Primary immune thrombocytopenia (ITP) is a condition where the immune system mistakenly destroys platelets, which are cells that help stop bleeding. This leads to a low number of platelets, making it easier to bruise or bleed. The main aim of this study is to learn whether mezagitamab, when given just under the skin (subcutaneously \[SC\]), is effective in keeping the platelet count of adults with ITP stable when compared to a placebo. A placebo looks like medicine but doesn't have any active ingredients in it.
The participants will be treated with mezagitamab for up to 6 months.
During the study, participants will visit their study clinic several times.
Participants who complete the TAK-079-3002 study or do not have any response to study treatment by week 16 (according to study criteria) will be given the opportunity to participate in a continuation study to receive open label mezagitamab (if they are eligible and the site is able to open the continuation study).
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria:
1. The participant has been diagnosed with ITP that has persisted for at least 12 months.
2. The participant's diagnosis of ITP is supported by a prior response to an ITP therapy (not including a thrombopoietin receptor agonist \[TPO-RA\]), defined as having achieved a platelet count ≥50,000/μL.
3. The participant has evidence of insufficient response or intolerance to at least 1 currently available first-line therapy for treatment of ITP (for example, corticosteroids), and at least 1 currently available second-line therapy for treatment of ITP (for example, TPO-RA, rituximab, fostamatinib, mycophenolate). Insufficient response to previous treatment is defined as failure to achieve a sustained platelet count of at least 50,000/μL or doubling of baseline platelet count after an appropriate course of prior ITP treatment. Intolerance is defined as a documented side effect causing discontinuation of the therapy.
4. The participant has a mean platelet count of less than (\<)30,000/μL.
5. If the participant is receiving allowed standard-of-care treatment for ITP at screening, and continued use is intended, treatment may continue during the trial if the dose, and frequency have been stable for at least 4 weeks before receiving the first dose of IMP (i.e., Day 1), and are expected to remain stable throughout the trial.
6. If the participant is an individual with potential for pregnancy, the participant is not pregnant as confirmed by negative human chorionic gonadotropin during screening, and before the first dose of trial intervention.
Key Exclusion Criteria:
1. The participant has secondary ITP.
2. The participant has had any thrombotic or embolic event within 12 months before signing the informed consent form (ICF).
3. The participant has had a splenectomy.
4. The participant has active infection with hepatitis B virus, hepatitis C virus, or human immunodeficiency virus (HIV).
5. History of malignancy (including myelodysplastic syndrome) within 5 years of signing the ICF, except for treated non-melanoma skin cancer or cervical carcinoma in situ.
6. In the opinion of the investigator, the participant has a serious medical or psychiatric illness that could potentially interfere with the completion of treatment according to this protocol.
7. The participant has received anti-cluster of differentiation (CD) 20 treatment within 12 months before screening, and either of the following applies:
1. The last dose was received within 6 months before screening.
2. The last dose was received between 6 and 12 months before screening, and the participant has a cluster of differentiation 19 positive (CD19+) count below the lower limit of normal.
8. The participant has received any monoclonal or polyclonal antibody for immunomodulation within 6 months before screening.
9. The participant has any prior exposure to mezagitamab or has been exposed to another investigational agent within 4 weeks or 5 half-lives, whichever is longer, before Day 1.
10. The participant has used anticoagulants (e.g., vitamin K antagonists, direct oral anticoagulants) within 3 weeks prior to the first dose of trial treatment.
11. The participant has received a live or live-attenuated vaccine within 4 weeks prior to the first dose of trial treatment or has any live or live-attenuated vaccine planned during the trial.
12. The participant has used the following immunosuppressive agents as specified prior to the first dose of trial treatment: alkylating agents (e.g., cyclophosphamide) within 8 weeks, vinca alkaloids (e.g., vincristine) within 4 weeks, sulfones (e.g., dapsone) within 3 weeks, antiproliferative agents: (e.g., mycophenolate mofetil, and azathioprine) within 2 weeks, and calcineurin inhibitors: (e.g., cyclosporine) within 2 weeks.
13. The participant has used intravenous immunoglobulin (IVIg), SC immunoglobulin, recombinant human thrombopoietin, anti-D immunoglobulin treatment, or efgartigimod within 4 weeks before signing the ICF or it is expected that any treatment for thrombocytopenia other than the participant's standard-of-care ITP therapy (e.g., rescue therapy, administration of blood products) may be used between screening, and Day 1.
14. The participant has a history of severe allergic or anaphylactic reactions to recombinant proteins or excipients used in the mezagitamab/placebo formulation.
Other protocol defined inclusion/exclusion criteria may apply.
Primary outcome measure(s)
Percentage of Participants With Durable Platelet Response — Up to Week 24 Durable platelet response is defined as platelet count greater than or equal to (≥)50,000/microliter (μL) on at least 4 of the 6 weekly platelet measurements between Weeks 19 and 24.
Trial sites (124)
Facility
City
Region
Status
Genesis Cancer and Blood Institute - SCRI
Hot Springs
Arkansas
Recruiting
USC Norris Comprehensive Cancer Center - Keck Medicine of USC
Los Angeles
California
Recruiting
University of California San Diego Center for Bleeding and Clotting Disorders
San Diego
California
Not Yet Recruiting
Rocky Mountain Cancer Center
Denver
Colorado
Withdrawn
Georgetown University Medical Center - Lombardi Comprehensive Cancer Center
Washington D.C.
District of Columbia
Recruiting
Emory University
Atlanta
Georgia
Recruiting
Innovative Hematology, Inc.
Indianapolis
Indiana
Not Yet Recruiting
The University of Iowa
Iowa City
Iowa
Recruiting
University Of Louisville Brown Cancer Center
Louisville
Kentucky
Recruiting
American Oncology Partners of Maryland, PA
Bethesda
Maryland
Recruiting
Massachusetts General Hospital
Boston
Massachusetts
Recruiting
University of Massachusetts Chan Medical School
Worcester
Massachusetts
Completed
MidAmerica Cancer Center
Kansas City
Missouri
Recruiting
Memorial Sloan Kettering Cancer Center
New York
New York
Not Yet Recruiting
Montefiore Medical Center
The Bronx
New York
Not Yet Recruiting
Duke University Hospital
Durham
North Carolina
Recruiting
East Carolina University
Greenville
North Carolina
Recruiting
Cleveland Clinic
Cleveland
Ohio
Not Yet Recruiting
Oregon Health & Science University
Portland
Oregon
Recruiting
Perelman Center for Advanced Medicine (PCAM) Hospital of The University of Pennsylvania Penn Blood Disorders Program
Philadelphia
Pennsylvania
Recruiting
Lewis Katz School of Medicine at Temple University
Philadelphia
Pennsylvania
Recruiting
Baylor College of Medicine
Houston
Texas
Recruiting
Virginia Oncology Associates
Norfolk
Virginia
Recruiting
University of Washingto
Seattle
Washington
Not Yet Recruiting
Versiti Wisconsin, Inc
Milwaukee
Wisconsin
Recruiting
Canberra Hospital
Garran
Australian Capital Territory
Recruiting
Concord Repatriation General Hospital
Concord
New South Wales
Recruiting
St George Hospital
Kogarah
New South Wales
Recruiting
University of New South Wales (UNSW) - Liverpool Hospital - Liverpool Cancer Therapy Centre
Liverpool
New South Wales
Recruiting
Westmead Hospital
Westmead
New South Wales
Recruiting
Peter MacCallum Cancer Centre
Melbourne
Victoria
Not Yet Recruiting
Monash University - Australian Centre for Blood Diseases (ACBD)
Melbourne
Victoria
Recruiting
The Alfred Hospital
Melbourne
Victoria
Recruiting
Fiona Stanley Hospital
Murdoch
Western Australia
Recruiting
Perth Blood Institute
West Perth
Western Australia
Recruiting
Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia
Sofia
Sofia-Grad
Recruiting
Medical Center "Fama Medical"
Plovdiv
Bulgaria
Recruiting
UMHAT Sv. Ivan Rilski
Sofia
Bulgaria
Recruiting
UMHAT SofiaMed, OOD
Sofia
Bulgaria
Recruiting
University Multiprofile Hospital for Active Treatment - Prof. Dr. Stoyan Kirkovich AD
Stara Zagora
Bulgaria
Recruiting
+ 84 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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