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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04598477




Registration number
NCT04598477
Ethics application status
Date submitted
8/10/2020
Date registered
22/10/2020
Date last updated
24/04/2024

Titles & IDs
Public title
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)
Scientific title
An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus
Secondary ID [1] 0 0
ARGX-113-1905
Universal Trial Number (UTN)
Trial acronym
ADDRESS+
Linked study record

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

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

Experimental: efgartigimod PH20 SC - patients receiving efgartigimod PH20 SC on top of prednisone


Other interventions: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer

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

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
Timepoint [1] 0 0
Up to 60 weeks
Primary outcome [2] 0 0
Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
Timepoint [2] 0 0
Up to 60 weeks
Secondary outcome [1] 0 0
Proportion of Pemphigus Vulgaris (PV) participants who achieve complete clinical remission (CR) on minimal prednisone therapy
Timepoint [1] 0 0
Up to 52 weeks treatment period
Secondary outcome [2] 0 0
Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone dose therapy
Timepoint [2] 0 0
Up to 52 weeks treatment period
Secondary outcome [3] 0 0
Time to Disease Control (DC)
Timepoint [3] 0 0
Up to 60 weeks
Secondary outcome [4] 0 0
Time to complete clinical remission (CR)
Timepoint [4] 0 0
Up to 60 weeks
Secondary outcome [5] 0 0
Time to complete clinical remission (CR) on minimal prednisone therapy
Timepoint [5] 0 0
Up to 60 weeks
Secondary outcome [6] 0 0
Time to complete clinical remission (CR) off prednisone therapy
Timepoint [6] 0 0
Up to 60 weeks
Secondary outcome [7] 0 0
Time to flare
Timepoint [7] 0 0
Up to 60 weeks
Secondary outcome [8] 0 0
Rate of treatment failure
Timepoint [8] 0 0
Up to 60 weeks
Secondary outcome [9] 0 0
Rate of flare
Timepoint [9] 0 0
Up to 60 weeks
Secondary outcome [10] 0 0
Cumulative prednisone dose over the trial
Timepoint [10] 0 0
Up to 52 weeks treatment period
Secondary outcome [11] 0 0
Pemphigus Disease Area Index (PDAI) at each visit
Timepoint [11] 0 0
Up to 52 weeks treatment period
Secondary outcome [12] 0 0
EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
Timepoint [12] 0 0
Up to 52 weeks treatment period
Secondary outcome [13] 0 0
Autoimmune Blister Quality of Life (ABQOL) score
Timepoint [13] 0 0
Up to 52 weeks treatment period
Secondary outcome [14] 0 0
Efgartigimod serum concentrations
Timepoint [14] 0 0
Up to 60 weeks
Secondary outcome [15] 0 0
Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
Timepoint [15] 0 0
Up to 60 weeks
Secondary outcome [16] 0 0
Anti-desmoglein (Dsg) -1 and -3 autoantibodies serum levels
Timepoint [16] 0 0
Up to 60 weeks
Secondary outcome [17] 0 0
Incidence of antidrug antibodies (ADA) against efgartigimod PH20 SC
Timepoint [17] 0 0
Up to 60 weeks
Secondary outcome [18] 0 0
Prevalence of antidrug antibodies (ADA) against efgartigimod PH20 SC
Timepoint [18] 0 0
Up to 60 weeks
Secondary outcome [19] 0 0
Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)
Timepoint [19] 0 0
Up to 52 weeks treatment period
Secondary outcome [20] 0 0
Percentage of participants who performed self-administration
Timepoint [20] 0 0
Up to 52 weeks
Secondary outcome [21] 0 0
Percentage of caregivers who administered the injection to the participant
Timepoint [21] 0 0
Up to 52 weeks
Secondary outcome [22] 0 0
Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
Timepoint [22] 0 0
Up to 52 weeks
Secondary outcome [23] 0 0
Frequency of self- or caregiver-supported administration at home
Timepoint [23] 0 0
Up to 52 weeks

Eligibility
Key inclusion criteria
1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits).
2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.
3. Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and:

1. Male participants:

Male participants must agree to use an acceptable method of contraception as described in the protocol, from signing the ICF until the last dose of the study drug.
2. Female participants

Women of childbearing potential (WOCBP) must:

* have a negative urine pregnancy test at baseline before the IMP can be administered,
* agree to use a highly effective or acceptable contraception method (as described in the protocol), which should be maintained at minimum until after the last dose of IMP
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Pregnant and lactating women and those intending to become pregnant during the trial.
2. Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk.
3. Known hypersensitivity to any of the components of the administered treatments.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/07/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/03/2024
Sample size
Target
Accrual to date
Final
183
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Investigator site 15 - AU0610006 - Sydney
Recruitment hospital [2] 0 0
Investigator site 11 - AU0610007 - Parkville
Recruitment hospital [3] 0 0
Investigator site 92 - AU0610013 - Melbourne
Recruitment postcode(s) [1] 0 0
2217 - Sydney
Recruitment postcode(s) [2] 0 0
3050 - Parkville
Recruitment postcode(s) [3] 0 0
3065 - Melbourne
Recruitment outside Australia
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United States of America
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Alabama
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Arizona
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California
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Minnesota
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Missouri
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New York
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North Carolina
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Ohio
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Pennsylvania
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Texas
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United States of America
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Virginia
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Bulgaria
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Pleven
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Bulgaria
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Plovdiv
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Bulgaria
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Sofia
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Beijing
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Chengdu
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Chongqing
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Fuzhou
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Guangzhou
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Guanzhou
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Wuhan
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Zhengzhou
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Bobigny
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Rouen
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Lübeck
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Ulm
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Athens
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Chaïdári
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Debrecen
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Pécs
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Hungary
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Szeged
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India
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Ahmedabad
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India
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Chandigarh
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Lucknow
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Nagpur
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Tel Aviv
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Catania
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Firenze
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Roma
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Lódz
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Bucharest
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Cluj-Napoca
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Romania
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Iasi
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Belgrade
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Niš
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Novi Sad
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Barcelona
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Granada
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Sevilla
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Gaziantep
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Istanbul
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Ukraine
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Dnipro
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Ukraine
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Ivano-Frankivs'k
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Ukraine
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Kyiv
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Ukraine
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Lviv
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Ukraine
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Zaporizhzhia
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United Kingdom
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Birmingham
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Bristol
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United Kingdom
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Southampton

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

Ethics approval
Ethics application status

Summary
Brief summary
This is a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provides extension of efgartigimod PH20 SC treatment and retreatment options for participants who have been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and the first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to placebo arm in trial ARGX-113-1904. Trial ARGX-113-1905 evaluates ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CRmin, and the ability to treat flare; and assess patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial.

Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration
Trial website
https://clinicaltrials.gov/study/NCT04598477
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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Email 0 0
Contact person for public queries
Name 0 0
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Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04598477