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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: EUCTR
Last refreshed on: 23 November 2020
Main ID:  EUCTR2020-002917-16-HU
Date of registration: 28/09/2020
Prospective Registration: Yes
Primary sponsor: argenx BV
Public title: A study to assess the 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 - ADDRESS+
Date of first enrolment: 17/11/2020
Target sample size: 150
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-002917-16
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
Number of treatment arms in the trial: 1
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Bulgaria France Georgia Germany Greece Hungary India
Israel Italy Japan Poland Romania Russian Federation Spain Turkey
Ukraine United Kingdom United States
Contacts
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: 003293103400
Email: regulatory@argenx.com
Affiliation:  argenx BV
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: 003293103400
Email: regulatory@argenx.com
Affiliation:  argenx BV
Key inclusion & exclusion criteria
Inclusion criteria:
Participants must meet all of the following 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:
a. Must have a negative urine pregnancy test at baseline before trial medication can be administered.
b. Must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 105
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 45

Exclusion criteria:
Participants are excluded from the trial if any of the following criteria apply:
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:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Pemphigus Vulgaris or Pemphigus Foliaceus
MedDRA version: 20.0 Level: LLT Classification code 10052802 Term: Pemphigus vulgaris System Organ Class: 100000004858
MedDRA version: 20.0 Level: LLT Classification code 10057069 Term: Pemphigus foliaceus System Organ Class: 100000004858
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Intervention(s)

Product Name: Efgartigimod PH20 SC
Pharmaceutical Form: Solution for injection
INN or Proposed INN: EFGARTIGIMOD ALFA
CAS Number: 1821402-21-4
Current Sponsor code: ARGX-113
Other descriptive name: ARGX-113
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 165-

Primary Outcome(s)
Main Objective: To assess the safety of extended treatment and re-treatment with efgartigimod PH20 SC in patients with PV or PF
Primary end point(s): 1. Incidence and severity of treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), and serious adverse events (SAEs) by System Organ Class (SOC) and Preferred Term (PT)
2. Vital signs, physical examination, electrocardiogram (ECG), and clinical laboratory safety evaluations
Secondary Objective: To evaluate the efficacy of efgartigimod PH20 SC treatment in PV and PF
To evaluate the effects of efgartigimod PH20 SC on quality of life (QoL) in patients with PV or PF
To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC in patients with PV or PF
To evaluate the PD of efgartigimod PH20 SC in patients with PV or PF
To evaluate the immunogenicity of efgartigimod PH20 SC in patients with PV or PF
Timepoint(s) of evaluation of this end point: Up to 60 weeks
Secondary Outcome(s)
Secondary end point(s): 1. Proportion of PV patients who achieve CR on minimal prednisone therapy
2. Proportion of PV and PF patients who achieve CR on minimal prednisone therapy in patients with PV and PF 3.
3. Time to DC
4. Time to CR
5. Time to CR on minimal prednisone therapy
6. Time to CR off therapy
7. Time to flare
8. Rate of treatment failure
9. Rate of flare
10. Cumulative prednisone dose over the trial
11. Pemphigus Disease Area Index (PDAI) at each visit
12. EuroQol 5-Dimension 5-Level (EQ-5D-5L) score
13. Autoimmune Bullous Disease Quality of Life (ABQOL) score
14. Efgartigimod serum concentrations
15. Total IgG and subtype (IgG1, IgG2, IgG3, IgG4) serum levels
16. Anti-Dsg-1 and -3 autoantibodies serum levels
17. Anti-drug antibodies (ADAs) to efgartigimod (serum levels) and rHuPH20 (plasma levels)


Timepoint(s) of evaluation of this end point: 1+2: up to 52 weeks treatment period
3,4,5,6,7,8,9: up to 60 weeks
10,11,12,13: up to 52 weeks treatment period
14,15,16, 17: up to 60 weeks
Secondary ID(s)
ARGX-113-1905
Source(s) of Monetary Support
argenx BV
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 12/10/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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