Study to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

• Register: EUCTR
• Last refreshed on: 3 December 2024
• Main ID: EUCTR2020-004033-20-IT
• Date of registration: 17/08/2021
• Prospective Registration: No
• Primary sponsor: ARGENX BV
• Public title: Study to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
• Scientific title: A Phase 3, Multicenter, Open-Label, Long-Term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia - ADVANCE SC+
• Date of first enrolment: 23/07/2021
• Target sample size: 156
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-004033-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes 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: yes Other specify the comparator: not applicable 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

Argentina, Australia, Bulgaria, Chile, China, Colombia, Denmark, France, Georgia, Germany, Greece, Ireland, Israel, Italy, Japan, Jordan, Korea, Republic of, Latvia, Mexico, New Zealand, Norway, Peru, Poland, Portugal, Romania, Russian Federation, Serbia, South Africa, Spain, Taiwan, Thailand, Tunisia, Turkey, United Kingdom, United States

Contacts

• Name: Regulatory
• Affiliation: argenx BV

• Name: Regulatory
• Affiliation: argenx BV

Key inclusion & exclusion criteria

Inclusion criteria:
Participants are eligible to be included in the trial only if all of the following criteria apply:
1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits).
2. Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period. Note: If a participant has had an SAE during the ARGX-113-2004 trial, his/her eligibility should be evaluated by the investigator and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case.
3. Women of childbearing potential:
• As defined in Woman of Childbearing Potential in the protocol, women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication can be administered
• Must be on a stable regimen for at least 1 month of a highly effective or acceptable method of contraception (see Female Contraception in the protocol) during the trial and for 90 days after the last administration of efgartigimod PH20 SC
4. Non-sterilized male participants who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, a condom (see Male Contraception in the protocol) from signing the informed consent form (ICF) until the end of the trial. Male participants are not allowed to donate sperm from signing the ICF until the end of the trial.
In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP or available through another patient program for patients with primary ITP):
5. Ability to understand the requirements of the additional 52-week treatment period 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).
6. Participant has completed a 52-week treatment period.
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 136
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
Participants are eligible to be included in the trial only if none of the following criteria apply:
1. Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines)
2. Use of any other investigational drug or participation in any other investigational trial
3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients
4. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary immune thrombocytopenia (ITP)
MedDRA version: 23.0 Level: LLT Classification code 10083843 Term: Primary immune thrombocytopenia System Organ Class: 100000004851

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Product Name: efgartigimod PH20 SC
Product Code: [ARGX-113]
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: efgartigimod alfa
CAS Number: 1821402-21-4
Current Sponsor code: ARGX-113
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 180-

Primary Outcome(s)

Primary end point(s): 1. Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
2. Vital signs, electrocardiogram (ECG), and laboratory safety evaluations

Main Objective: To evaluate the safety and tolerability of efgartigimod PH20 SC in adult patients with primary ITP

Secondary Objective: First 52-week treatment period only
• To evaluate the efficacy of efgartigimod PH20 SC on overall platelet count response
• To evaluate the use of rescue treatment and reduction in concurrent ITP therapy while receiving treatment with efgartigimod PH20 SC
• To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod PH20 SC
• To assess the PK of efgartigimod PH20 SC
• To evaluate the effects of efgartigimod PH20 SC treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO)
• To assess the PD effects of efgartigimod PH20 SC
• To evaluate the feasibility of self-administration of efgartigimod PH20 SC

First 52-week treatment period and additional 52-week treatment
periods
• To assess the immunogenicity of efgartigimod and/or rHuPH20

Timepoint(s) of evaluation of this end point: 1. At all visits
2. At planned visits per schedule of assessment

Secondary Outcome(s)

Secondary end point(s): First 52-week treatment period only
1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of > and =50×10^9/L
2. Proportion of patients with overall platelet count response defined as achieving a platelet count of > and =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-113-2004 trial with a platelet count of <30×10^9/L: time to response defined as the time to achieve 2 consecutive platelet counts of > and =50×10^9/L
5. The percentage of weeks in the trial with platelet counts of > and = 30×10^9/L and > and =20×10^9/L above baseline
6. In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of > and =30×10^9/L and > and =20×10^9/L above baseline
7. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of > and =50×10^9/L for at least 4 of the 6 visits between week 19 and week 24
8. In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of > and =50×10^9/L for at least 6 of the 8 visits between week 17 and week 24
9. Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
10. Rate of receipt of rescue therapy (rescue per patient per month)
11. Incidence and severity of the World Health Organization (WHO)- classified bleeding events
12. Serum efgartigimod concentration observed predose (Ctrough)
13. Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) and QoL (Short Form-36 [SF-36]) at planned visits
14. Pharmacodynamics markers: total IgG
15. Number and percentage of patients who performed self-administration at home over time
16. Number and percentage of caregivers who administered the injection to the patient at home over time
17. Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
18. Number and percentage of self- or caregiver-supported administrations at home

First 52-week treatment period and additional 52-week treatment periods
19. Incidence and prevalence of antibodies to efgartigimod
20. Titers of antibodies to efgartigimod
21 Presence of neutralizing antibodies (NAb) against efgartigimod

Timepoint(s) of evaluation of this end point: 1,2 At any time during the 52-week treatment period
3. At each visit
4, 5, 6 At planned visits per schedule of assessment
7. Between week 19 and week 24
8. Between week 17 and week 24
9,10,11,12, 13, 14 At planned visits per schedule of assessment
15, 16, 17, 18 Throughout the treatment period
19, 20, 21 At planned visits per schedule of assessments

Secondary ID(s)

2020-004033-20-NO

ARGX-113-2005

NCT04812925

Source(s) of Monetary Support

argenx BV

Ethics review

Status: Approved
Approval date: 20/07/2021
Contact: