- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03102593
A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening.
The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Vienna, Austria
- Vienna
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Wien, Austria
- Wien
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Leuven, Belgium
- Leuven
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Namur, Belgium
- Mont-Godinne
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Brno, Czechia
- Brno
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Praha, Czechia
- Praha
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Bordeaux, France
- Bordeaux
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Grenoble, France
- Grenoble
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Paris, France
- Paris
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Berlin, Germany
- Berlin
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Hanover, Germany
- Hanover
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Tubingen, Germany
- Tübingen
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Budapest, Hungary
- Budapest
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Debrecen, Hungary
- Debrecen
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Gyula, Hungary
- Gyula
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Kaposvar, Hungary
- Kaposvar
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Nyiregyhaza, Hungary
- Nyíregyháza
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Pecs, Hungary
- Pecs
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Lublin, Poland
- Lublin
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Opole, Poland
- Opole
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Wroclaw, Poland
- Wroclaw
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A Coruña, Spain
- A Coruna
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Barcelona, Spain
- Barcelona
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Madrid, Spain
- Madrid
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Valencia, Spain
- Valencia
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Dnipro, Ukraine
- Dnipro
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Ivano-Frankivsk, Ukraine
- Ivano-Frankivsk
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Nikolaev, Ukraine
- Nikolaev
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Uzhgorod, Ukraine
- Uzhgorod
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London, United Kingdom
- London
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Male or female patients aged ≥ 18 to ≤ 85 years.
- Must receive SoC treatment for ITP that has been stable in dose and frequency for at least 4 weeks prior to Screening. SoC may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist.
- Confirmed diagnosis of ITP with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.
Exclusion Criteria:
- Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
- Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
- Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
- Use of recombinant thrombopoietin at any time.
- Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
- Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine, danazol, mycophenolate mofetil, mycophenolate sodium which must have been stable for at least 4 weeks prior to Screening.
- Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
- Received vaccinations within 4 weeks prior to Screening or planned during the study.
- At Screening, have clinically significant laboratory abnormalities
- History of any thrombotic or embolic event within 12 months prior to Screening.
- Known auto-immune disease other than ITP.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: ARGX-113 Dose A + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
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ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
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Experimental: ARGX-113 Dose B +SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
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ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
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Placebo Comparator: Placebo + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
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ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Incidence and severity of serious adverse events (SAEs).
Time Frame: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
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Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
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After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Frequency and proportion of patients with initial response
Time Frame: Over the study period (up to 13 weeks).
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Mean change from Baseline in platelet counts
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Over the study period (up to 13 weeks).
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Chair: Adrian Newland, Barts Hospital, Cancer Centre in London
Publications and helpful links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura, Thrombocytopenic
- Purpura
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
Other Study ID Numbers
- ARGX-113-1603
- 2016-003038-26 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Novartis PharmaceuticalsRecruitingPrimary Immune Thrombocytopenia (ITP)Spain, Italy, France, Germany, China, Korea, Republic of, Czechia, Turkey, Malaysia, Argentina, Australia, United Kingdom, Poland, United States
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Institute of Hematology & Blood Diseases Hospital...Xijing Hospital; Xi'an Central Hospital; The Second Hospital of Hebei Medical... and other collaboratorsActive, not recruitingPreviously Treated Primary Immune ThrombocytopeniaChina
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Hutchison Medipharma LimitedActive, not recruiting
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Institute of Hematology & Blood Diseases HospitalHenan Cancer Hospital; Nantong University; Tianjin Medical University Second... and other collaboratorsUnknownPrimary Immune Thrombocytopenia (ITP)China
Clinical Trials on ARGX-113
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argenxCompleted
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argenxQuintiles, Inc.CompletedMyasthenia GravisItaly, United States, Belgium, Canada, Netherlands, Poland, Spain, Sweden
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argenxCompletedPrimary Immune ThrombocytopeniaUnited States, Austria, Belgium, Bulgaria, Czechia, France, Georgia, Germany, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, Turkey, Ukraine, United Kingdom
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