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

NCT04687072 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: ARGX-113-20042020-004032-21
• Study Type: interventional
• Target: 207
• Locations: 31 countries
• Sites: 197

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

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24

  A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.

  Up to 6 weeks (between Weeks 19 and 24)

Secondary Outcomes (23)

• Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population

  Up to 24 weeks

• Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24

  Up to 6 weeks (between Weeks 19 and 24)

• Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24

  Up to 8 weeks (between Weeks 17 and 24)

• Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period

  Up to 24 weeks

• Extent of Disease Control Until Week 12 in the Overall Population

  Up to 12 weeks

Study Arms (2)

Efgartigimod PH20 SC

EXPERIMENTAL

Patients receiving efgartigimod PH20 SC treatment

Biological: Efgartigimod PH20 SC

Placebo PH20 SC

PLACEBO COMPARATOR

Patients receiving placebo PH20 SC treatment

Other: Placebo PH20 SC

Interventions

Efgartigimod PH20 SC BIOLOGICAL

Subcutaneous injection with efgartigimod PH20 SC

Also known as: ARGX-113 PH20 SC

Efgartigimod PH20 SC

Placebo PH20 SC OTHER

Subcutaneous injection with placebo PH20 SC

Placebo PH20 SC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• argenx: lead

Study Sites (200)

Investigator Site 0010116

Springdale, Arkansas, 72758, United States

Location

Investigator site 0010036

Los Angeles, California, 90033, United States

Location

Investigator Site 0010045

Washington D.C., District of Columbia, 20007, United States

Location

Investigator Site 0010104

Weston, Florida, 33331, United States

Location

Investigator site 0010112

Chicago, Illinois, 60612, United States

Location

Investigator site 0010193

Chicago, Illinois, 60637, United States

Location

Investigator Site 0010079

Lisle, Illinois, 60187, United States

Location

Investigator Site 0010062

Fort Wayne, Indiana, 46804, United States

Location

Investigator site 0010042

Iowa City, Iowa, 52242, United States

Location

Investigator Site 0010083

Detroit, Michigan, 48202, United States

Location

Investigator Site 0010102

Minneapolis, Minnesota, 55455, United States

Location

Investigator site 0010040

Columbus, Ohio, 43210, United States

Location

Investigator Site 0010095

Oklahoma City, Oklahoma, 73142, United States

Location

Investigator Site 0010115

Pittsburgh, Pennsylvania, 15222, United States

Location

Investigator Site 0540001

Buenos Aires, Argentina

Location

Investigator Site 0540004

Buenos Aires, Argentina

Location

Investigator Site 0540003

Córdoba, Argentina

Location

Investigator Site 0610009

Adelaide, Australia

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Investigator Site 0610004

Bedford Park, Australia

Location

Investigator Site 0610002

Box Hill, Australia

Location

Investigator Site 0610010

Clayton, Australia

Location

Investigator Site 0610012

Garran, Australia

Location

Investigator Site 0610001

Hobart, Australia

Location

Investigator Site 0610011

Perth, Australia

Location

Investigator Site 0610003

West Perth, Australia

Location

Investigator Site 0610005

Westmead, Australia

Location

Investigator Site 3590017

Plovdiv, Bulgaria

Location

Investigator Site 3590015

Sofia, Bulgaria

Location

Investigator Site 0560002

Santiago, Chile

Location

Investigator Site 0560004

Temuco, Chile

Location

Investigator Site 0560003

Viña del Mar, Chile

Location

Investigator Site 0860003

Beijing, China

Location

Investigator Site 0860013

Beijing, China

Location

Investigator Site 0860008

Bengbu, China

Location

Investigator Site 0860055

Huizhou, China

Location

Investigator Site 0860009

Kunming, China

Location

Investigator Site 0860012

Nanchang, China

Location

Investigator Site 0860014

Shanxi, China

Location

Investigator Site 0860015

Shenzhen, China

Location

Investigator Site 0860001

Tianjin, China

Location

Investigator Site 0860006

Wenzhou, China

Location

Investigator Site 0860010

Wuhan, China

Location

Investigator Site 0860002

Wuxi, China

Location

Investigator Site 0860005

Zhejiang, China

Location

Investigator Site 0860011

Zhengzhou, China

Location

Investigator Site 0860058

Zhenjiang, China

Location

Investigator site 0860062

Zhenjiang, China

Location

Investigator Site 0450005

Roskilde, Denmark

Location

Investigator Site 0330009

Créteil, France

Location

Investigator Site 0330018

Montpellier, France

Location

Investigator Site 9950006

Tbilisi, Georgia

Location

Investigator Site 9950007

Tbilisi, Georgia

Location

Investigator Site 9950008

Tbilisi, Georgia

Location

Investigator Site 9950009

Tbilisi, Georgia

Location

Investigator Site 9950011

Tbilisi, Georgia

Location

Investigator Site 9950019

Tbilisi, Georgia

Location

Investigator site 0490008

Essen, 45147, Germany

Location

Investigator Site 0490012

Giessen, Germany

Location

Investigator Site 0300008

Athens, Greece

Location

Investigator Site 0300010

Athens, Greece

Location

Investigator Site 0300007

Pátrai, Greece

Location

Investigator Site 0300009

Thessaloniki, Greece

Location

Investigator Site 3530002

Dublin, Ireland

Location

Investigator Site 3530003

Dublin, Ireland

Location

Investigator Site 3530001

Galway, Ireland

Location

Investigator Site 9720013

Ashkelon, Israel

Location

Investigator Site 9720010

Haifa, Israel

Location

Investigator Site 9720012

Haifa, Israel

Location

Investigator Site 9720008

Jerusalem, Israel

Location

Investigator Site 9720011

Jerusalem, Israel

Location

Investigator Site 9720007

Petah Tikva, Israel

Location

Investigator Site 9720009

Tel Aviv, Israel

Location

Investigator Site 0390037

Alessandria, Italy

Location

Investigator Site 0390043

Ferrara, Italy

Location

Investigator Site 0390045

Meldola, Italy

Location

Investigator site 0390014

Milan, 20122, Italy

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Investigator Site 0390032

Milan, Italy

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Investigator Site 0390041

Napoli, Italy

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Investigator Site 0390044

Napoli, Italy

Location

Investigator Site 0390015

Novara, Italy

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Investigator Site 0390035

Potenza, Italy

Location

Investigator Site 0390011

Reggio Calabria, Italy

Location

Investigator site 0390018

Reggio Emilia, 42100, Italy

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Investigator Site 0390046

Rome, Italy

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Investigator Site 0390033

Terni, Italy

Location

Investigator Site 0390036

Varese, Italy

Location

Investigator Site 0810056

Chiba, Japan

Location

Investigator Site 0810015

Hirakata, Japan

Location

Investigator Site 0810010

Hiroshima, Japan

Location

Investigator Site 0810053

Kanagawa, Japan

Location

Investigator Site 0810051

Kitakyushu, Japan

Location

Investigator Site 0810054

Kumamoto, Japan

Location

Investigator Site 0810018

Maebashi, Japan

Location

Investigator Site 0810057

Morioka, Japan

Location

Investigator Site 0810012

Ōgaki, Japan

Location

Investigator Site 0810017

Saitama, Japan

Location

Investigator Site 0810016

Shibukawa, Japan

Location

Investigator Site 0810023

Shimotsuke, Japan

Location

Investigator Site 0810039

Shinagawa-Ku, Japan

Location

Investigator Site 0810038

Tama, Japan

Location

Investigator Site 0810052

Tokyo, Japan

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Investigator Site 0810048

Tsukuba, Japan

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Investigator Site 0810044

Yamanashi, Japan

Location

Investigator Site 9620002

Amman, Jordan

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Investigator Site 9620001

Irbid, Jordan

Location

Investigator Site 0520002

Aguascalientes, Mexico

Location

Investigator Site 0520004

Chihuahua City, Mexico

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Investigator Site 0520007

México, Mexico

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Investigator Site 0520003

Monterrey, Mexico

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Investigator Site 0520001

Oaxaca City, Mexico

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Investigator Site 0640001

Auckland, New Zealand

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Investigator Site 0640005

Christchurch, New Zealand

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Investigator Site 0640002

Palmerston North, New Zealand

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Investigator Site 0470002

Bergen, Norway

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Investigator Site 0470003

Oslo, Norway

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Investigator Site 0480013

Katowice, 40519, Poland

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Investigator Site 0480014

Lublin, Poland

Location

Investigator Site 0480026

Nowy Sącz, Poland

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Investigator Site 0480037

Skorzewo, Poland

Location

Investigator Site 0480039

Torun, Poland

Location

Investigator Site 0480033

Warsaw, Poland

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Investigator Site 3510006

Braga, Portugal

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Investigator Site 3510003

Coimbra, Portugal

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Investigator Site 3510002

Lisbon, Portugal

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Investigator Site 3510005

Lisbon, Portugal

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Investigator Site 3510007

Lisbon, Portugal

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Investigator Site 3510001

Porto, Portugal

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Investigator Site 3510004

Porto, Portugal

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Investigator Site 0400005

Bucharest, Romania

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Investigator Site 0400006

Bucharest, Romania

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Investigator Site 0400009

Bucharest, Romania

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Investigator Site 0400012

Bucharest, Romania

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Investigator Site 0400016

Cluj-Napoca, Romania

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Investigator Site 0400007

Craiova, Romania

Location

Investigator Site 0400011

Sibiu, Romania

Location

Investigator Site 0400008

Târgu Mureş, Romania

Location

Investigator Site 0070006

Kaluga, Russia

Location

Investigator Site 0070040

Kirov, Russia

Location

Investigator Site 0070026

Moscow, Russia

Location

Investigator Site 0070038

Nizhny Novgorod, Russia

Location

Investigator Site 0070037

Novosibirsk, Russia

Location

Investigator Site 0070024

Pyatigorsk, Russia

Location

Investigator Site 0070025

Saint Petersburg, Russia

Location

Investigator Site 0070039

Smolensk, Russia

Location

Investigator Site 0070015

Syktyvkar, Russia

Location

Investigator Site 0070012

Tula, Russia

Location

Investigator Site 3810006

Belgrade, Serbia

Location

Investigator Site 3810008

Kragujevac, Serbia

Location

Investigator Site 0270005

George, South Africa

Location

Investigator Site 0270003

Johannesburg, South Africa

Location

Investigator Site 0270004

Observatory, South Africa

Location

Investigator Site 0270001

Pretoria, South Africa

Location

Investigator Site 0270002

Randburg, South Africa

Location

Investigator Site 0820005

Seongnam, South Korea

Location

Investigator Site 0820003

Seoul, South Korea

Location

Investigator Site 0820004

Seoul, South Korea

Location

Investigator Site 0820006

Seoul, South Korea

Location

Investigator Site 0820007

Seoul, South Korea

Location

Investigator Site 0820008

Seoul, South Korea

Location

Investigator Site 0340024

Alava, Spain

Location

Investigator Site 0340007

Barcelona, 08035, Spain

Location

Investigator Site 0340006

Barcelona, Spain

Location

Investigator Site 0340023

Barcelona, Spain

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Investigator Site 0340037

Madrid, Spain

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Investigator Site 0340022

Murcia, Spain

Location

Investigator Site 0340036

Sabadell, Spain

Location

Investigator site 0340013

Seville, 41013, Spain

Location

Investigator Site 0340004

Valencia, Spain

Location

Investigator Site 8860001

New Taipei City, Taiwan

Location

Investigator Site 8860003

Taoyuan District, Taiwan

Location

Investigator Site 0660002

Bangkok, Thailand

Location

Investigator Site 0660003

Bangkok, Thailand

Location

Investigator Site 0660005

Bangkok, Thailand

Location

Investigator Site 0660008

Bangkok, Thailand

Location

Investigator Site 0660001

Bangkok Noi, Thailand

Location

Investigator Site 0660004

Chiang Mai, Thailand

Location

Investigator Site 0660009

Khon Kaen, Thailand

Location

Investigator Site 0660006

Pathum Thani, Thailand

Location

Investigator Site 2160006

Sfax, Tunisia

Location

Investigator Site 2160001

Sousse, Tunisia

Location

Investigator Site 2160002

Tunis, Tunisia

Location

Investigator Site 0900007

Adapazarı, Turkey (Türkiye)

Location

Investigator Site 0900003

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900006

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900008

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900015

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900016

Edirne, Turkey (Türkiye)

Location

Investigator Site 0900013

Istanbul, Turkey (Türkiye)

Location

Investigator Site 0900004

Izmir, Turkey (Türkiye)

Location

Investigator Site 0900014

Kocaeli, Turkey (Türkiye)

Location

Investigator Site 0900018

Malatya, Turkey (Türkiye)

Location

Investigator Site 0900010

Mersin, Turkey (Türkiye)

Location

Investigator Site 0900009

Samsun, Turkey (Türkiye)

Location

Investigator Site 0900017

Tekirdağ, Turkey (Türkiye)

Location

Investigator Site 0900019

Trabzon, Turkey (Türkiye)

Location

Investigator site 0440011

Bradford, BD9 6RJ, United Kingdom

Location

Investigator Site 0440005

Coventry, United Kingdom

Location

Investigator Site 0440008

London, United Kingdom

Location

Investigator Site 0440041

London, United Kingdom

Location

Investigator Site 0440014

Truro, United Kingdom

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Regulatory manager
• Organization: Argenx

regulatory@argenx.com

Please email

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2020
• First Posted: December 29, 2020
• Study Start: December 16, 2020
• Primary Completion: October 9, 2023
• Study Completion: October 9, 2023
• Last Updated: October 31, 2024
• Results First Posted: October 31, 2024

Record last verified: 2024-10

Locations

KR (6); GR (4); TH (8); DE (2); JO (2); RO (8); GE (6); SE (2); IT (14); NO (2); NZ (3); TU (14); PL (6); GB (5); US (14); DK (1); RU (10); ZA (5); TW (2); IL (7); BU (2); ES (9); CN (16); IE (3); AU (9); AR (3); CL (3); FR (2); JP (17); PT (7); ME (5)