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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 8 February 2021
Main ID:  NCT04598477
Date of registration: 08/10/2020
Prospective Registration: Yes
Primary sponsor: argenx
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) ADDRESS+
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
Date of first enrolment: February 2021
Target sample size: 150
Recruitment status: Not yet recruiting
URL:  https://clinicaltrials.gov/show/NCT04598477
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 3
Countries of recruitment
Bulgaria United States
Contacts
Name:     Antonio Guglietta, MD
Address: 
Telephone: 857-350-4834
Email: clinicaltrials@argenx.com
Affiliation: 
Key inclusion & exclusion criteria

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 patient participated in trial ARGX-113-1904 and completed the study or has the
defined criteria for rollover.

3. Women of childbearing potential:

1. Must have a negative urine pregnancy test at baseline before trial medication can
be administered.

2. Must be on a stable regimen for at least 1 month of at least one highly effective
method of contraception (i.e., failure rate of less than 1% per year) during the
trial and for 90 days after the last administration of IMP.

4. Non-sterilized male patients who are sexually active with a female partner of
childbearing potential must use effective contraception from first administration of
IMP through 90 days after the last administration of the IMP. Male patients practicing
true sexual abstinence (as consistent with preferred and usual life style) can be
included. Sterilized male patients who have had a vasectomy and with documented
absence of sperm post-procedure can be included. Male patients are not allowed to
donate sperm from first administration of IMP through 90 days after the last dose of
IMP.

Exclusion Criteria:

1. Pregnant and lactating women and those intending to become pregnant during the trial
or within 90 days after the last administration of IMP.

2. Patients with clinical evidence of other significant serious disease or patients 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 patient at undue risk.

3. Known hypersensitivity to any of the components of the administered treatments.



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All
Health Condition(s) or Problem(s) studied
Pemphigus Foliaceus
Pemphigus Vulgaris
Intervention(s)
Biological: efgartigimod PH20 SC
Drug: prednisone
Primary Outcome(s)
Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) [Time Frame: Up to 60 weeks]
Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) [Time Frame: Up to 60 weeks]
Secondary Outcome(s)
Anti-Desmoglein -1 and -3 autoantibodies serum levels [Time Frame: Up to 60 weeks]
Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and recombinant human hyaluronidase PH20 (plasma levels) [Time Frame: Up to 60 weeks]
Autoimmune Blister Quality of Life (ABQOL) score [Time Frame: Up to 52 weeks treatment period]
Cumulative prednisone dose over the trial [Time Frame: Up to 52 weeks treatment period]
Efgartigimod serum concentrations [Time Frame: Up to 60 weeks]
EuroQol 5-Dimension 5-Level (EQ-5D-5L) score [Time Frame: Up to 52 weeks treatment period]
Pemphigus Disease Area Index (PDAI) at each visit [Time Frame: Up to 52 weeks treatment period]
Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) patients who achieve complete clinical remission (CR) on minimal prednisone dose therapy [Time Frame: Up to 52 weeks treatment period]
Proportion of Pemphigus Vulgaris (PV) patients who achieve complete clinical remission (CR) on minimal prednisone therapy [Time Frame: Up to 52 weeks treatment period]
Rate of flare [Time Frame: Up to 60 weeks]
Rate of treatment failure [Time Frame: Up to 60 weeks]
Time to complete clinical remission (CR) [Time Frame: Up to 60 weeks]
Time to complete clinical remission (CR) off prednisone therapy [Time Frame: Up to 60 weeks]
Time to complete clinical remission (CR) on minimal prednisone therapy [Time Frame: Up to 60 weeks]
Time to Disease Control (DC) [Time Frame: Up to 60 weeks]
Time to flare [Time Frame: Up to 60 weeks]
Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels [Time Frame: Up to 60 weeks]
Secondary ID(s)
ARGX-113-1905
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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