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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05681481




Registration number
NCT05681481
Ethics application status
Date submitted
8/12/2022
Date registered
12/01/2023
Date last updated
15/08/2024

Titles & IDs
Public title
A Phase 3 Study to Evaluate the Long-term Safety, Tolerability and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Scientific 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 ID [1] 0 0
ARGX-113-2010
Universal Trial Number (UTN)
Trial acronym
BALLAD+
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Bullous Pemphigoid 0 0
Condition category
Condition code
Skin 0 0 0 0
Dermatological conditions
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - efgartigimod PH20 SC
Treatment: Drugs - Prednisone

Experimental: efgartigimod PH20 SC - participants receiving efgartigimod PH20 SC on top of Prednisone


Treatment: Other: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Treatment: Drugs: Prednisone
Oral Prednisone

Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events
Timepoint [1] 0 0
Up to 56 weeks
Primary outcome [2] 0 0
Severity of treatment-emergent adverse events
Timepoint [2] 0 0
Up to 56 weeks
Primary outcome [3] 0 0
Incidence of serious adverse events
Timepoint [3] 0 0
Up to 56 weeks
Primary outcome [4] 0 0
Severity of serious adverse events
Timepoint [4] 0 0
Up to 56 weeks
Primary outcome [5] 0 0
Incidence of adverse events of special interest
Timepoint [5] 0 0
Up to 56 weeks
Primary outcome [6] 0 0
Severity of adverse events of special interest
Timepoint [6] 0 0
Up to 56 weeks
Primary outcome [7] 0 0
Rate of treatment discontinuation because of safety concerns
Timepoint [7] 0 0
Up to 56 weeks
Secondary outcome [1] 0 0
Proportion of participants achieving complete remission while off oral corticosteroids for = 8 weeks
Timepoint [1] 0 0
Up to 56 weeks
Secondary outcome [2] 0 0
Proportion of participants achieving complete remission or partial remission while off oral corticosteroids for = 8 weeks
Timepoint [2] 0 0
Up to 56 weeks
Secondary outcome [3] 0 0
Proportion of participants achieving complete remission while on minimal oral corticosteroids therapy for = 8 weeks
Timepoint [3] 0 0
Up to 56 weeks
Secondary outcome [4] 0 0
Proportion of participants achieving complete remission while off both oral corticosteroids and efgartigimod PH20 SC for = 8 weeks
Timepoint [4] 0 0
Up to 56 weeks
Secondary outcome [5] 0 0
Proportion of participants achieving complete remission or partial remission while off both oral corticosteroids and efgartigimod PH20 SC for = 8 weeks
Timepoint [5] 0 0
Up to 56 weeks
Secondary outcome [6] 0 0
Duration of sustained remission
Timepoint [6] 0 0
Up to 56 weeks
Secondary outcome [7] 0 0
Proportion of participants who relapse
Timepoint [7] 0 0
Up to 56 weeks
Secondary outcome [8] 0 0
Time to relapse
Timepoint [8] 0 0
Up to 56 weeks
Secondary outcome [9] 0 0
Incidence of relapse
Timepoint [9] 0 0
Up to 56 weeks
Secondary outcome [10] 0 0
Severity of relapse
Timepoint [10] 0 0
Up to 56 weeks
Secondary outcome [11] 0 0
Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time
Timepoint [11] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [12] 0 0
Bullous Pemphigoid Disease Area Index (BPDAI) activity scores over time
Timepoint [12] 0 0
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.
Secondary outcome [13] 0 0
Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time
Timepoint [13] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [14] 0 0
Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores over time
Timepoint [14] 0 0
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.
Secondary outcome [15] 0 0
Itch Numerical Rating Scale (NRS) over time
Timepoint [15] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [16] 0 0
Itch Numerical Rating Scale (NRS) over time
Timepoint [16] 0 0
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.
Secondary outcome [17] 0 0
Rate of treatment failure
Timepoint [17] 0 0
Up to 56 weeks
Secondary outcome [18] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
Timepoint [18] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
Secondary outcome [19] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index - Aggregate Improvement Score (GTI-AIS) over time
Timepoint [19] 0 0
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.
Secondary outcome [20] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
Timepoint [20] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
Secondary outcome [21] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Cumulative Worsening Score (GTI-CWS) over time
Timepoint [21] 0 0
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.
Secondary outcome [22] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
Timepoint [22] 0 0
For participants not requiring treatment with efgartigimod at rollover: at weeks 0, 24 and 48.
Secondary outcome [23] 0 0
Glucocorticoid Toxicity Index (GTI)-related scores, including the Glucocorticoid Toxicity Index Specific List (GTI-SL) over time
Timepoint [23] 0 0
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.
Secondary outcome [24] 0 0
EQ-5D-5L scores over time
Timepoint [24] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
Secondary outcome [25] 0 0
EQ-5D-5L scores over time
Timepoint [25] 0 0
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.
Secondary outcome [26] 0 0
Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time
Timepoint [26] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
Secondary outcome [27] 0 0
Autoimmune Bullous Disease Quality of Life (ABQoL) scores over time
Timepoint [27] 0 0
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.
Secondary outcome [28] 0 0
Dermatology Life Quality Index (DLQI) scores over time
Timepoint [28] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 24 and 48.
Secondary outcome [29] 0 0
Dermatology Life Quality Index (DLQI) scores over time
Timepoint [29] 0 0
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.
Secondary outcome [30] 0 0
Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
Timepoint [30] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 2, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [31] 0 0
Percent changes from baseline over time for anti-BP180 and anti-BP-230 antibody levels
Timepoint [31] 0 0
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.
Secondary outcome [32] 0 0
Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Timepoint [32] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [33] 0 0
Incidence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Timepoint [33] 0 0
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.
Secondary outcome [34] 0 0
Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Timepoint [34] 0 0
For participants not requiring treatment with efgartigimod PH20 SC at rollover: at weeks 0, 4, 8, 16, 24, 32, 40, 48 and 56.
Secondary outcome [35] 0 0
Prevalence of antidrug antibody(ies) (ADA) against efgartigimod (serum levels)
Timepoint [35] 0 0
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.

Eligibility
Key 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 the study drug and must use one of the contraception methods described in the protocol from signing the ICF until the last dose of the study drug
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 the study drug or 1 of its excipients
* Permanently discontinued IMP in ARGX-113-2009 due to an adverse event (AE) considered related to the study drug and for whom the benefit/risk balance is not considered positive

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Premier Specialists - Kogarah
Recruitment postcode(s) [1] 0 0
2217 - Kogarah
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Michigan
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
Bulgaria
State/province [7] 0 0
Sofia
Country [8] 0 0
China
State/province [8] 0 0
Chengdu
Country [9] 0 0
Croatia
State/province [9] 0 0
Zagreb
Country [10] 0 0
Czechia
State/province [10] 0 0
Praha
Country [11] 0 0
Germany
State/province [11] 0 0
Berlin
Country [12] 0 0
Germany
State/province [12] 0 0
Dresden
Country [13] 0 0
Germany
State/province [13] 0 0
Düsseldorf
Country [14] 0 0
Germany
State/province [14] 0 0
Kiel
Country [15] 0 0
Germany
State/province [15] 0 0
München
Country [16] 0 0
Germany
State/province [16] 0 0
Würzburg
Country [17] 0 0
Greece
State/province [17] 0 0
Athens
Country [18] 0 0
Greece
State/province [18] 0 0
Thessaloníki
Country [19] 0 0
Hungary
State/province [19] 0 0
Budapest
Country [20] 0 0
Israel
State/province [20] 0 0
Ramat Gan
Country [21] 0 0
Italy
State/province [21] 0 0
Catania
Country [22] 0 0
Italy
State/province [22] 0 0
Firenze
Country [23] 0 0
Italy
State/province [23] 0 0
Genova
Country [24] 0 0
Italy
State/province [24] 0 0
Milano
Country [25] 0 0
Italy
State/province [25] 0 0
Pavia
Country [26] 0 0
Italy
State/province [26] 0 0
Roma
Country [27] 0 0
Italy
State/province [27] 0 0
Rome
Country [28] 0 0
Japan
State/province [28] 0 0
Sapporo
Country [29] 0 0
Netherlands
State/province [29] 0 0
Groningen
Country [30] 0 0
Serbia
State/province [30] 0 0
Belgrade
Country [31] 0 0
Slovakia
State/province [31] 0 0
Bratislava
Country [32] 0 0
Slovakia
State/province [32] 0 0
Trnava
Country [33] 0 0
Spain
State/province [33] 0 0
Granada
Country [34] 0 0
Spain
State/province [34] 0 0
Madrid
Country [35] 0 0
Spain
State/province [35] 0 0
Valencia
Country [36] 0 0
United Kingdom
State/province [36] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
argenx
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sabine Coppieters, MD
Address 0 0
Country 0 0
Phone 0 0
857-350-4834
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.