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A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)

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ClinicalTrials.gov Identifier: NCT05681481
Recruitment Status : Recruiting
First Posted : January 12, 2023
Last Update Posted : September 11, 2023
Sponsor:
Information provided by (Responsible Party):
argenx

Study Description
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Brief Summary:

ARGX-113-2010 is an open-label extension study with the aim to provide supporting evidence that efgartigimod PH20 SC is a safe and effective long-term treatment for bullous pemphigoid (BP), providing symptom control and eventually remission, while also reducing the cumulative exposure to oral corticosteroids (OCS).

All participants who complete the end-of-treatment period (EoTP) visit at week 36 in ARGX-113-2009 will be invited to enroll.

In ARGX-113-2009, participants received efgartigimod PH20 SC or placebo with concurrent OCS, or rescue therapy (without efgartigimod PH20 SC or placebo). Depending on their clinical status at the time of rollover into ARGX-113-2010, participants may stop, continue or initiate efgartigimod PH20 SC treatment. In ARGX-113-2010, participants will stop efgartigimod PH20 SC treatment when they achieve complete remission (CR) or partial remission (PR) while being off other concurrent BP therapy for at least 8 weeks. Participants not in CR or PR while off OCS for ≥8 weeks and not on rescue therapy will either start or continue efgartigimod PH20 SC treatment, while maintaining the treatment allocation of ARGX-113-2009 blinded. Participants may also be retreated with efgartigimod PH20 SC after a relapse. In this study, loading doses of 2000 mg (on day 1 and day 8 of a treatment course) and weekly maintenance doses of 1000 mg will be used.


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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long Term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Actual Study Start Date : March 22, 2023
Estimated Primary Completion Date : January 9, 2026
Estimated Study Completion Date : March 6, 2026

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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events [ Time Frame: Up to 56 weeks ]
    Incidence of treatment-emergent adverse events

  2. Severity of treatment-emergent adverse events [ Time Frame: Up to 56 weeks ]
    Severity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events ( CTCAE) definitions (current version): Grade 1 (mild) to Grade 5 (death related to adverse event)

  3. Incidence of serious adverse events [ Time Frame: Up to 56 weeks ]
    Incidence of serious adverse events

  4. Severity of serious adverse events [ Time Frame: Up to 56 weeks ]
    Severity of serious adverse events

  5. Incidence of adverse events of special interest [ Time Frame: Up to 56 weeks ]
    Incidence of adverse events of special interest

  6. Severity of adverse events of special interest [ Time Frame: Up to 56 weeks ]
    Severity will be assessed using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events ( CTCAE) definitions (current version): Grade 1 (mild) to Grade 5 (death related to adverse event)

  7. Rate of treatment discontinuation because of safety concerns [ Time Frame: Up to 56 weeks ]
    Rate of treatment discontinuation because of safety concerns


Secondary Outcome Measures :
  1. Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]
    Proportion of participants achieving complete remission while off oral corticosteroids for ≥ 8 weeks

  2. Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]
    Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for ≥ 8 weeks

  3. Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]
    Minimal oral corticosteroid therapy is defined as ≤0.10 mg/kg/day of prednisone (or an equivalent dose of another oral corticosteroid)

  4. Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]
    Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks

  5. Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks [ Time Frame: Up to 56 weeks ]
    Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for ≥ 8 weeks

  6. Duration of sustained remission [ Time Frame: Up to 56 weeks ]
    Duration of sustained remission

  7. Proportion of participants who relapse [ Time Frame: Up to 56 weeks ]
    Proportion of participants who relapse

  8. Time to relapse [ Time Frame: Up to 56 weeks ]
    Time to relapse

  9. Incidence of relapse [ Time Frame: Up to 56 weeks ]
    Incidence of relapse

  10. Severity of relapse [ Time Frame: Up to 56 weeks ]
    Severity of relapse will be assessed based on the Bullous Pemphigoid Disease Area Index (BPDAI)

  11. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time

  12. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]
    Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time

  13. Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time

  14. Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]
    Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time

  15. Itch Numerical Rating Scale (NRS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Itch Numerical Rating Scale (NRS) over time

  16. Itch Numerical Rating Scale (NRS) over time [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks until efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]
    Itch Numerical Rating Scale (NRS) over time

  17. Rate of treatment failure [ Time Frame: Up to 56 weeks ]
    Rate of treatment failure

  18. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time

  19. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time

  20. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time

  21. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time

  22. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time [ Time Frame: For participants not requiring treatment with efgartigimod at rollover: at weeks 0, 24 and 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time

  23. Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: weeks 0, 8, every 16 weeks until and after efgartigimod treatment stop and at week 48. ]
    Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time

  24. EQ-5D-5L scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]
    EQ-5D-5L scores over time

  25. EQ-5D-5L scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]
    EQ-5D-5L scores over time

  26. Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]
    Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time

  27. Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]
    Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time

  28. Dermatology Life Quality Index (DLQI) scores over time [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48. ]
    Dermatology Life Quality Index (DLQI) scores over time

  29. Dermatology Life Quality Index (DLQI) scores over time [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 8, every 16 weeks until and after efgartigimod PH20 SC treatment stop and at week 48. ]
    Dermatology Life Quality Index (DLQI) scores over time

  30. Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels

  31. Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels [ Time Frame: For participants continuing/starting efgartigimod PH20 SC treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 4 weeks to efgartigimod stop, every 8 weeks after efgartigimod stop and at weeks 48, 52 and 56. ]
    Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels

  32. Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)

  33. Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 8 weeks until and after efgartigimod PH20 SC stop and at weeks 48, 52 and 56. ]
    Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)

  34. Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56. ]
    Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)

  35. Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels) [ Time Frame: For participants continuing/starting efgartigimod treatment at rollover or relapse: at weeks 0, 2, 4 and 8 and then every 8 weeks until and after efgartigimod PH20 SC stop and at weeks 48, 52 and 56. ]
    Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)


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.


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

Inclusion Criteria:

  • Has completed the week 36 visit of ARGX-113-2009
  • Is capable of providing signed informed consent and complying with protocol requirements
  • Agrees to use contraceptive measures consistent with local regulations and the following: Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP

Exclusion Criteria:

  • Clinically significant disease, recent major surgery (within 3 months of baseline), or intends to have surgery during the study; or any other medical condition that, in the investigator's opinion would confound the results of the study or put the participant at undue risk
  • Known hypersensitivity to IMP or 1 of its excipients
  • Permanently discontinued IMP in ARGX-113-2009 due to an AE considered related to IMP and for whom the benefit/risk balance is not considered positive
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): NCT05681481


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

Locations
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United States, Missouri
Investigator site 6 - US0010098 Active, not recruiting
Saint Louis, Missouri, United States, 63110
Croatia
Investigator site HR3850002 Recruiting
Zagreb, Croatia, 10000
Contact: Sabine Coppieters, MD    857-350-4834    Clinicaltrials@argenx.com   
Germany
Investigator site 4 - DE0490039 Active, not recruiting
Berlin, Germany, 10117
Hungary
Investigator site HU0360023 Recruiting
Budapest, Hungary, 1085
Contact: Sabine Coppieters, MD    857-350-4834    Clinicaltrials@argenx.com   
Netherlands
Investigator site 5 - NL0310015 Active, not recruiting
Groningen, Netherlands, 9713 GZ
Spain
Investigator site ES0340053 Recruiting
Granada, Spain, 18016
Contact: Sabine Coppieters, MD    857-350-4834    Clinicaltrials@argenx.com   
Sponsors and Collaborators
argenx
More Information
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT05681481    
Other Study ID Numbers: ARGX-113-2010
First Posted: January 12, 2023    Key Record Dates
Last Update Posted: September 11, 2023
Last Verified: September 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.: Yes
Additional relevant MeSH terms:
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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