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures (including those assessing participant QoL), PK, and PD of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) and eventually remission while reducing their cumulative exposure to OCS.

study will consist of 2 parts:

  • Part A of the study is a phase 2 evaluation that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP.
  • Part B of the study is a phase 3 evaluation that intends to confirm the results obtained from part A in a separate, larger group of participants with BP.

An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several secondary endpoints, confirm the appropriate sample size for part B of the study, and determine whether the efficacy results observed through week 26 of part A warrant continued study of efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).

Other than differences in main goals, endpoints, and statistical analyses, parts A and B are identical in schedule, structure, assessments, and conduct.


Condition or disease Intervention/treatment Phase
Bullous Pemphigoid Biological: efgartigimod PH20 SC Other: placebo Drug: Prednisone Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Actual Study Start Date : June 9, 2022
Estimated Primary Completion Date : March 2024
Estimated Study Completion Date : April 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus Steroids
Drug Information available for: Prednisone

Arms and Interventions
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Arm Intervention/treatment
Experimental: efgartigimod PH20 SC
participants receiving efgartigimod PH20 SC on top of Prednisone
 Biological: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Drug: Prednisone
Oral Prednisone
Placebo Comparator: placebo PH20 SC
participants receiving placebo PH20 SC on top of Prednisone
 Other: placebo
Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Drug: Prednisone
Oral Prednisone


Outcome Measures
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Primary Outcome Measures :
  1. Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 [ Time Frame: at week 36 ]

Secondary Outcome Measures :
  1. Cumulative dose of oral corticosteroid (OCS) from baseline to week 36 [ Time Frame: up to week 36 ]
  2. Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 [ Time Frame: at week 36 ]
  3. Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 [ Time Frame: at week 36 ]
  4. Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while receiving efgartigimod PH20 SC or placebo at any time through week 36 [ Time Frame: up to week 36 ]
  5. Changes from baseline in the Bullous Pemphigoid Disease Area Index (BPDAI) activity score [ Time Frame: up to week 36 ]
  6. Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been receiving minimal oral corticosteroid (OCS) therapy for ≥8 weeks at week 36 [ Time Frame: at week 36 ]
    Minimal oral corticosteroid (OCS) therapy is defined as ≤0.1 mg/kg/day of prednisone (or an equivalent dose of another OCS).

  7. Time to achieve control of disease activity (CDA) [ Time Frame: up to week 36 ]
  8. Time to achieve complete remission (CR) [ Time Frame: up to week 36 ]
  9. Time to achieve complete remission (CR) while on minimal oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  10. Time to achieve complete remission (CR)/partial remission (PR) while off oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  11. Time to achieve complete remission (CR) while off oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  12. Time to achieve relapse [ Time Frame: up to week 36 ]
  13. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit control of disease activity (CDA) [ Time Frame: up to week 36 ]
  14. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) [ Time Frame: up to week 36 ]
  15. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) while on minimal oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  16. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR)/partial remission (PR) while off oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  17. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit complete remission (CR) while off oral corticosteroid (OCS) therapy for ≥8 weeks [ Time Frame: up to week 36 ]
  18. Cumulative oral corticosteroid (OCS) dose for the participant at the time points when they exhibit relapse [ Time Frame: up to week 36 ]
  19. Proportion of participants who receive rescue therapy before week 36 [ Time Frame: at week 36 ]
  20. Proportion of participants who achieve control of disease activity (CDA) while receiving efgartigimod PH20 SC or placebo and remain free of relapse through week 36 [ Time Frame: up to week 36 ]
  21. Changes from baseline in the 24-hour average itch score from the Itch Numerical Rating Scale (Itch NRS) [ Time Frame: up to week 36 ]
  22. Changes from baseline in the 24-hour worst itch score from the Itch Numerical Rating Scale (Itch NRS) [ Time Frame: up to week 36 ]
  23. Incidence of treatment emergent adverse events (TEAEs) [ Time Frame: up to 46 weeks ]
  24. Severity of treatment emergent adverse events (TEAEs) [ Time Frame: up to 46 weeks ]
  25. Incidence of adverse events of special interest (AESIs) [ Time Frame: up to 46 weeks ]
  26. Severity of adverse events of special interest (AESIs) [ Time Frame: up to 46 weeks ]
  27. Incidence of serious adverse events (SAEs) [ Time Frame: up to 46 weeks ]
  28. Severity of serious adverse events (SAEs) [ Time Frame: up to 46 weeks ]
  29. The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI) [ Time Frame: up to week 36 ]
  30. The Cumulative Worsening Score (CWS) from the Glucocorticoid Toxicity Index (GTI) [ Time Frame: up to week 36 ]
  31. The Glucocorticoid Toxicity Index Specific List (GTI-SL) [ Time Frame: up to week 36 ]
  32. EuroQol 5-Dimension 5-Level (EQ-5D-5L) scores over time [ Time Frame: up to week 36 ]
  33. Dermatology Life Quality Index (DLQI) scores over time [ Time Frame: up to week 36 ]
  34. Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [ Time Frame: up to week 36 ]
  35. Efgartigimod serum concentrations [ Time Frame: up to week 43 ]
  36. Percent change of total IgG serum levels from baseline over time [ Time Frame: up to 46 weeks ]
  37. Percent change of Anti-BP180 and anti-BP230 antibodies from baseline over time [ Time Frame: up to 46 weeks ]
  38. Incidence of Antidrug antibodies (ADA) against efgartigimod (in serum) and antibodies produced against rHuPH20 (in plasma) [ Time Frame: up to 46 weeks ]
  39. Number of participants (or their caregivers) who complete the (self-)administration training at study sites [ Time Frame: up to week 32 ]
  40. Percentage of participants (or their caregivers) who complete the (self-)administration training at study sites [ Time Frame: up to week 32 ]
  41. Number of participants (or their caregivers) who are determined by site staff to be sufficiently competent in (self-)administering efgartigimod PH20 SC [ Time Frame: up to week 32 ]
  42. Percentage of participants (or their caregivers) who are determined by site staff to be sufficiently competent in (self-)administering efgartigimod PH20 SC [ Time Frame: up to week 32 ]
  43. Number of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision [ Time Frame: up to week 35 ]
  44. Percentage of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision [ Time Frame: up to week 35 ]

Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The participant is willing and able to do the following:

    1. understand the requirements of the study
    2. provide written informed consent
    3. comply with the study protocol procedures.
  • The participant is male or female and has reached the age of consent at the time of signing the informed consent form (ICF).
  • Participants have clinical signs of BP.
  • Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and: Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.

The full list of inclusion criteria can be found in the protocol.

Exclusion Criteria:

  • Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs).
  • Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit
  • Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone.
  • Known contraindication to OCS therapy
  • Active, chronic or latent infection at screening
  • Positive COVID-19 test result at screening (testing performed if required per local regulations).
  • History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological finding of prostate cancer
  • Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk or prevent participants from complying with protocol requirements
  • Use of an investigational product within 3 months before the first dose of IMP
  • Previously participated in a clinical study with efgartigimod or currently participating in another interventional clinical study
  • Known hypersensitivity to any of the components of the administered treatments
  • Positive serum test at screening for an active infection: HBV, HCV, HIV
  • Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse as assessed by the investigator
  • Pregnant or lactating females and those who intend to become pregnant during the study
  • Live or live-attenuated vaccine received <4 weeks before baseline visit

The full list of exclusion criteria can be found in the protocol.

Contacts and Locations
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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): NCT05267600


Contacts
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Contact: Sabine Coppieters, MD 857-350-4834 clinicaltrials@argenx.com

Locations
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Sponsors and Collaborators
argenx
More Information
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT05267600    
Other Study ID Numbers: ARGX-113-2009
First Posted: March 4, 2022    Key Record Dates
Last Update Posted: May 12, 2023
Last Verified: May 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Prednisone
Anti-Inflammatory Agents
 Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents