Primary Immune Thrombocytopenia (ITP)Phase 3ADVANCE2NCT04274452

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

A Phase 3 study testing efgartigimod in people with Primary Immune Thrombocytopenia (ITP), run by argenx.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial summary

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in 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 (ITP)
What is being tested?: efgartigimod
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 0 participants
Where: No sites listed yet
Study dates: 29 Jun 2020to 1 Sep 2021
Phase: Phase 3
Are there results?: None posted in the registry

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 (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).
Male/female aged ≥18 years at the time the informed consent form (ICF) is signed.
Confirmed diagnosis of ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and without a 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 <30×10^9/L from 2 counts during the screening period including the predose count at visit 1. Additionally, a documented history of a platelet count of <30×109/L prior to screening.
At the start of the trial, the patient either takes concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient does not take treatment for ITP but has received at least 2 prior treatments for ITP. Patients receiving permitted concurrent ITP treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization. Permitted concurrent ITP medications include oral corticosteroids, oral immunosuppressants, dapsone/danazol, fostamatinib, and/or oral TPO-RAs. Patients not receiving concurrent ITP therapy are also eligible for the trial if they have not received prior ITP therapy for at least 4 weeks prior to baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (eg, rituximab).
Women of childbearing potential: Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before trial medication can be administered. Women must be on a stable regimen for at least 1 month of at least 1 highly effective method of contraception (ie, failure rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP.
Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective contraception from signing the ICF through 90 days after the last administration of the IMP. Male patients practicing true sexual abstinence (as consistent with preferred and usual lifestyle) can be included. Sterilized male patients who have had a vasectomy and with documented absence of sperm postprocedure can be included. Male patients are not allowed to donate sperm from signing the ICF through 90 days after the last dose of the IMP.

Who cannot join

ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
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, subcutaneous, or intramuscular route) or plasmapheresis (PLEX), 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 22Show 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 within the 6 months before the first dose of the IMP.
At the screening visit, clinically significant laboratory abnormalities as below: Hemoglobin ≤9 g/dL - OR - International normalized ratio >1.5 or activated partial thromboplastin time >1.5×ULN - OR - Total IgG <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 the IMP. Patients 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, 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 a major thrombotic or embolic event (eg, myocardial infarction, stroke, deep venous thrombosis, or pulmonary embolism) within 12 months prior to randomization.
History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
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 patient 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, Hepatitis C virus (HCV) based on HCV-antibody assay, 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.
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 patient at undue risk.
Known hypersensitivity reaction to efgartigimod or 1 of its excipients.
Previously participated in a clinical trial with efgartigimod.
Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of the IMP.
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 patient at undue risk.
Current or history of (ie, within 12 months of screening) alcohol, drugs, or medication abuse.

About this study

Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP)

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in patients with primary ITP.

Conditions: Primary Immune Thrombocytopenia (ITP)
Being tested: efgartigimod, Placebo
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.

efgartigimodBiological
Also called ARGX-113
Intravenous infusion of efgartigimod
PlaceboOther
Intravenous infusion of placebo

Study groups in NCT04274452
Study group	Role	What they receive
efgartigimod	Experimental	Patients receiving an intravenous infusion of efgartigimod Receives: Biological: efgartigimod
placebo	Placebo comparator	Patients receiving an intravenous infusion of placebo Receives: Other: Placebo

What is being measured

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

Number of cumulative weeks over the planned 24 week treatment period with platelet counts of ≥50×10^9/L in patients with chronic ITP.
Measured: Up to 24 weeks

Secondary outcomes (21)

Number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^09/L in the overall population (chronic and persistent ITP).
Measured: Up to 24 weeks
Proportion of patients in the overall population with a sustained platelet count response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial
Measured: Up to 6 weeks
Proportion of patients in the overall population achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial
Measured: Up to 8 weeks
Proportion of patients in the overall population 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 treatment period
Measured: Up to 24 weeks
Extent of disease control 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
Proportion of patients in the overall population with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time until week 12
Measured: Up to 12 weeks
Mean change from baseline in platelet count at each visit in the overall population.
Measured: Up to 31 weeks
Time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L in the overall population
Measured: Up to 31 weeks
The number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Measured: Up to 24 weeks
In patients with baseline platelet count of <15×10^9/L, the number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Measured: Up to 31 weeks
Incidence and severity of the World Health Organization (WHO)-classified bleeding events in the overall population
Measured: Up to 31 weeks
Rate of receipt of rescue therapy (rescue per patient per month)
Measured: Up to 31 weeks
Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have increased at week 12 or later
Measured: Up to 24 weeks
Incidence and severity of adverse events (AEs), AEs of special interest (AESIs)and serious AEs (SAEs).
Measured: Up to 31 weeks
Vital signs, electrocardiogram (ECG), and laboratory assessments
Measured: Up to 31 weeks
Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) at planned visits
Measured: Up to 31 weeks
Change from baseline in QoL (SF-36) at planned visits
Measured: Up to 31 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod
Measured: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : maximum observed serum concentration (Cmax)
Measured: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : serum concentration observed predose (Cthrough)
Measured: Up to 31 weeks
Pharmacodynamics markers: total immunoglobulin (Ig) G, IgG isotypes (IgG1, IgG2, IgG3, IgG4), antiplatelet antibody levels
Measured: Up to 31 weeks

Timeline

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

29 Jun 20201 Sep 2021

First posted18 Feb 2020
Study start29 Jun 2020
Primary completion1 Sep 2021 (estimated)
Study completion1 Sep 2021 (estimated)

Why it stopped: The ARGX-113-1908 study will be replaced by a similar design with a subcutaneous formulation (ARGX-113-2004)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
More trials run by argenxThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04274452
Sponsor's protocol ID: ARGX-113-1908
Acronym: ADVANCE2
Status last verified: Sep 2020
First submitted: 14 Feb 2020
Last updated: 14 Sep 2020
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

All Primary Immune Thrombocytopenia (ITP) trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP)
ClinicalTrials.gov ID: NCT04274452
Acronym / sponsor ID: ADVANCE2 / ARGX-113-1908
Conditions: Primary Immune Thrombocytopenia (ITP)
Phase: Phase 3
Study type: Interventional
Recruitment status: WithdrawnReason stopped: The ARGX-113-1908 study will be replaced by a similar design with a subcutaneous formulation (ARGX-113-2004)
Enrolment: 0 participants (actual)
Lead sponsor: argenxIndustry (pharma, biotech, devices)
Study start: 29 Jun 2020 (actual)
Primary completion: 1 Sep 2021 (estimated)
Study completion: 1 Sep 2021 (estimated)
First posted: 18 Feb 2020 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in patients with primary ITP.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
efgartigimod	Experimental	Biological: efgartigimodPatients receiving an intravenous infusion of efgartigimod
placebo	Placebo comparator	Other: PlaceboPatients receiving an intravenous infusion of placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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).
Male/female aged ≥18 years at the time the informed consent form (ICF) is signed.
Confirmed diagnosis of ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and without a 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 <30×10^9/L from 2 counts during the screening period including the predose count at visit 1. Additionally, a documented history of a platelet count of <30×109/L prior to screening.

Key exclusion criteria (22 listed)

ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
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, subcutaneous, or intramuscular route) or plasmapheresis (PLEX), 4 weeks prior to randomization.
Use of romiplostim within 4 weeks prior to randomization.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
efgartigimodAlso: ARGX-113Intravenous infusion of efgartigimod	Biological	efgartigimod
PlaceboIntravenous infusion of placebo	Other	placebo

Primary outcome

Number of cumulative weeks over the planned 24 week treatment period with platelet counts of ≥50×10^9/L in patients with chronic ITP.
Time frame: Up to 24 weeks

21 secondary outcomes

Number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^09/L in the overall population (chronic and persistent ITP).
Time frame: Up to 24 weeks
Proportion of patients in the overall population with a sustained platelet count response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial
Time frame: Up to 6 weeks
Proportion of patients in the overall population achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial
Time frame: Up to 8 weeks
Proportion of patients in the overall population 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 treatment period
Time frame: Up to 24 weeks
Extent of disease control defined as the number of cumulative weeks until week 12, with platelet counts of ≥50×10^9/L in the overall population
Time frame: Up to 12 weeks
Proportion of patients in the overall population with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time until week 12
Time frame: Up to 12 weeks
Mean change from baseline in platelet count at each visit in the overall population.
Time frame: Up to 31 weeks
Time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L in the overall population
Time frame: Up to 31 weeks
The number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Time frame: Up to 24 weeks
In patients with baseline platelet count of <15×10^9/L, the number of cumulative weeks over the treatment period with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline in the overall population
Time frame: Up to 31 weeks
Incidence and severity of the World Health Organization (WHO)-classified bleeding events in the overall population
Time frame: Up to 31 weeks
Rate of receipt of rescue therapy (rescue per patient per month)
Time frame: Up to 31 weeks
Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have increased at week 12 or later
Time frame: Up to 24 weeks
Incidence and severity of adverse events (AEs), AEs of special interest (AESIs)and serious AEs (SAEs).
Time frame: Up to 31 weeks
Vital signs, electrocardiogram (ECG), and laboratory assessments
Time frame: Up to 31 weeks
Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) at planned visits
Time frame: Up to 31 weeks
Change from baseline in QoL (SF-36) at planned visits
Time frame: Up to 31 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod
Time frame: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : maximum observed serum concentration (Cmax)
Time frame: Up to 31 weeks
Pharmacokinetic parameters of efgartigimod : serum concentration observed predose (Cthrough)
Time frame: Up to 31 weeks
Pharmacodynamics markers: total immunoglobulin (Ig) G, IgG isotypes (IgG1, IgG2, IgG3, IgG4), antiplatelet antibody levels
Time frame: Up to 31 weeks

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2020.
Individual participant dataSharing planned: Undecided

Related trials for Primary Immune Thrombocytopenia (ITP)

RecruitingPhase 2
A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP)
Primary Immune Thrombocytopenia (ITP), Primary Evans Syndrome (ES)
RecruitingPhase 1/2
A Study of Pirtobrutinib in Participants With Immune Thrombocytopenia
Immune Thrombocytopenia (ITP)
RecruitingPhase 3
A Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)
RecruitingPhase 2
Study to Assess the Safety and Tolerability of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders
Immune Thrombocytopenia
RecruitingPhase 3
A Follow-up Study of Mezagitamab in Adults With Chronic Primary Immune Thrombocytopenia
Immune Thrombocytopenic Purpura (ITP)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing efgartigimod in people with Primary Immune Thrombocytopenia (ITP), run by argenx.

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 8 inclusion and 22 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: withdrawn, as of the sponsor's last update on 14 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.

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?

The study was withdrawn before enrolling participants, so there are no results.

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?

Number of cumulative weeks over the planned 24 week treatment period with platelet counts of ≥50×10^9/L in patients with chronic ITP. (time frame: Up to 24 weeks). 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 NCT04274452.

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 14 Sep 2020.