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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
30/03/2025
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)
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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
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Secondary ID [1]
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ARGX-113-1905
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Universal Trial Number (UTN)
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Trial acronym
ADDRESS+
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pemphigus Vulgaris
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0
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Pemphigus Foliaceus
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Condition category
Condition code
Skin
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0
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0
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Dermatological conditions
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Skin
0
0
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0
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Other skin conditions
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Inflammatory and Immune System
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0
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0
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Other - efgartigimod PH20 SC
Treatment: Drugs - prednisone
Experimental: efgartigimod PH20 SC - patients receiving efgartigimod PH20 SC on top of prednisone
Treatment: Other: efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
Treatment: Drugs: prednisone
Oral prednisone tablets
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Intervention code [1]
0
0
Treatment: Other
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Intervention code [2]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)
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Assessment method [1]
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Incidence rates were calculated as 100 × n/PYFU. PYFU=participant-years of follow-up. The safety data sets includes participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF).
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Timepoint [1]
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Up to 60 weeks
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Secondary outcome [1]
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Proportion of Participants With Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Who Achieve CRmin
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Assessment method [1]
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CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at =10 mg/day for at least 8 weeks.
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Timepoint [1]
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Up to 60 weeks
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Secondary outcome [2]
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Proportion of Participants With Pemphigus Vulgaris (PV) Who Achieve CRmin
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Assessment method [2]
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CRmin (complete clinical remission on minimal prednisone therapy) defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at =10 mg/day for at least 8 weeks.
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Timepoint [2]
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Up to 60 weeks
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Secondary outcome [3]
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Time to DC in Participants With PV and PF
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Assessment method [3]
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Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions
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Timepoint [3]
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Up to 52 weeks
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Secondary outcome [4]
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Time to CR in Participants With PV and PF
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Assessment method [4]
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CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions
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Timepoint [4]
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Up to 52 weeks
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Secondary outcome [5]
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Time to CRmin in Participants With PV and PF
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Assessment method [5]
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CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at =10 mg/day for at least 8 weeks.
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Timepoint [5]
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Up to 52 weeks
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Secondary outcome [6]
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Time to CRoff in Participants With PV and PF
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Assessment method [6]
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Complete remission off therapy (CRoff) is defined as the absence of new and established lesions completely healed while the patient is receiving no prednisone therapy for at least 8 weeks.
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Timepoint [6]
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Up to 52 weeks
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Secondary outcome [7]
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Time to Flare After CRmin in Participants With PV and PF
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Assessment method [7]
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CRmin defined as defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at =10 mg/day for at least 8 weeks.
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Timepoint [7]
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Up to 52 weeks
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Secondary outcome [8]
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Rate of Treatment Failure in Participants With PV and PF
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Assessment method [8]
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The absence of DC with oral prednisone 1.5 mg/kg/day for a minimum of 3 weeks, or absence of DC due to prednisone-related SAE, or flare before CRmin resulting in withdrawal of the participant.
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Timepoint [8]
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Up to 52 weeks
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Secondary outcome [9]
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Number of Flares in Participants With PV and PF
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Assessment method [9]
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A flare is defined as the appearance of 3 or more new lesions in a 4-week period that do not heal spontaneously within 1 week or the extension, of established lesions in a participant who had achieved DC.
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Timepoint [9]
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Up to 60 weeks
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Secondary outcome [10]
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Normalized Cumulative Prednisone Dose in Participants With PV and PF
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Assessment method [10]
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Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study
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Timepoint [10]
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Up to 60 weeks
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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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
15/07/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
25/03/2024
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Sample size
Target
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Accrual to date
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Final
183
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Investigator site 15 - AU0610006 - Sydney
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Investigator site 11 - AU0610007 - Parkville
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Investigator site 92 - AU0610013 - Melbourne
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2217 - Sydney
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3050 - Parkville
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3065 - Melbourne
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Funding & Sponsors
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Name
argenx
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Ethics approval
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Summary
Brief summary
This was 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 provided extension of efgartigimod PH20 SC treatment and retreatment options for participants who had been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to the placebo arm in trial ARGX-113-1904. The participants could also receive concomitant prednisone therapy. Investigators could increase or decrease the prednisone dose based on protocol-specified criteria. Trial ARGX-113-1905 evaluated the 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 CR or CRmin, and the ability to treat flare; it also assessed 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
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Trial website
https://clinicaltrials.gov/study/NCT04598477
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Contacts
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No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/77/NCT04598477/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/77/NCT04598477/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04598477
Download to PDF