A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

Primary Immune Thrombocytopenia Clinical Trial

— ADVANCE SC+  

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04812925
• Other study ID #: ARGX-113-2005
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 17, 2021
• Est. completion date: October 1, 2026

Study information

• Verified date: November 2023
• Source: argenx
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 173
• Est. completion date: October 1, 2026
• Est. primary completion date: October 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

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, their 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.

3a. Agree to use contraceptives consistent with local regulations and the following:

• Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. 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), the following criteria

apply:

4. 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).

5. Participant has completed a 52-week treatment period.

Exclusion criteria:

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

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Biological:
• efgartigimod PH20 SC
Subcutaneous injection with efgartigimod PH20 SC

Locations

Country	Name	City	State
Argentina	Investigator Site 0540001	Buenos Aires
Argentina	Investigator site 540004	Buenos Aires
Australia	Investigator Site 0610012	Garran
Australia	Investigator Site 0610003	West Perth
Australia	Investigator site 610005	Westmead
Bulgaria	Investigator Site 3590017	Plovdiv
Chile	Investigator site 0560003	Reñaca
Chile	Investigator site 560002	Santiago
Chile	Investigator site 0560004	Temuco
China	Investigator site 860013	Beijing
China	Investigator Site 0860008	Bengbu
China	Investigator site 860055	Huizhou
China	Investigator Site 0860015	Shenzhen
China	Investigator Site 0860001	Tianjin
China	Investigator Site 0860010	Wuhan
China	Investigator Site 0860002	Wuxi
China	Investigator Site 0860011	Zhengzhou
China	Investigator site 860058	Zhenjiang
Georgia	Investigator Site 9950007	Tbilisi
Georgia	Investigator site 9950009	Tbilisi
Georgia	Investigator site 9950011	Tbilisi
Greece	Investigator site 300008	Athens
Greece	Investigator Site 0300009	Thessaloníki
Ireland	Investigator site 3530003	Dublin
Italy	Investigator site 390043	Ferrara
Italy	Investigator Site 0390032	Milan
Italy	Investigator Site 0390044	Napoli
Italy	Investigator site 390041	Napoli
Japan	Investigator site JP0810015	Hirakata
Japan	Investigator Site 0810017	Iruma
Japan	Investigator Site 0810053	Kanagawa
Japan	Investigator Site 0810051	Kitakyushu
Japan	Investigator site 0810016	Shibukawa
Japan	Investigator site 810023	Shimotsuke
Japan	Investigator Site 0810038	Tama
Jordan	Investigator site 9620001	Irbid
Korea, Republic of	Investigator site 820004	Seoul
Korea, Republic of	Investigator site KO0820007	Seoul
Mexico	Investigator site 520002	Aguascalientes
New Zealand	Investigator site 640005	Christchurch
Norway	Investigator site 0470003	Sarpsborg
Poland	Investigator site PL0480013	Katowice
Poland	Investigator Site 0480026	Nowy Sacz
Poland	Investigator Site 0480037	Skorzewo
Portugal	Investigator Site 3510007	Lisboa
Portugal	Investigator site 3510004	Porto
Portugal	Investigator site 3510001	Vila Nova De Gaia
Romania	Investigator Site 0400005	Bucharest
Romania	Investigator site 400012	Bucuresti
Romania	Investigator Site 0400007	Craiova
Russian Federation	Investigator site 0070040	Kirov
Russian Federation	Investigator Site 0070026	Moscow
Russian Federation	Investigator Site 0070038	Nizhny Novgorod
Russian Federation	Investigator Site 0070037	Novosibirsk
South Africa	Investigator site 270003	Johannesburg
South Africa	Investigator site 270004	Observatory
South Africa	Investigator site 270001	Pretoria
South Africa	Investigator site 270002	Randburg
Thailand	Investigator Site 0660002	Bangkok
Thailand	Investigator Site 0660003	Bangkok
Thailand	Investigator site 660005	Bangkok
Thailand	Investigator Site 0660001	Bangkok Noi
Thailand	Investigator site 0660004	Chiang Mai
Thailand	Investigator site TH0660009	Khon Kaen
Tunisia	Investigator site 2610001	Sousse
Tunisia	Investigator site 2160002	Tunis
Turkey	Investigator Site 0900007	Adapazari
Turkey	Investigator Site 0900003	Ankara
Turkey	Investigator Site 0900008	Ankara
Turkey	Investigator Site 0900015	Ankara
Turkey	Investigator site 900004	Izmir
Turkey	Investigator Site 0900014	Kocaeli
Turkey	Investigator site 900010	Mersin
Turkey	Investigator site 0900017	Tekirdag
Turkey	Investigator site 900019	Trabzon
United Kingdom	Investigator site 440005	Coventry
United Kingdom	Investigator site UK044041	London
United Kingdom	Investigator site UK0440014	Penzance
United States	Investigator Site 0010116	Bentonville	Arkansas
United States	Investigator Site 0010062	Fort Wayne	Indiana
United States	Investigator site US0010042	Iowa City	Iowa
United States	Investigator Site 0010095	Oklahoma City	Oklahoma
United States	Investigator site 0010045	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Bulgaria,  Chile,  China,  Georgia,  Greece,  Ireland,  Italy,  Japan,  Jordan,  Korea, Republic of,  Mexico,  New Zealand,  Norway,  Poland,  Portugal,  Romania,  Russian Federation,  South Africa,  Thailand,  Tunisia,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)		216 weeks
Primary	Vital sign measurement: blood pressure in the overall population		216 weeks
Primary	ECG: PR, QT and QRS interval in the overall population		216 weeks
Primary	Laboratory safety evaluations: CRP analysis in the overall population		216 weeks
Secondary	Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10E9/L		52 weeks
Secondary	Proportion of patients with overall platelet count response defined as achieving a platelet count of =50×10E9/L on at least 4 occasions at any time during the 52-week treatment period		52 weeks
Secondary	Mean change from baseline in platelet count at each visit		52 weeks
Secondary	For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of =50×10E9/L		52 weeks
Secondary	The percentage of weeks in the trial with platelet counts of =30×10E9/L and =20×10E9/L above baseline		52 weeks
Secondary	In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of =30×10E9/L and =20×10E9/L above baseline		52 weeks
Secondary	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 =50×10E9/L for at least 4 of the 6 visits between week 19 and week 24		5 weeks (week 19-24)
Secondary	In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of =50×10E9/L for at least 6 of the 8 visits between week 17 and week 24		7 weeks (week 17-24)
Secondary	Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline		52 weeks
Secondary	Rate of receipt of rescue therapy (rescue per patient per month)		52 weeks
Secondary	Incidence of the World Health Organization (WHO)-classified bleeding events		52 weeks
Secondary	Severity of the World Health Organization (WHO)-classified bleeding events		52 weeks
Secondary	Serum efgartigimod concentration observed predose (Ctrough)		52 weeks
Secondary	Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits		52 weeks
Secondary	Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits		52 weeks
Secondary	Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits		52 weeks
Secondary	Pharmacodynamics markers: total IgG		52 weeks
Secondary	Number of patients who performed self-administration at home over time		52 weeks
Secondary	Percentage of patients who performed self-administration at home over time		52 weeks
Secondary	Number of caregivers who administered the injection to the patient at home over time		52 weeks
Secondary	Percentage of caregivers who administered the injection to the patient at home over time		52 weeks
Secondary	Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC		52 weeks
Secondary	Number of self- or caregiver-supported administrations at home		52 weeks
Secondary	Percentage of self- or caregiver-supported administrations at home		52 weeks
Secondary	Incidence and prevalence of antibodies to efgartigimod		216 weeks
Secondary	Titers of antibodies to efgartigimod		216 weeks
Secondary	Presence of neutralizing antibodies (NAb) against efgartigimod		216 weeks