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History of Changes for Study: NCT05681481
A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)
Latest version (submitted April 7, 2024) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 December 27, 2022 None (earliest Version on record)
2 May 10, 2023 Recruitment Status, Study Status, Contacts/Locations and Oversight
3 July 6, 2023 Contacts/Locations, Study Status and Eligibility
4 August 24, 2023 Study Status and Contacts/Locations
5 September 8, 2023 Study Status
6 September 21, 2023 Contacts/Locations and Study Status
7 October 24, 2023 Contacts/Locations and Study Status
8 November 28, 2023 Contacts/Locations and Study Status
9 January 11, 2024 Contacts/Locations and Study Status
10 February 13, 2024 Study Status and Contacts/Locations
11 April 7, 2024 Contacts/Locations and Study Status
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Study NCT05681481
Submitted Date:  December 27, 2022 (v1)
Open or close this module Study Identification
Unique Protocol ID: ARGX-113-2010
Brief Title: A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid (BALLAD+)
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
Secondary IDs:
Open or close this module Study Status
Record Verification: December 2022
Overall Status: Not yet recruiting
Study Start: March 22, 2023
Primary Completion: November 6, 2025 [Anticipated]
Study Completion: November 6, 2025 [Anticipated]
First Submitted: December 8, 2022
First Submitted that
Met QC Criteria:		 December 27, 2022
First Posted: January 12, 2023 [Actual]
Last Update Submitted that
Met QC Criteria:		 December 27, 2022
Last Update Posted: January 12, 2023 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: argenx
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: No
Open or close this module Study Description
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.
Detailed Description:
Open or close this module Conditions
Conditions: Bullous Pemphigoid
Keywords:
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 3
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 160 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
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
Open or close this module Outcome Measures
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)
Open or close this module Eligibility
Minimum Age: 18 Years
Maximum Age:
Sex: All
Gender Based:
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:
    • Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP
    • Women of childbearing potential (WOCBP) must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP 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
Open or close this module Contacts/Locations
Central Contact Person: Sabine Coppieters, MD
Telephone: 857-350-4834
Email: clinicaltrials@argenx.com
Locations:
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:
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