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

NCT ID: NCT04278924

Last Updated: 2025-06-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 41 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-11-09
• Study Completion Date: 2024-04-29

Brief Summary

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.

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.

Conditions

Primary Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

TAK-079 placebo-matching SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

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.

Group Type: EXPERIMENTAL

TAK-079

Intervention Type: DRUG

TAK-079 SC injection.

Interventions

Placebo

TAK-079 placebo-matching SC injection.

Intervention Type: DRUG

TAK-079

TAK-079 SC injection.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Takeda

Locations

Arizona Clinical Research Center - Hunt - PPDS

Tucson, Arizona, United States

University of Florida

Gainesville, Florida, United States

Bleeding and Clotting Disorders Institute

Peoria, Illinois, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Boston Medical Center

Boston, Massachusetts, United States

Leo W. Jenkins Cancer Center

Greenville, North Carolina, United States

University of Virginia

Charlottesville, Virginia, United States

Acibadem City Clinic Multiprofile Hospital for Active Treatment Tokuda

Sofia, Sofia-Grad, Bulgaria

University Multiprofile Hospital for Active Treatment Sofiamed OOD

Sofia, Sofia-Grad, Bulgaria

University Multiprofile Hospital for Active Treatment - Dr. Georgi Stranski EAD

Pleven, , Bulgaria

University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD

Plovdiv, , Bulgaria

University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD

Sofia, , Bulgaria

Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia

Sofia, , Bulgaria

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, China

Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, China

Clinical Hospital Centre Osijek

Osijek, , Croatia

Klinicki bolnicki centar Zagreb

Zagreb, , Croatia

University Hospital Merkur

Zagreb, , Croatia

Universitatsklinikum Frankfurt

Frankfurt am Main, Hesse, Germany

Onkologische Schwerpunktpraxis Kurfurstendamm

Berlin, , Germany

OnkoNet Marburg GmbH

Marburg, , Germany

Rotkreuzklinikum Munchen

München, , Germany

University General Hospital of Patras

Pátrai, Achaia, Greece

General Hospital of Athens - George Gennimatas

Athens, Attica, Greece

Georgios Papanikolaou General Hospital of Thessaloniki

Thessaloniki, , Greece

Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI)

Trieste, Friuli Venezia Giulia, Italy

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, Lombardy, Italy

Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi

Catania, Sicily, Italy

Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi

Bologna, , Italy

Azienda Ospedaliera Universitaria Federico II

Napoli, , Italy

A.O.U. Maggiore della Carita

Novara, , Italy

Azienda Policlinico Umberto I

Roma, , Italy

Saiseikai Central Hospital

Minato-Ku, Tokyo, Japan

Nihon University Itabashi Hospital

tabashi City, Tokyo, Japan

Univerzitetni klinicni Center Ljubljana

Ljubljana, , Slovenia

University Clinical Centre Maribor

Maribor, , Slovenia

Corporacio Sanitaria Parc Tauli

Sabadell, Barcelona, Spain

Hospital Universitario Principe de Asturias

Meco, Madrid, Spain

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, Spain

Hospital del Mar

Barcelona, , Spain

Hospital de La Santa Creu i Sant Pau

Barcelona, , Spain

C.A.U de Burgos - Hospital Universitario de Burgos

Burgos, , Spain

Hospital Universitario Quironsalud Madrid

Madrid, , Spain

Hospital General Universitario Gregorio Maranon

Madrid, , Spain

Hospital Universitario 12 de Octubre

Madrid, , Spain

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Hospital Universitario Virgen del Rocio - PPDS

Málaga, , Spain

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

Municipal Non-profit Enterprise Mykolayiv Regional Clinical Hospital the Mykolayiv Regional Council

Mykolaiv, Mykolaivs'ka Oblast, Ukraine

Municipal Non-profit Enterprise Ternopil Regional Clinical Hospital of Ternopil Regional Council

Ternopil, Ternopil Oblast, Ukraine

MNE Regional Clinical Hospital n a O F Herbachevskyi of Zhytomyr Regional Council

Zhytomyr, Zhytomyr Oblast, Ukraine

Municipal Non-profit Enterprise "City Clinical Hospital # 4" of Dnipro City Council - PPDS

Dnipro, , Ukraine

Medical Center OK!Clinic+LLC International Institute of Clinical Research

Kyiv, , Ukraine

CNE Kyiv City Clinical Hospital #9 of Exec. Body of Kyiv City Council Kyiv City State Admin

Kyiv, , Ukraine

State Institution Institute of Blood Pathology and Transfusion Medicine of NAMS of Ukraine

Lviv, , Ukraine

Countries

United States; Bulgaria; China; Croatia; Germany; Greece; Italy; Japan; Slovenia; Spain; Ukraine

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://clinicaltrials.takeda.com/study-detail/5f6b603d4db2bf003ab4a391?idFilter=%5B%22TAK-079-1004%22%5D

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

Other Identifiers

2019-004103-12

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

jRCT2031220408

Identifier Type: REGISTRY

Identifier Source: secondary_id

TAK-079-1004

Identifier Type: -

Identifier Source: org_study_id