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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: 18 January 2021
Main ID:  EUCTR2019-003107-35-BE
Date of registration: 20/03/2020
Prospective Registration: Yes
Primary sponsor: argenx BVBA
Public title: A study to assess the long-term safety and efficacy of a subcutaneous formulation of efgartigimod in adults with chronic inflammatory demyelinating polyneuropathy (an autoimmune disorder that affects the peripheral nerves)
Scientific title: Open-label Extension of the ARGX-113-1802 Trial to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) - ADHERE+
Date of first enrolment: 04/08/2020
Target sample size: 360
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-003107-35
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): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no
Countries of recruitment
Belgium Bulgaria Canada Czechia Denmark France Georgia Germany
Hungary Israel Italy Japan Latvia Netherlands Poland Romania
Russian Federation Serbia Spain Ukraine United Kingdom United States
Contacts
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: 00329310 3400
Email: regulatory@argenx.com
Affiliation:  argenx BVBA
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: 00329310 3400
Email: regulatory@argenx.com
Affiliation:  argenx BVBA
Key inclusion & exclusion criteria
Inclusion criteria:
1. Ability to understand the requirements of the trial, 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) of this trial.

2. Male or female patient with one of the following options:
- Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or
- Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or
- Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or
- Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment.

3. Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration.

4. Women of childbearing potential must use a highly effective method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP

5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use a condom and his partner must use a highly effective method of contraception (failure rate of less than 1% per year) from baseline to 90 days after the last administration of IMP. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual life style of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post-procedure can be included. In addition, male patients are not allowed to donate sperm from baseline to 90 days after the last administration 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 216
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 144

Exclusion criteria:
1. Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP.

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

3. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or any other reason which could confound the results of the trial or put the patient at undue risk.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Chronic Inflammatory Demyelinating Polyneuropathy
MedDRA version: 20.0 Level: LLT Classification code 10077384 Term: Chronic inflammatory demyelinating polyneuropathy System Organ Class: 100000004852
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
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 long-term safety and tolerability of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20] for subcutaneous [SC] administration)
Primary end point(s): - Incidence of TEAEs and SAEs by system organ class (SOC) and preferred term (PT).
- Incidence of clinically significant laboratory abnormalities.
Secondary Objective: - To determine the long-term efficacy
- To evaluate the immunogenicity (anti-drug antibodies [ADA]) of efgartigimod and rHuPH20
- To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC
- To evaluate the pharmacodynamic (PD) effect of efgartigimod PH20 SC (ie, immunoglobulin G [IgG] levels)
- To evaluate additional patient-reported outcomes (PROs) (including patient-reported quality of life and satisfaction with treatment)
- To explore self-administration of the treatment
Timepoint(s) of evaluation of this end point: TEAEs and SAEs are monitored throughout the entire study duration
Blood sampling for laboratory analysis is taken at every study visit
Secondary Outcome(s)
Secondary end point(s): Efficacy;
Change from baseline over time in the following scores and measurements:
- Adjusted INCAT score;
- MRC Sum score;
- 24-item I-RODS disability scores;
- Mean grip strength assessed by Martin vigorimeter;
- TUG score.
Percentage of patients without clinical deterioration over time, defined by adjusted INCAT increase =1 point compared to baseline.

Immunogenicity;
- Percentage of patients with and titers of binding antibodies (BAb) towards efgartigimod and/or rHuPH20; and the presence of neutralizing antibodies (NAb) against efgartigimod and titers of NAb against rHuPH20.

Pharmacokinetics;
- Efgartigimod serum concentrations over time during the trial.

Pharmacodynamics;
- Changes from baseline over time of serum IgG levels (total and IgG subtypes)

Additional Patient-reported Outcome;
Change from baseline over time in:
- Health-related quality-of-life questionnaire (EQ-5D-5L);
- Brief Pain Inventory – Short Form (BPI-SF);
- 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9);
- Rasch-transformed-Fatigue Severity Scale (RT-FSS);
- Hospital Anxiety and Depression Scale (HADS).
Timepoint(s) of evaluation of this end point: Efficacy, immunogenicity, pharmacokinetic and pharmacodynamic endpoints are assessed at every study visit
Patient report outcomes are assessed at every study visit with the exception of visit 2 (week 4)
Secondary ID(s)
2019-003107-35-DE
ARGX-113-1902
Source(s) of Monetary Support
argenx BVBA
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 04/08/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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