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.
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
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 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.
- 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)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
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