AzTrials
AzTrials

Primary Immune ThrombocytopeniaPhase 3ADVANCE SCNCT04687072

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

A Phase 3 study testing Efgartigimod PH20 SC in people with Primary Immune Thrombocytopenia, run by argenx at 200 sites in 32 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Primary Immune Thrombocytopenia
What is being tested?
Efgartigimod PH20 SC
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
207 participants
Where
200 sitesin 32 countries
Study dates
16 Dec 2020to 9 Oct 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 9Show fewer
  • 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

Who cannot join

  • 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
Show all 25Show fewer
  • 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

Trial locations and contacts

200 sites in 32 countries.

200 sites in 32 countries

Japan17 sites

  • Investigator Site 0810056

    Chiba

  • Investigator Site 0810015

    Hirakata

  • Investigator Site 0810010

    Hiroshima

  • Investigator Site 0810053

    Kanagawa

  • Investigator Site 0810051

    Kitakyushu

  • Investigator Site 0810054

    Kumamoto

  • Investigator Site 0810018

    Maebashi

  • Investigator Site 0810057

    Morioka

  • Investigator Site 0810012

    Ōgaki

  • Investigator Site 0810017

    Saitama

  • Investigator Site 0810016

    Shibukawa

  • Investigator Site 0810023

    Shimotsuke

  • Investigator Site 0810039

    Shinagawa-Ku

  • Investigator Site 0810038

    Tama

  • Investigator Site 0810052

    Tokyo

  • Investigator Site 0810048

    Tsukuba

  • Investigator Site 0810044

    Yamanashi

Other trials in Japan

China16 sites

  • Investigator Site 0860003

    Beijing

  • Investigator Site 0860013

    Beijing

  • Investigator Site 0860008

    Bengbu

  • Investigator Site 0860055

    Huizhou

  • Investigator Site 0860009

    Kunming

  • Investigator Site 0860012

    Nanchang

  • Investigator Site 0860014

    Shanxi

  • Investigator Site 0860015

    Shenzhen

  • Investigator Site 0860001

    Tianjin

  • Investigator Site 0860006

    Wenzhou

  • Investigator Site 0860010

    Wuhan

  • Investigator Site 0860002

    Wuxi

  • Investigator Site 0860005

    Zhejiang

  • Investigator Site 0860011

    Zhengzhou

  • Investigator Site 0860058

    Zhenjiang

  • Investigator site 0860062

    Zhenjiang

Other trials in China

United States14 sites

  • Investigator site 0010112

    Chicago, Illinois, 60612

  • Investigator site 0010193

    Chicago, Illinois, 60637

  • Investigator site 0010040

    Columbus, Ohio, 43210

  • Investigator Site 0010083

    Detroit, Michigan, 48202

  • Investigator Site 0010062

    Fort Wayne, Indiana, 46804

  • Investigator site 0010042

    Iowa City, Iowa, 52242

  • Investigator Site 0010079

    Lisle, Illinois, 60187

  • Investigator site 0010036

    Los Angeles, California, 90033

  • Investigator Site 0010102

    Minneapolis, Minnesota, 55455

  • Investigator Site 0010095

    Oklahoma City, Oklahoma, 73142

  • Investigator Site 0010115

    Pittsburgh, Pennsylvania, 15222

  • Investigator Site 0010116

    Springdale, Arkansas, 72758

  • Investigator Site 0010045

    Washington D.C., District of Columbia, 20007

  • Investigator Site 0010104

    Weston, Florida, 33331

Other trials in United States

Italy14 sites

  • Investigator Site 0390037

    Alessandria

  • Investigator Site 0390043

    Ferrara

  • Investigator Site 0390045

    Meldola

  • Investigator site 0390014

    Milan, 20122

  • Investigator Site 0390032

    Milan

  • Investigator Site 0390041

    Naples

  • Investigator Site 0390044

    Naples

  • Investigator Site 0390015

    Novara

  • Investigator Site 0390035

    Potenza

  • Investigator Site 0390011

    Reggio Calabria

  • Investigator site 0390018

    Reggio Emilia, 42100

  • Investigator Site 0390046

    Rome

  • Investigator Site 0390033

    Terni

  • Investigator Site 0390036

    Varese

Other trials in Italy

Turkey (Türkiye)14 sites

  • Investigator Site 0900007

    Adapazarı

  • Investigator Site 0900003

    Ankara

  • Investigator Site 0900006

    Ankara

  • Investigator Site 0900008

    Ankara

  • Investigator Site 0900015

    Ankara

  • Investigator Site 0900016

    Edirne

  • Investigator Site 0900013

    Istanbul

  • Investigator Site 0900004

    Izmir

  • Investigator Site 0900014

    Kocaeli

  • Investigator Site 0900018

    Malatya

  • Investigator Site 0900010

    Mersin

  • Investigator Site 0900009

    Samsun

  • Investigator Site 0900017

    Tekirdağ

  • Investigator Site 0900019

    Trabzon

Other trials in Turkey (Türkiye)

Russia10 sites

  • Investigator Site 0070006

    Kaluga

  • Investigator Site 0070040

    Kirov

  • Investigator Site 0070026

    Moscow

  • Investigator Site 0070038

    Nizhny Novgorod

  • Investigator Site 0070037

    Novosibirsk

  • Investigator Site 0070024

    Pyatigorsk

  • Investigator Site 0070025

    Saint Petersburg

  • Investigator Site 0070039

    Smolensk

  • Investigator Site 0070015

    Syktyvkar

  • Investigator Site 0070012

    Tula

Other trials in Russia

Australia9 sites

  • Investigator Site 0610009

    Adelaide

  • Investigator Site 0610004

    Bedford Park

  • Investigator Site 0610002

    Box Hill

  • Investigator Site 0610010

    Clayton

  • Investigator Site 0610012

    Garran

  • Investigator Site 0610001

    Hobart

  • Investigator Site 0610011

    Perth

  • Investigator Site 0610003

    West Perth

  • Investigator Site 0610005

    Westmead

Other trials in Australia

Spain9 sites

  • Investigator Site 0340024

    Alava

  • Investigator Site 0340007

    Barcelona, 08035

  • Investigator Site 0340006

    Barcelona

  • Investigator Site 0340023

    Barcelona

  • Investigator Site 0340037

    Madrid

  • Investigator Site 0340022

    Murcia

  • Investigator Site 0340036

    Sabadell

  • Investigator site 0340013

    Seville, 41013

  • Investigator Site 0340004

    Valencia

Other trials in Spain

Romania8 sites

  • Investigator Site 0400005

    Bucharest

  • Investigator Site 0400006

    Bucharest

  • Investigator Site 0400009

    Bucharest

  • Investigator Site 0400012

    Bucharest

  • Investigator Site 0400016

    Cluj-Napoca

  • Investigator Site 0400007

    Craiova

  • Investigator Site 0400011

    Sibiu

  • Investigator Site 0400008

    Târgu Mureş

Other trials in Romania

Thailand8 sites

  • Investigator Site 0660002

    Bangkok

  • Investigator Site 0660003

    Bangkok

  • Investigator Site 0660005

    Bangkok

  • Investigator Site 0660008

    Bangkok

  • Investigator Site 0660001

    Bangkok Noi

  • Investigator Site 0660004

    Chiang Mai

  • Investigator Site 0660009

    Khon Kaen

  • Investigator Site 0660006

    Pathum Thani

Other trials in Thailand

Israel7 sites

  • Investigator Site 9720013

    Ashkelon

  • Investigator Site 9720010

    Haifa

  • Investigator Site 9720012

    Haifa

  • Investigator Site 9720008

    Jerusalem

  • Investigator Site 9720011

    Jerusalem

  • Investigator Site 9720007

    Petah Tikva

  • Investigator Site 9720009

    Tel Aviv

Other trials in Israel

Portugal7 sites

  • Investigator Site 3510006

    Braga

  • Investigator Site 3510003

    Coimbra

  • Investigator Site 3510002

    Lisbon

  • Investigator Site 3510005

    Lisbon

  • Investigator Site 3510007

    Lisbon

  • Investigator Site 3510001

    Porto

  • Investigator Site 3510004

    Porto

Other trials in Portugal

Georgia6 sites

  • Investigator Site 9950006

    Tbilisi

  • Investigator Site 9950007

    Tbilisi

  • Investigator Site 9950008

    Tbilisi

  • Investigator Site 9950009

    Tbilisi

  • Investigator Site 9950011

    Tbilisi

  • Investigator Site 9950019

    Tbilisi

Other trials in Georgia

Poland6 sites

  • Investigator Site 0480013

    Katowice, 40519

  • Investigator Site 0480014

    Lublin

  • Investigator Site 0480026

    Nowy Sącz

  • Investigator Site 0480037

    Skorzewo

  • Investigator Site 0480039

    Torun

  • Investigator Site 0480033

    Warsaw

Other trials in Poland

South Korea6 sites

  • Investigator Site 0820005

    Seongnam

  • Investigator Site 0820003

    Seoul

  • Investigator Site 0820004

    Seoul

  • Investigator Site 0820006

    Seoul

  • Investigator Site 0820007

    Seoul

  • Investigator Site 0820008

    Seoul

Other trials in South Korea

Mexico5 sites

  • Investigator Site 0520002

    Aguascalientes

  • Investigator Site 0520004

    Chihuahua City

  • Investigator Site 0520007

    México

  • Investigator Site 0520003

    Monterrey

  • Investigator Site 0520001

    Oaxaca City

Other trials in Mexico

South Africa5 sites

  • Investigator Site 0270005

    George

  • Investigator Site 0270003

    Johannesburg

  • Investigator Site 0270004

    Observatory

  • Investigator Site 0270001

    Pretoria

  • Investigator Site 0270002

    Randburg

Other trials in South Africa

United Kingdom5 sites

  • Investigator site 0440011

    Bradford, BD9 6RJ

  • Investigator Site 0440005

    Coventry

  • Investigator Site 0440008

    London

  • Investigator Site 0440041

    London

  • Investigator Site 0440014

    Truro

Other trials in United Kingdom

Greece4 sites

  • Investigator Site 0300008

    Athens

  • Investigator Site 0300010

    Athens

  • Investigator Site 0300007

    Pátrai

  • Investigator Site 0300009

    Thessaloniki

Other trials in Greece

Argentina3 sites

  • Investigator Site 0540001

    Buenos Aires

  • Investigator Site 0540004

    Buenos Aires

  • Investigator Site 0540003

    Córdoba

Other trials in Argentina

Chile3 sites

  • Investigator Site 0560002

    Santiago

  • Investigator Site 0560004

    Temuco

  • Investigator Site 0560003

    Viña del Mar

Other trials in Chile

Ireland3 sites

  • Investigator Site 3530002

    Dublin

  • Investigator Site 3530003

    Dublin

  • Investigator Site 3530001

    Galway

Other trials in Ireland

New Zealand3 sites

  • Investigator Site 0640001

    Auckland

  • Investigator Site 0640005

    Christchurch

  • Investigator Site 0640002

    Palmerston North

Other trials in New Zealand

Tunisia3 sites

  • Investigator Site 2160006

    Sfax

  • Investigator Site 2160001

    Sousse

  • Investigator Site 2160002

    Tunis

Other trials in Tunisia

Bulgaria2 sites

  • Investigator Site 3590017

    Plovdiv

  • Investigator Site 3590015

    Sofia

Other trials in Bulgaria

France2 sites

  • Investigator Site 0330009

    Créteil

  • Investigator Site 0330018

    Montpellier

Other trials in France

Germany2 sites

  • Investigator site 0490008

    Essen, 45147

  • Investigator Site 0490012

    Giessen

Other trials in Germany

Jordan2 sites

  • Investigator Site 9620002

    Amman

  • Investigator Site 9620001

    Irbid

Other trials in Jordan

Norway2 sites

  • Investigator Site 0470002

    Bergen

  • Investigator Site 0470003

    Oslo

Other trials in Norway

Serbia2 sites

  • Investigator Site 3810006

    Belgrade

  • Investigator Site 3810008

    Kragujevac

Other trials in Serbia

Taiwan2 sites

  • Investigator Site 8860001

    New Taipei City

  • Investigator Site 8860003

    Taoyuan

Other trials in Taiwan

Denmark1 site

  • Investigator Site 0450005

    Roskilde

Other trials in Denmark

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

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.

Being tested
Efgartigimod PH20 SC, Placebo PH20 SC
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Efgartigimod PH20 SCBiological

    Also called ARGX-113 PH20 SC

    Subcutaneous injection with efgartigimod PH20 SC

  • Placebo PH20 SCOther

    Subcutaneous injection with placebo PH20 SC

Study groups in NCT04687072
Study groupRoleWhat they receive
Efgartigimod PH20 SCExperimentalPatients receiving efgartigimod PH20 SC treatment
Receives: Biological: Efgartigimod PH20 SC
Placebo PH20 SCPlacebo comparatorPatients receiving placebo PH20 SC treatment
Receives: Other: Placebo PH20 SC

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Up to 6 weeks (between Weeks 19 and 24)

Secondary outcomes (23)

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

    Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population.

    Measured: Up to 24 weeks

  2. Percentage of Participants in the Overall Population (Chronic and Persistent 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.

    Measured: Up to 6 weeks (between Weeks 19 and 24)

  3. Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 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 ≥6 of the 8 analysis visits between weeks 17 and 24.

    Measured: Up to 8 weeks (between Weeks 17 and 24)

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

    A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time during the 24-week treatment period.

    Measured: Up to 24 weeks

  5. Extent of Disease Control Until Week 12 in the Overall Population

    Extent of disease control was defined as the number of cumulative weeks until Week 12 with platelet counts of ≥50×10^9/L in the overall population.

    Measured: Up to 12 weeks

  6. Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12

    A participant was considered a responder for this endpoint (i.e., had an overall platelet count response) if the participant had platelet counts of ≥50 × 10^9/L for ≥4 analysis visits at any time until Week 12.

    Measured: Up to 12 weeks

  7. Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population

    Change from Baseline at time point t = value at time point t - Baseline value. Baseline was defined as the last available value prior to first administration of the investigational medicinal product (IMP).

    Measured: Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33)

  8. Time to Platelet Count Response in the Overall Population

    Time to platelet count response, defined as the time to have 2 consecutive platelet counts of ≥50 × 10^9/L via Kaplan-Meier estimates.

    Measured: Up to 24 weeks

  9. Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population

    Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population.

    Measured: Up to 24 weeks

  10. Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L

    Extent of disease control was defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥30×10^9/L with at least ≥20×10^9/L above Baseline in the overall population.

    Measured: Up to 24 weeks

  11. Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population

    Assessed using the WHO bleeding scale. The WHO bleeding scale is a five-point scale where Grade 0 = no bleeding; Grade 1 = petechial bleeding; Grade 2 = mild blood loss; Grade 3 = gross blood loss (requires transfusion); and Grade 4 = debilitating blood loss, associated with fatality.

    Measured: Up to 24 weeks

  12. Percentage of Participants With a Platelet Count International Working Group (IWG) Response

    IWG complete response was defined as platelet counts of ≥100 × 10^9/L and the absence of bleeding events (WHO Grading = 0 [no bleeding]) for at least 2 separate, consecutive analysis visits at least 7 days apart.

    IWG response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase of platelet count from Baseline and the absence of bleeding events (WHO grading = 0) for at least 2 separate, consecutive analysis visits that were at least 7 days apart.

    Initial response was defined as platelet counts of ≥30 × 10^9/L and a 2-fold increase from the Baseline platelet count at analysis visit 5.

    Measured: Up to 24 weeks

  13. Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population

    Rescue therapy was defined as an occurrence where the participant needed treatment with 1 or more rescue treatments. An occurrence was defined as a period of maximum 5 days where 1 or more rescue treatments were administered simultaneously or consecutively to the trial participant. The following rescue treatments were permitted: methylprednisolone, dexamethasone, prednisone, normal immunoglobulins, anti-D (Rho) immunoglobins, or platelet transfusions.

    Measured: Up to 24 weeks

  14. Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population

    A change in ITP therapy was defined as either an increase in the dose and/or frequency of a concurrent ITP therapy relative to Baseline or the initiation of a new concurrent ITP therapy.

    Measured: Up to 13 weeks (between Weeks 12 and 24)

  15. Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population

    The FACIT-fatigue scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during his/her usual daily activities over the past week. The level of fatigue was measured by recording item responses on a 5-point Likert scale ranging from 0 "not at all" to 4 "very much". All items were summed to create a single fatigue score with a range from 0 to 52, where a higher FACIT-F score indicated more severe symptoms. A negative change score from Baseline indicated improvement in quality of life (QoL).

    Measured: Baseline and Week 24

  16. Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population

    The FACT-Th6 uses a 5-level Likert scale (0=not at all to 4=very much), with participants rating their degree of concern in the past 7 days. The 6 selected items pertain to ability to do usual activities, worry about problems with bleeding or bruising, worry about the possibility of serious bleeding, avoidance of physical or social activity because of concern with bleeding or bruising and frustration due to the inability to carry out usual activities. All items were summed to create a single score with a range from 0 to 24, where a higher score indicated less severe symptoms. A positive change score from Baseline indicated improvement in QoL.

    Measured: Baseline and Weeks 4, 8, 12, 16, 20, and 24

  17. Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population

    The SF-36 is a 36-item scale constructed to survey health-related QoL on 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations in usual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions. The scores from the 8 domains were evaluated independently and aggregated into 2 norm-based summary component measures of physical and mental health. The summary component scores could range from 0 to 100, where a higher score indicated improvement in QoL. A positive change score from Baseline indicated improvement in QoL.

    Measured: Baseline and Week 24

  18. Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population

    Anti-drug antibody (ADA) incidence was defined as the percentage of participants with treatment-induced or treatment boosted ADA (denominator: number of evaluable participants). ADA prevalence was defined as the percentage of participants with treatment-unaffected ADA, treatment-induced ADA or treatment-boosted ADA (denominator: number of evaluable participants).

    Measured: Up to 35 weeks

  19. Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population

    A titer was determined in the samples with a positive assay response.

    Measured: Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33)

  20. Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population

    Samples were tested for the presence of NAb against efgartigimod and/or rHuPH20 and titers for NAb against rHuPH20.

    NAb incidence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive" and "baseline positive-postbaseline positive". NAb prevalence is defined as the total percentage of participants with participant classification "baseline negative-postbaseline positive," "baseline positive-postbaseline positive," or "baseline positive-postbaseline negative".

    Measured: Up to 35 weeks

  21. Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population

    All pharmacokinetic (PK) samples were collected predose, on the day of IMP administration.

    Measured: Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24

  22. Percentage Change From Baseline in Total IgG in the Overall Population

    Samples were collected predose, on the day of IMP administration.

    Measured: Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24

  23. Number of Participants With Antiplatelet Antibodies in the Overall Population

    The antiplatelet antibody was positive if optical density value >0.129.

    Measured: Weeks 7, 15, 23, and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Dec 2020
  2. Study start16 Dec 2020
  3. Primary completion9 Oct 2023
  4. Study completion9 Oct 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
207 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04687072
Sponsor's protocol ID
ARGX-113-2004
Other IDs
2020-004032-21
Acronym
ADVANCE SC
Status last verified
Oct 2024
First submitted
18 Dec 2020
Last updated
31 Oct 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Cooper N, Broome CM, Miyakawa Y, McDonald V, Al-Samkari H, Khelif A, Cataland SR, Barcellini W, Yang R, Mei H, Matthijssens F, Hultberg A, Ciurlia G, Gandini D, Ayguasanosa J, De Beuf K, Pastouret S, Ghanima W, Rodeghiero F, Bussel JB; ADVANCE SC Investigator Trial Group. Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Adults With Primary Immune Thrombocytopenia (ADVANCE SC): A Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Phase 3 Trial. Am J Hematol. 2026 Jul;101(7):1494-1506. doi: 10.1002/ajh.70303. Epub 2026 Apr 6.

    PubMed 41943179

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Efgartigimod PH20 SC in people with Primary Immune Thrombocytopenia, run by argenx at 200 sites in 32 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 200 sites in Japan, China, United States, Italy, Turkey (Türkiye) and 27 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 31 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24 (time frame: Up to 6 weeks (between Weeks 19 and 24)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04687072, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Oct 2024.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in