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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: 5 January 2021
Main ID:  EUCTR2019-002101-21-DE
Date of registration: 26/10/2020
Prospective Registration: Yes
Primary sponsor: argenx BVBA
Public title: A long-term study to assess the safety and efficacy of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)
Scientific title: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia - ADVANCE+
Date of first enrolment: 15/12/2020
Target sample size: 156
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-002101-21
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
Austria Belgium Bulgaria Czech Republic France Georgia Germany Hungary
Italy Japan Netherlands Poland Russian Federation Spain Turkey Ukraine
United Kingdom United States
Contacts
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: +32 9 310 3400
Email: regulatory@argenx.com
Affiliation:  argenx BVBA
Name: Regulatory   
Address:  Industriepark Zwijnaarde 7 B-9052 Zwijnaarde Belgium
Telephone: +32 9 310 3400
Email: regulatory@argenx.com
Affiliation:  argenx BVBA
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), and to comply with the trial protocol procedures (including required trial visits).
2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-week trial period.
3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with a FSH of >40 IU/L or are surgically sterilized (ie, women who had a hysterectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
4. Women of childbearing potential should use a highly effective method of contraception (ie, pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month:
• combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
- oral
- intravaginal
- transdermal
• progestogen-only hormonal contraception associated with inhibition of ovulation:
- oral
- injectable
- implantable
• intrauterine device (IUD)
• intrauterine hormone-releasing system
• bilateral tubal occlusion
• vasectomized partner(provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post procedure)
• continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable.
5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, being a condom for male patients and a highly effective form of contraception for the female partner of childbearing potential (same as for female patients described in inclusion criterion 4). Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle 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 during this period from signing of informed consent form, throughout the duration of the trial, and for 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 140
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 16

Exclusion criteria:
1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
4. Use of any other investigational drug or participation in any other investigational trial.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Primary immune thrombocytopenia
MedDRA version: 23.0 Level: LLT Classification code 10050245 Term: Autoimmune thrombocytopenia System Organ Class: 100000004851
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Intervention(s)

Product Name: Efgartigimod
Product Code: ARGX-113
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Efgartigimod
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: 20-

Primary Outcome(s)
Main Objective: To evaluate the long-term safety of efgartigimod in adult patients with primary immune thrombocytopenia (ITP).
Primary end point(s): Frequency and severity of AEs, vital signs, and laboratory assessments.
Secondary Objective: • To evaluate the long-term efficacy of efgartigimod on overall platelet count response.
• To explore the potential for reduction in concurrent ITP therapy.
• To evaluate the effects of efgartigimod treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO).
• To assess the pharmacodynamic (PD) effects of efgartigimod.
• To evaluate the pharmacokinetics (PK) of efgartigimod.
• To assess the immunogenicity of efgartigimod.
Timepoint(s) of evaluation of this end point: Frequency and severity of AEs - at each visit
Vital signs - at baseline visit (week 1), followed by every even week (week 2 to week 52) and Early discontinuation, follow-up 1 and 2, unscheduled visits.
Laboratory assessments - please refer to protocol Table 1 Schedule of Assessments for various laboratory assessments
Secondary Outcome(s)
Secondary end point(s): 1. Extent of disease control defined as the number of cumulative weeks over the planned 52-week treatment period with platelet counts of =50×10^9/L.
2. Percentage of patients with overall platelet count response defined as achieving a platelet count of =50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
3. Mean change from baseline in platelet count at each visit.
4. For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of =50×10^9/L.
5. The number of cumulative weeks over the planned 52-week treatment period with platelet counts of =30×10^9/L and at least 20×10^9/L above baseline.
6. In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the number of cumulative weeks over the planned 52-week treatment period with platelet counts of =30×10^9/L and at least 20×10^9/L above baseline.
7. In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
8. In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
9. Rate of receipt of rescue therapy (rescue per patient per month).
10. Reduction in concurrent ITP therapy.
11. Incidence and severity of the WHO-classified bleeding events.
12. Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) and QoL (SF-36) at planned visits.
13. Incidence of anti-drug antibodies (ADA) to efgartigimod.
14. Pharmacokinetic parameters of efgartigimod: maximum observed serum concentration (Cmax) and serum concentration observed predose (Ctrough).
15. Pharmacodynamics markers: total IgG, IgG isotypes (IgG1, IgG2, IgG3, IgG4).
Timepoint(s) of evaluation of this end point: 1. Over the 52-week treatment period
2. Over the 52-week treatment period
3. At each visit
4. At each visit
5. Over the 52-week treatment period
6. Over the 52-week treatment period
7. Between visit 19 and 24
8. Between visits 17 and 24
9. At each visit
10. At each visit
11. At each visit
12. PRO: Weeks 1, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 47, 49, 52 of Treatment period, Early Discontinuation
QoL: Weeks 1, 9, 17, 25, 33, 41, 49, 52 of Treatment period, Early Discontinuation
13. Weeks 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 of Treatment period, Early Discontinuation, Follow-up 2, Unscheduled Visit
14 and 15. Weeks 1, 5, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 of Treatment period, Early Discontinuation, Follow-up 1/2, Unscheduled Visit

Secondary ID(s)
2019-002101-21-NL
ARGX-113-1803
Source(s) of Monetary Support
argenx BVBA
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 15/12/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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