A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

Primary Immune Thrombocytopenia Clinical Trial

— ADVANCE+  

Official 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.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04225156
• Other study ID #: ARGX-113-1803
• Secondary ID: 2019-002101-21
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 2, 2020
• Est. completion date: November 2025

Study information

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

Clinical Trial Summary

This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 101
• Est. completion date: November 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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-weeks trial period.

3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.

4. Women of childbearing potential should use a highly effective or acceptable method of contraception 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 (as listed in the protocol)

6. 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).

7. Patient has completed a 52-week treatment period.

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.

Related Conditions & MeSH terms

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

Intervention

Biological:
• efgartigimod
Intravenous infusion of efgartigimod

Locations

Country	Name	City	State
Austria	Investigator Site 0430002	Vienna
Austria	Investigator Site 0430003	Vienna
Belgium	Investigator Site 0320012	Brasschaat
Belgium	Investigator Site 0320011	Brugge
Belgium	Investigator Site 0320014	Turnhout
Belgium	Investigator Site 0320002	Yvoir
Bulgaria	Investigator Site 3590001	Pleven
Bulgaria	Investigator Site 3590002	Sofia
Czechia	Investigator Site 4200001	Brno
Czechia	Investigator Site 4200008	Olomouc
Czechia	Investigator Site 4200006	Ostrava
Czechia	Investigator Site 4200007	Praha
France	Investigator Site 0330009	Créteil
France	Investigator Site 0330018	Montpellier
France	Investigator Site 0330016	Périgueux
France	Investigator Site 0330008	Pessac
Georgia	Investigator site 9950007	Tbilisi
Georgia	Investigator site 9950008	Tbilisi
Georgia	Investigator site 9950009	Tbilisi
Georgia	Investigator site 9950011	Tbilisi
Georgia	Investigator site 9950012	Tbilisi
Germany	Investigator Site 0490010	Düsseldorf
Germany	Investigator Site 0490008	Essen
Hungary	Investigator Site 0360004	Budapest
Hungary	Investigator Site 0360006	Debrecen
Hungary	Investigator Site 0360015	Gyor
Hungary	Investigator Site 0360010	Nyíregyháza
Hungary	Investigator Site 0360014	Szombathely
Italy	Investigator Site 0390014	Milano
Italy	Investigator Site 0390020	Monza
Italy	Investigator Site 0390015	Novara
Italy	Investigator Site 0390010	Ravenna
Italy	Investigator Site 0390011	Reggio Calabria
Italy	Investigator Site 0390018	Reggio Emilia
Italy	Investigator Site 0390019	Rimini
Italy	Investigator Site 0390009	Siena
Italy	Investigator Site 0390016	Trieste
Japan	Investigator Site 0810015	Hirakata
Japan	Investigator Site 0810010	Hiroshima
Japan	Investigator Site 0810017	Iruma
Japan	Investigator Site 0810022	Kashiwa
Japan	Investigator Site 0810018	Maebashi
Japan	Investigator Site 0810021	Niigata
Japan	Investigator Site 0810014	Sapporo
Japan	Investigator Site 0810016	Shibukawa
Japan	Investigator Site 0810023	Shimotsuke
Netherlands	Investigator Site 0310006	Den Haag
Netherlands	Investigator Site 0310005	Rotterdam
Poland	Investigator Site 0480012	Gdansk
Poland	Investigator Site 0480013	Katowice
Poland	Investigator Site 0480011	Lódz
Poland	Investigator Site 0480014	Lublin
Poland	Investigator Site 0480026	Nowy Sacz
Russian Federation	Investigator Site 0070006	Kaluga
Russian Federation	Investigator Site 0070008	Moscow
Russian Federation	Investigator Site 0070007	Petrozavodsk
Russian Federation	Investigator Site 0070013	Rostov-on-Don
Russian Federation	Investigator Site 0070015	Syktyvkar
Russian Federation	Investigator Site 0070012	Tula
Russian Federation	Investigator Site 0070010	Ufa
Spain	Investigator Site 0340006	Barcelona
Spain	Investigator Site 0340007	Barcelona
Spain	Investigator Site 0340009	Madrid
Spain	Investigator Site 0340014	Madrid
Spain	Investigator Site 0340012	Palma De Mallorca
Spain	Investigator Site 0340015	Pozuelo De Alarcón
Spain	Investigator Site 0340013	Sevilla
Spain	Investigator Site 0340004	Valencia
Spain	Investigator Site 0340011	Valencia
Turkey	Investigator Site 0900003	Ankara
Turkey	Investigator Site 0900006	Ankara
Turkey	Investigator Site 0900015	Ankara
Turkey	Investigator Site 0900016	Edirne
Turkey	Investigator Site 0900013	Istanbul
Turkey	Investigator Site 0900004	Izmir
Turkey	Investigator Site 0900010	Mersin
Turkey	Investigator Site 0900007	Sakarya
Turkey	Investigator Site 0900009	Samsun
Turkey	Investigator Site 0900017	Tekirdag
Turkey	Investigator Site 0900019	Trabzon
Ukraine	Investigator Site 3800006	Mykolaiv
United Kingdom	Investigator Site 0440008	London
United Kingdom	Investigator Site 0440012	Southampton
United States	Investigator Site 0010040	Columbus	Ohio
United States	Investigator Site 0010042	Iowa City	Iowa
United States	Investigator Site 0010037	Ocala	Florida
United States	Investigator Site 0010045	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Bulgaria,  Czechia,  France,  Georgia,  Germany,  Hungary,  Italy,  Japan,  Netherlands,  Poland,  Russian Federation,  Spain,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Frequency and severity of Adverse Events		Up to 60 weeks
Primary	Frequency and severity of vital signs		Up to 60 weeks
Primary	Frequency and severity of laboratory assessments		Up to 60 weeks
Secondary	Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10E9/L.		Over the 52 weeks of treatment
Secondary	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.		Over the 52 weeks of treatment
Secondary	Mean change from baseline in platelet count at each visit.		Up to 60 weeks, at each visit
Secondary	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		Up to 60 weeks, at each visit
Secondary	The percentage of weeks in the trial with platelet counts of =30×109/L and at least 20×10E9/L above baseline.		Over the 52 weeks of treatment
Secondary	In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of =30×10E9/L and at least 20×10E9/L above baseline.		Over the 52 weeks of treatment
Secondary	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.		Up to 5 weeks, between visit 19 and 24 of the trial
Secondary	In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.		Up to 7 weeks, between visit 17 and 24 of the trial
Secondary	Rate of receipt of rescue therapy (rescue per patient per month).		Up to 60 weeks, at each visit
Secondary	Reduction in concurrent ITP therapy.		Up to 60 weeks, at each visit
Secondary	Incidence and severity of the WHO-classified bleeding events.		Up to 60 weeks, at each visit
Secondary	Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.		Up to 52 weeks
Secondary	Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.		Up to 52 weeks
Secondary	Change from baseline in Quality of Life (SF-36) at planned visits.		Up to 52 weeks
Secondary	Incidence of anti-drug antibodies (ADA) to efgartigimod.		Up to 216 weeks
Secondary	Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).		Up to 60 weeks
Secondary	Pharmacodynamics markers: total IgG.		Up to 60 weeks