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

NCT ID: NCT04687072

Last Updated: 2024-10-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 207 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-16
• Study Completion Date: 2023-10-09

Brief Summary

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Conditions

Primary Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Efgartigimod PH20 SC

Patients receiving efgartigimod PH20 SC treatment

Group Type: EXPERIMENTAL

Efgartigimod PH20 SC

Intervention Type: BIOLOGICAL

Subcutaneous injection with efgartigimod PH20 SC

Placebo PH20 SC

Patients receiving placebo PH20 SC treatment

Group Type: PLACEBO_COMPARATOR

Placebo PH20 SC

Intervention Type: OTHER

Subcutaneous injection with placebo PH20 SC

Interventions

Efgartigimod PH20 SC

Subcutaneous injection with efgartigimod PH20 SC

Intervention Type: BIOLOGICAL

Placebo PH20 SC

Subcutaneous injection with placebo PH20 SC

Intervention Type: OTHER

Other Intervention Names

ARGX-113 PH20 SC

Eligibility Criteria

Inclusion Criteria

• Ability to understand the requirements of the trial and provide written informed consent, willing and able to comply with the trial protocol procedures
• Is at least the local age of consent for clinical studies when signing the ICF.
• Confirmed diagnosis of primary ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and no known etiology for thrombocytopenia
• Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in the opinion of the investigator
• Mean platelet count of <30×10E9/L from at least 3 documented, qualifying counts within the 3 preceding months where at least 2 of the qualifying counts must be taken during the screening period: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization (visit 1). If the third count is not available from the 3 preceding months, this third platelet count can be obtained during the screening period.
• A documented history of a platelet count of <30×10E9/L before screening
• At the start of the trial, the participant either takes concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the participant does not take treatment for ITP (see note) but has received at least 2 prior treatments for ITP. Participants receiving permitted concurrent ITP treatment(s) at baseline must have been stable in dose and frequency for at least 4 weeks before randomization.

Permitted concurrent ITP medications include corticosteroids, danazol, vinca alkaloids, oral immunosuppressants, dapsone, fostamatinib, and/or oral TPO-RAs.

-Agree to use contraceptive measures consistent with local regulations and the protocol

Exclusion Criteria

• Secondary ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, thrombocytopenia associated with myeloid dysplasia, or hematopoietic stem cell transplant
• Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization
• Use of any transfusions within 4 weeks prior to randomization
• Use of Ig (IV, SC, or intramuscular route) or plasmapheresis (PLEX) within 4 weeks prior to randomization
• Use of romiplostim within 4 weeks prior to randomization
• Undergone splenectomy less than 4 weeks prior to randomization
• Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP
• Use of any monoclonal antibody or Fc fusion proteins, other than those previously indicated, within 6 months before the first dose of the IMP (eg, anti-CD20)
• At the screening visit, clinically significant laboratory abnormalities as follows: Hemoglobin ≤9 g/dL - OR - International normalized ratio >1.5 or activated partial thromboplastin time >1.5×upper limit of normal - OR - total IgG level <6 g/L
• History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancer can be included at any time: Adequately treated basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast or Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
• Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments
• History of any major thrombotic or embolic event (eg, myocardial infarction, stroke, deep venous thrombosis, or pulmonary embolism) within 5 years prior to randomization
• History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia
• Evidence of an active clinically significant bleeding of an organ or internal mucosal bleeding, other than expected in ITP, that warrants emergent treatment or therapeutic procedure based on the investigator's judgment (eg, intracranial hemorrhage, pulmonary hemorrhage, bleeding with ongoing need for packed red blood cell transfusion)
• Estimated high risk of a clinically significant bleeding of an organ or internal mucosal bleeding, other than expected in ITP, that warrants emergent treatment or therapeutic procedure according to the investigator's judgment
• Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk
• Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection, unless associated with a negative HBV DNA test, Hepatitis C virus (HCV) based on HCV-antibody assay (unless associated with a negative HCV RNA test), Human immunodeficiency virus (HIV) based on test results that are associated with an acquired immunodeficiency syndrome (AIDS)-defining condition or a CD4 count ≤200 cells/mm3
• Known hypersensitivity reaction to efgartigimod, rHuPH20, or 1 of its excipients
• Previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP
• Pregnant or lactating or intends to become pregnant during the trial
• Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
• Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of ITP or put the participant at undue risk
• Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (eg, cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the participant at undue risk
• Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
• Received a live/live-attenuated vaccine less than 4 weeks before screening. The receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time before screening is not considered an exclusion criterion

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

argenx

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator Site 0010116

Springdale, Arkansas, United States

Investigator site 0010036

Los Angeles, California, United States

Investigator Site 0010045

Washington D.C., District of Columbia, United States

Investigator Site 0010104

Weston, Florida, United States

Investigator site 0010112

Chicago, Illinois, United States

Investigator site 0010193

Chicago, Illinois, United States

Investigator Site 0010079

Lisle, Illinois, United States

Investigator Site 0010062

Fort Wayne, Indiana, United States

Investigator site 0010042

Iowa City, Iowa, United States

Investigator Site 0010083

Detroit, Michigan, United States

Investigator Site 0010102

Minneapolis, Minnesota, United States

Investigator site 0010040

Columbus, Ohio, United States

Investigator Site 0010095

Oklahoma City, Oklahoma, United States

Investigator Site 0010115

Pittsburgh, Pennsylvania, United States

Investigator Site 0540001

Buenos Aires, , Argentina

Investigator Site 0540004

Buenos Aires, , Argentina

Investigator Site 0540003

Córdoba, , Argentina

Investigator Site 0610009

Adelaide, , Australia

Investigator Site 0610004

Bedford Park, , Australia

Investigator Site 0610002

Box Hill, , Australia

Investigator Site 0610010

Clayton, , Australia

Investigator Site 0610012

Garran, , Australia

Investigator Site 0610001

Hobart, , Australia

Investigator Site 0610011

Perth, , Australia

Investigator Site 0610003

West Perth, , Australia

Investigator Site 0610005

Westmead, , Australia

Investigator Site 3590017

Plovdiv, , Bulgaria

Investigator Site 3590015

Sofia, , Bulgaria

Investigator Site 0560002

Santiago, , Chile

Investigator Site 0560004

Temuco, , Chile

Investigator Site 0560003

Viña del Mar, , Chile

Investigator Site 0860003

Beijing, , China

Investigator Site 0860013

Beijing, , China

Investigator Site 0860008

Bengbu, , China

Investigator Site 0860055

Huizhou, , China

Investigator Site 0860009

Kunming, , China

Investigator Site 0860012

Nanchang, , China

Investigator Site 0860014

Shanxi, , China

Investigator Site 0860015

Shenzhen, , China

Investigator Site 0860001

Tianjin, , China

Investigator Site 0860006

Wenzhou, , China

Investigator Site 0860010

Wuhan, , China

Investigator Site 0860002

Wuxi, , China

Investigator Site 0860005

Zhejiang, , China

Investigator Site 0860011

Zhengzhou, , China

Investigator Site 0860058

Zhenjiang, , China

Investigator site 0860062

Zhenjiang, , China

Investigator Site 0450005

Roskilde, , Denmark

Investigator Site 0330009

Créteil, , France

Investigator Site 0330018

Montpellier, , France

Investigator Site 9950006

Tbilisi, , Georgia

Investigator Site 9950007

Tbilisi, , Georgia

Investigator Site 9950008

Tbilisi, , Georgia

Investigator Site 9950009

Tbilisi, , Georgia

Investigator Site 9950011

Tbilisi, , Georgia

Investigator Site 9950019

Tbilisi, , Georgia

Investigator site 0490008

Essen, , Germany

Investigator Site 0490012

Giessen, , Germany

Investigator Site 0300008

Athens, , Greece

Investigator Site 0300010

Athens, , Greece

Investigator Site 0300007

Pátrai, , Greece

Investigator Site 0300009

Thessaloniki, , Greece

Investigator Site 3530002

Dublin, , Ireland

Investigator Site 3530003

Dublin, , Ireland

Investigator Site 3530001

Galway, , Ireland

Investigator Site 9720013

Ashkelon, , Israel

Investigator Site 9720010

Haifa, , Israel

Investigator Site 9720012

Haifa, , Israel

Investigator Site 9720008

Jerusalem, , Israel

Investigator Site 9720011

Jerusalem, , Israel

Investigator Site 9720007

Petah Tikva, , Israel

Investigator Site 9720009

Tel Aviv, , Israel

Investigator Site 0390037

Alessandria, , Italy

Investigator Site 0390043

Ferrara, , Italy

Investigator Site 0390045

Meldola, , Italy

Investigator site 0390014

Milan, , Italy

Investigator Site 0390032

Milan, , Italy

Investigator Site 0390041

Napoli, , Italy

Investigator Site 0390044

Napoli, , Italy

Investigator Site 0390015

Novara, , Italy

Investigator Site 0390035

Potenza, , Italy

Investigator Site 0390011

Reggio Calabria, , Italy

Investigator site 0390018

Reggio Emilia, , Italy

Investigator Site 0390046

Rome, , Italy

Investigator Site 0390033

Terni, , Italy

Investigator Site 0390036

Varese, , Italy

Investigator Site 0810056

Chiba, , Japan

Investigator Site 0810015

Hirakata, , Japan

Investigator Site 0810010

Hiroshima, , Japan

Investigator Site 0810053

Kanagawa, , Japan

Investigator Site 0810051

Kitakyushu, , Japan

Investigator Site 0810054

Kumamoto, , Japan

Investigator Site 0810018

Maebashi, , Japan

Investigator Site 0810057

Morioka, , Japan

Investigator Site 0810012

Ōgaki, , Japan

Investigator Site 0810017

Saitama, , Japan

Investigator Site 0810016

Shibukawa, , Japan

Investigator Site 0810023

Shimotsuke, , Japan

Investigator Site 0810039

Shinagawa-Ku, , Japan

Investigator Site 0810038

Tama, , Japan

Investigator Site 0810052

Tokyo, , Japan

Investigator Site 0810048

Tsukuba, , Japan

Investigator Site 0810044

Yamanashi, , Japan

Investigator Site 9620002

Amman, , Jordan

Investigator Site 9620001

Irbid, , Jordan

Investigator Site 0520002

Aguascalientes, , Mexico

Investigator Site 0520004

Chihuahua City, , Mexico

Investigator Site 0520007

México, , Mexico

Investigator Site 0520003

Monterrey, , Mexico

Investigator Site 0520001

Oaxaca City, , Mexico

Investigator Site 0640001

Auckland, , New Zealand

Investigator Site 0640005

Christchurch, , New Zealand

Investigator Site 0640002

Palmerston North, , New Zealand

Investigator Site 0470002

Bergen, , Norway

Investigator Site 0470003

Oslo, , Norway

Investigator Site 0480013

Katowice, , Poland

Investigator Site 0480014

Lublin, , Poland

Investigator Site 0480026

Nowy Sącz, , Poland

Investigator Site 0480037

Skorzewo, , Poland

Investigator Site 0480039

Torun, , Poland

Investigator Site 0480033

Warsaw, , Poland

Investigator Site 3510006

Braga, , Portugal

Investigator Site 3510003

Coimbra, , Portugal

Investigator Site 3510002

Lisbon, , Portugal

Investigator Site 3510005

Lisbon, , Portugal

Investigator Site 3510007

Lisbon, , Portugal

Investigator Site 3510001

Porto, , Portugal

Investigator Site 3510004

Porto, , Portugal

Investigator Site 0400005

Bucharest, , Romania

Investigator Site 0400006

Bucharest, , Romania

Investigator Site 0400009

Bucharest, , Romania

Investigator Site 0400012

Bucharest, , Romania

Investigator Site 0400016

Cluj-Napoca, , Romania

Investigator Site 0400007

Craiova, , Romania

Investigator Site 0400011

Sibiu, , Romania

Investigator Site 0400008

Târgu Mureş, , Romania

Investigator Site 0070006

Kaluga, , Russia

Investigator Site 0070040

Kirov, , Russia

Investigator Site 0070026

Moscow, , Russia

Investigator Site 0070038

Nizhny Novgorod, , Russia

Investigator Site 0070037

Novosibirsk, , Russia

Investigator Site 0070024

Pyatigorsk, , Russia

Investigator Site 0070025

Saint Petersburg, , Russia

Investigator Site 0070039

Smolensk, , Russia

Investigator Site 0070015

Syktyvkar, , Russia

Investigator Site 0070012

Tula, , Russia

Investigator Site 3810006

Belgrade, , Serbia

Investigator Site 3810008

Kragujevac, , Serbia

Investigator Site 0270005

George, , South Africa

Investigator Site 0270003

Johannesburg, , South Africa

Investigator Site 0270004

Observatory, , South Africa

Investigator Site 0270001

Pretoria, , South Africa

Investigator Site 0270002

Randburg, , South Africa

Investigator Site 0820005

Seongnam, , South Korea

Investigator Site 0820003

Seoul, , South Korea

Investigator Site 0820004

Seoul, , South Korea

Investigator Site 0820006

Seoul, , South Korea

Investigator Site 0820007

Seoul, , South Korea

Investigator Site 0820008

Seoul, , South Korea

Investigator Site 0340024

Alava, , Spain

Investigator Site 0340007

Barcelona, , Spain

Investigator Site 0340006

Barcelona, , Spain

Investigator Site 0340023

Barcelona, , Spain

Investigator Site 0340037

Madrid, , Spain

Investigator Site 0340022

Murcia, , Spain

Investigator Site 0340036

Sabadell, , Spain

Investigator site 0340013

Seville, , Spain

Investigator Site 0340004

Valencia, , Spain

Investigator Site 8860001

New Taipei City, , Taiwan

Investigator Site 8860003

Taoyuan District, , Taiwan

Investigator Site 0660002

Bangkok, , Thailand

Investigator Site 0660003

Bangkok, , Thailand

Investigator Site 0660005

Bangkok, , Thailand

Investigator Site 0660008

Bangkok, , Thailand

Investigator Site 0660001

Bangkok Noi, , Thailand

Investigator Site 0660004

Chiang Mai, , Thailand

Investigator Site 0660009

Khon Kaen, , Thailand

Investigator Site 0660006

Pathum Thani, , Thailand

Investigator Site 2160006

Sfax, , Tunisia

Investigator Site 2160001

Sousse, , Tunisia

Investigator Site 2160002

Tunis, , Tunisia

Investigator Site 0900007

Adapazarı, , Turkey (Türkiye)

Investigator Site 0900003

Ankara, , Turkey (Türkiye)

Investigator Site 0900006

Ankara, , Turkey (Türkiye)

Investigator Site 0900008

Ankara, , Turkey (Türkiye)

Investigator Site 0900015

Ankara, , Turkey (Türkiye)

Investigator Site 0900016

Edirne, , Turkey (Türkiye)

Investigator Site 0900013

Istanbul, , Turkey (Türkiye)

Investigator Site 0900004

Izmir, , Turkey (Türkiye)

Investigator Site 0900014

Kocaeli, , Turkey (Türkiye)

Investigator Site 0900018

Malatya, , Turkey (Türkiye)

Investigator Site 0900010

Mersin, , Turkey (Türkiye)

Investigator Site 0900009

Samsun, , Turkey (Türkiye)

Investigator Site 0900017

Tekirdağ, , Turkey (Türkiye)

Investigator Site 0900019

Trabzon, , Turkey (Türkiye)

Investigator site 0440011

Bradford, , United Kingdom

Investigator Site 0440005

Coventry, , United Kingdom

Investigator Site 0440008

London, , United Kingdom

Investigator Site 0440041

London, , United Kingdom

Investigator Site 0440014

Truro, , United Kingdom

Countries

United States; Argentina; Australia; Bulgaria; Chile; China; Denmark; France; Georgia; Germany; Greece; Ireland; Israel; Italy; Japan; Jordan; Mexico; New Zealand; Norway; Poland; Portugal; Romania; Russia; Serbia; South Africa; South Korea; Spain; Taiwan; Thailand; Tunisia; Turkey (Türkiye); United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2020-004032-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ARGX-113-2004

Identifier Type: -

Identifier Source: org_study_id