A Study of TAK-079 in Adults With Persistent/Chronic Primary Immune Thrombocytopenia

A Phase 2, Randomized, Double-blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Patients With Persistent/Chronic Primary Immune Thrombocytopenia

Primary immune thrombocytopenia (ITP) is a rare disease that results in low levels of platelets - the cells that help blood clot.

The main aim of the study is to check for side effects from taking TAK-079 at three different dose levels. Another aim is to learn if TAK-079 can increase the platelet count in people with ITP.

In addition to receiving stable background therapy for ITP, participants will receive an injection of either TAK-079 or a placebo once a week for 2 months. A placebo looks like TAK-079 but will not have any medicine in it. After treatment, all participants will be followed-up for another 2 months.

Then, participants who received TAK-079 will continue to be followed-up for an extra 4 months. Participants who received the placebo and would like to receive TAK-079 may be able to do this in an extension period in the study.

Study Overview

• Status: Completed
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Drug: Placebo; Drug: TAK-079

Detailed Description

The drug being tested in this study is called TAK-079. TAK-079 is being tested to treat people who have primary immune thrombocytopenia (ITP). This study will evaluate the safety and biologic activity of TAK-079 or matching placebo in combination with stable ITP background therapy.

The study will enroll approximately 36 to 54 participants. In Part A of the study, participants will be randomly assigned (by chance, like flipping a coin) to one of the three treatment groups. Those who received placebo in this period will have the choice to receive TAK-079 after a safety follow-up period and will be randomized to one of the two open-label TAK-079 treatment arms. An unblinded safety review will take place once a minimum of 24 evaluable participants are available for analysis in Part A to decide whether to open enrollment into Part B.

In Part B participants will be randomly assigned to one of two treatment groups. Those who received placebo in this period will have the choice to receive study drug after a safety follow-up period in a single open-label TAK-079 treatment arm.

This multi-center trial will be conducted worldwide. All participants will be followed for at least 8 weeks in a Safety Follow-up Period, and a 16-week Long-term Follow-up Period after the 8 weeks of treatment.

• Study Type: Interventional
• Enrollment (Actual): 41
• Phase: Phase 2

Contacts and Locations

Study Locations

• Bulgaria
  • Pleven, Bulgaria, 5800
    • University Multiprofile Hospital for Active Treatment - Dr. Georgi Stranski EAD
  • Plovdiv, Bulgaria, 4002
    • University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
  • Sofia, Bulgaria, 1431
    • University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD
  • Sofia, Bulgaria, 1606
    • Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia
  • Sofia-Grad
    • Sofia, Sofia-Grad, Bulgaria, 1407
      • Acibadem City Clinic Multiprofile Hospital for Active Treatment Tokuda
    • Sofia, Sofia-Grad, Bulgaria, 1750
      • University Multiprofile Hospital for Active Treatment Sofiamed OOD
• China
  • Hubei
    • Wuhan, Hubei, China, 430022
      • Union Hospital Tongji Medical College Huazhong University of Science and Technology
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences
• Croatia
  • Osijek, Croatia, 31000
    • Clinical Hospital Centre Osijek
  • Zagreb, Croatia, 10000
    • University Hospital Merkur
  • Zagreb, Croatia, 10000
    • Klinicki bolnicki centar Zagreb
• Germany
  • Berlin, Germany, 10707
    • Onkologische Schwerpunktpraxis Kurfürstendamm
  • Marburg, Germany, 35037
    • OnkoNet Marburg GmbH
  • Munchen, Germany, 80634
    • Rotkreuzklinikum München
  • Hessen
    • Frankfurt am Main, Hessen, Germany, 60590
      • Universitätsklinikum Frankfurt
• Greece
  • Thessaloniki, Greece, 57010
    • Georgios Papanikolaou General Hospital of Thessaloniki
  • Achaia
    • Patra, Achaia, Greece, 26500
      • University General Hospital of Patras
  • Attiki
    • Athens, Attiki, Greece, 115 27
      • General Hospital of Athens - George Gennimatas
• Italy
  • Bologna, Italy, 40138
    • Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi
  • Napoli, Italy, 80131
    • Azienda Ospedaliera Universitaria Federico II
  • Novara, Italy, 20100
    • A.O.U. Maggiore della Carità
  • Roma, Italy, 161
    • Azienda Policlinico Umberto I
  • Friuli-Venezia Giulia
    • Trieste, Friuli-Venezia Giulia, Italy, 34149
      • Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI)
  • Lombardia
    • Milano, Lombardia, Italy, 20122
      • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
  • Sicilia
    • Catania, Sicilia, Italy, 95122
      • Azienda Ospedaliera di Rilievo Nazionale e di Alta Specializzazione Garibaldi
• Japan
  • Tokyo
    • Itabashi, Tokyo, Japan, 173-8610
      • Nihon University Itabashi Hospital
    • Minato-Ku, Tokyo, Japan, 108-0073
      • Saiseikai Central Hospital
• Slovenia
  • Ljubljana, Slovenia, 1000
    • Univerzitetni klinicni center Ljubljana
  • Maribor, Slovenia, 2000
    • University Clinical Centre Maribor
• Spain
  • Barcelona, Spain, 08003
    • Hospital del Mar
  • Barcelona, Spain, 8041
    • Hospital De La Santa Creu I Sant Pau
  • Burgos, Spain, 9005
    • C.A.U de Burgos - Hospital Universitario de Burgos
  • Madrid, Spain, 28034
    • Hospital Universitario Ramon y Cajal
  • Madrid, Spain, 28007
    • Hospital General Universitario Gregorio Marañon
  • Madrid, Spain, 28026
    • Hospital Universitario 12 de Octubre
  • Madrid, Spain, 28006
    • Hospital Universitario Quirónsalud Madrid
  • Malaga, Spain, 29010
    • Hospital Universitario Virgen del Rocio - PPDS
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe de Valencia
  • Asturias
    • Oviedo, Asturias, Spain, 33006
      • Hospital Universitario Central de Asturias
  • Barcelona
    • Sabadell, Barcelona, Spain, 8208
      • Corporacio Sanitaria Parc Tauli
  • Madrid
    • Meco, Madrid, Spain, 28880
      • Hospital Universitario Príncipe de Asturias
• Ukraine
  • Dnipro, Ukraine, 49102
    • Municipal Non-profit Enterprise "City Clinical Hospital # 4" of Dnipro City Council - PPDS
  • Kyiv, Ukraine, 2091
    • Medical Center OK!Clinic+LLC International Institute of Clinical Research
  • Kyiv, Ukraine, 4060
    • CNE Kyiv City Clinical Hospital #9 of Exec. Body of Kyiv City Council Kyiv City State Admin
  • Lviv, Ukraine, 79044
    • State Institution Institute of Blood Pathology and Transfusion Medicine of NAMS of Ukraine
  • Mykolaivs'ka Oblast
    • Mykolaiv, Mykolaivs'ka Oblast, Ukraine, 54058
      • Municipal Non-profit Enterprise Mykolayiv Regional Clinical Hospital the Mykolayiv Regional Council
  • Ternopil's'ka Oblast
    • Ternopil, Ternopil's'ka Oblast, Ukraine, 46002
      • Municipal Non-profit Enterprise Ternopil Regional Clinical Hospital of Ternopil Regional Council
  • Zhytomyrs'ka Oblast
    • Zhytomyr, Zhytomyrs'ka Oblast, Ukraine, 10002
      • MNE Regional Clinical Hospital n a O F Herbachevskyi of Zhytomyr Regional Council
• United States
  • Arizona
    • Tucson, Arizona, United States, 85715
      • Arizona Clinical Research Center - Hunt - PPDS
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Illinois
    • Peoria, Illinois, United States, 61614-3542
      • Bleeding and Clotting Disorders Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02118-2905
      • Boston Medical Center
  • North Carolina
    • Greenville, North Carolina, United States, 27834
      • Leo W. Jenkins Cancer Center
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • University of Virginia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosed with ITP that has persisted for ≥3 months, diagnosed in accordance to The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia as locally applicable.
2. Has a mean platelet count of <30,000/μL (and individually ≤35,000/μL) on at least 2 measurements at least 1 week apart during screening.
3. Diagnosis of ITP supported by a prior response to an ITP therapy (other than a thrombopoietin receptor agonists [TPO-RA]) that achieved a platelet count of ≥50,000/μL.

4. If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least 4 weeks before dosing.

   1. Permitted standard background treatments may include: 1 oral corticosteroid; ±1 immunosuppressant from the following list: azathioprine, danazol, dapsone, cyclosporine, mycophenolate mofetil, mycophenolate sodium; ±1 TPO-RA (romiplostim, eltrombopag, avatrombopag); ±fostamatinib. Corticosteroids, including dexamethasone, must be given as oral, daily or every-other-day therapy as opposed to pulse therapy.
   2. The dose of any permitted standard background therapy must be expected to remain stable through the study, unless dose reduction is required because of toxicities.

Exclusion Criteria:

1. Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within 3 weeks before screening.
2. Has a history of any thrombotic or embolic event within 12 months before screening.
3. Has a history of splenectomy within 3 months before screening.
4. Use of intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin or anti-D immunoglobulin treatment within 4 weeks of screening, or an expectation that any therapy besides the participant's standard background therapies may be used for treatment of thrombocytopenia (e.g., a rescue therapy) between screening and dosing.
5. Diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 second (FEV1) <50% of predicted normal.
6. Use of rituximab or any monoclonal antibody (mAb) for immunomodulation within 4 months before first dosing. Note: Participants with prior exposure to rituximab must have cluster of differentiation (CD) 19 counts within the normal range at screening.
7. Use of immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing.
8. Has been diagnosed with myelodysplastic syndrome.
9. Has received a live vaccine within 4 weeks before screening or has any live vaccine planned during the study.

10 Has had an opportunistic infection ≤12 weeks before initial study dosing or is currently undergoing treatment for a chronic opportunistic infection, such as tuberculosis (TB), pneumocystis pneumonia, cytomegalovirus, herpes simplex virus, herpes zoster, or atypical mycobacteria.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

7

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Part A & B: Double Blind Period: Placebo; Participants received TAK-079 placebo-matching injection subcutaneously (SC), once weekly (QW) for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded short follow-up period (SFP) up to Week 16. Participants who opted to receive treatment with TAK-079 were then randomized to receive TAK-079, SC injection, QW for 8 weeks in Open-label Extension (OLE) Period of Part A or Part B. Participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded long follow-up period (LFP) up to Week 32.	Drug: Placebo; TAK-079 placebo-matching SC injection.
Experimental: Part A: Double Blind Period: TAK-079 100 mg; Participants received TAK-079 100 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants who opted to receive treatment with TAK-079 were then randomized to receive TAK-079, SC injection, QW for 8 weeks in OLE Period of Part A or Part B. Participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.	Drug: TAK-079; TAK-079 SC injection.
Experimental: Part A: Double Blind Period: TAK-079 300 mg; Participants received TAK-079 300 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants who opted to receive treatment with TAK-079 were then randomized to receive TAK-079, SC injection, QW for 8 weeks in OLE Period of Part A or Part B. Participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.	Drug: TAK-079; TAK-079 SC injection.
Experimental: Part B: Double Blind Period: TAK-079 600 mg; Participants received TAK-079 600 mg, SC injection, QW for 8 weeks. Following treatment participants were followed up for 8 weeks in a double blinded SFP up to Week 16. Participants who opted to receive treatment with TAK-079 were then randomized to receive TAK-079, SC injection, QW for 8 weeks in OLE Period of Part A or Part B. Participants who did not opt to receive treatment with TAK-079 were followed up for another 16 weeks in an unblinded LFP up to Week 32.	Drug: TAK-079; TAK-079 SC injection.
Experimental: Part A: Open-label Extension (OLE) Period: TAK-079 100 mg; Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 100 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.	Drug: TAK-079; TAK-079 SC injection.
Experimental: Part A: OLE Period: TAK-079 300 mg; Participants who received placebo in double-blind Part A and opted to receive treatment with TAK-079 were randomized to receive TAK-079 300 mg, SC injection, QW for 8 weeks in OLE Period of Part A. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.	Drug: TAK-079; TAK-079 SC injection.
Experimental: Part B: OLE Period: TAK-079 600 mg; Participants who received placebo in double-blind Part B and opted to receive treatment with TAK-079 received TAK-079 600 mg, SC injection, QW for 8 weeks in OLE Period of Part B. Following treatment participants were followed up for 8 weeks in a SFP and then for another 16 weeks in a LFP.	Drug: TAK-079; TAK-079 SC injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation	An adverse event (AE) was defined as any untoward medical occurrence in a participant administered a pharmaceutical product; the untoward medical occurrence does not necessarily have a causal relationship with the treatment. SAE means any untoward medical occurrence that at any dose: a) results in death; b) is life-threatening; c) requires inpatient hospitalization or prolongation of an existing hospitalization; d) results in persistent or significant disability or incapacity; e) is a congenital anomaly/birth defect; f) is a medically important event. TEAEs were defined as an AE having a start date and time equal to or later than the start date and time of the first dose of investigational medicinal product (IMP). Percentages were rounded off to the nearest single decimal place.	Up to Week 32 in each Period of the study

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants With Platelet Response at Weeks 16 and 32	Platelet response is defined as a platelet count ≥50,000/microliter (μL) and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.	At Weeks 16 and 32
Percentage of Participants With Complete Platelet Response at Weeks 16 and 32	Complete platelet response is defined as a platelet count ≥100,000/μL on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.	At Weeks 16 and 32
Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32	A clinically meaningful platelet response is defined as a platelet count ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.	At Weeks 16 and 32
Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32	A hemostatic platelet response is defined for participants with a baseline platelet count of <15,000/μL who achieved a platelet count of ≥30,000/μL and ≥20,000/μL above baseline on at least 2 visits without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy. Percentages were rounded off to the nearest single decimal place.	At Weeks 16 and 32

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Medical Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information about this study, click this link.

Study record dates

Study Major Dates

• Study Start (Actual): November 9, 2020
• Primary Completion (Actual): April 29, 2024
• Study Completion (Actual): April 29, 2024

Study Registration Dates

• First Submitted: February 19, 2020
• First Submitted That Met QC Criteria: February 19, 2020
• First Posted (Actual): February 20, 2020

Study Record Updates

• Last Update Posted (Actual): June 15, 2025
• Last Update Submitted That Met QC Criteria: May 28, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: TAK-079-1004; 2019-004103-12 (EudraCT Number); jRCT2031220408 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No