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

NCT04278924 | Completed Phase 2 Results FDA Drug

• 3 other identifiers: TAK-079-10042019-004103-12jRCT2031220408
• Study Type: interventional
• Target: 41
• Locations: 11 countries
• Sites: 55

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.

Conditions

Primary Immune Thrombocytopenia

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Percentage of Participants With Platelet Response at Weeks 16 and 32

  At Weeks 16 and 32

• Percentage of Participants With Complete Platelet Response at Weeks 16 and 32

  At Weeks 16 and 32

• Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32

  At Weeks 16 and 32

• Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32

  At Weeks 16 and 32

Study Arms (7)

Part A & B: Double Blind Period: Placebo

PLACEBO COMPARATOR

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

Part A: Double Blind Period: TAK-079 100 mg

EXPERIMENTAL

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

Part A: Double Blind Period: TAK-079 300 mg

EXPERIMENTAL

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

Part B: Double Blind Period: TAK-079 600 mg

EXPERIMENTAL

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

Part A: Open-label Extension (OLE) Period: TAK-079 100 mg

EXPERIMENTAL

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

Part A: OLE Period: TAK-079 300 mg

EXPERIMENTAL

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

Part B: OLE Period: TAK-079 600 mg

EXPERIMENTAL

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

Interventions

Placebo DRUG

TAK-079 placebo-matching SC injection.

Part A & B: Double Blind Period: Placebo

TAK-079 DRUG

TAK-079 SC injection.

Part A: Double Blind Period: TAK-079 100 mg; Part A: Double Blind Period: TAK-079 300 mg; Part A: OLE Period: TAK-079 300 mg; Part A: Open-label Extension (OLE) Period: TAK-079 100 mg; Part B: Double Blind Period: TAK-079 600 mg; Part B: OLE Period: TAK-079 600 mg

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within 3 weeks before screening.
• Has a history of any thrombotic or embolic event within 12 months before screening.
• Has a history of splenectomy within 3 months before screening.
• 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.
• Diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 second (FEV1) \<50% of predicted normal.
• 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.
• Use of immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing.
• Has been diagnosed with myelodysplastic syndrome.
• Has received a live vaccine within 4 weeks before screening or has any live vaccine planned during the study.
• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (55)

Arizona Clinical Research Center - Hunt - PPDS

Tucson, Arizona, 85715, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Bleeding and Clotting Disorders Institute

Peoria, Illinois, 61614-3542, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118-2905, United States

Location

Leo W. Jenkins Cancer Center

Greenville, North Carolina, 27834, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Acibadem City Clinic Multiprofile Hospital for Active Treatment Tokuda

Sofia, Sofia-Grad, 1407, Bulgaria

Location

University Multiprofile Hospital for Active Treatment Sofiamed OOD

Sofia, Sofia-Grad, 1750, Bulgaria

Location

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

Pleven, 5800, Bulgaria

Location

University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD

Plovdiv, 4002, Bulgaria

Location

University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD

Sofia, 1431, Bulgaria

Location

Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia

Sofia, 1606, Bulgaria

Location

Union Hospital Tongji Medical College Huazhong University of Science and Technology

Wuhan, Hubei, 430022, China

Location

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

Tianjin, Tianjin Municipality, 300020, China

Location

Clinical Hospital Centre Osijek

Osijek, 31000, Croatia

Location

Klinicki bolnicki centar Zagreb

Zagreb, 10000, Croatia

Location

University Hospital Merkur

Zagreb, 10000, Croatia

Location

Universitatsklinikum Frankfurt

Frankfurt am Main, Hesse, 60590, Germany

Location

Onkologische Schwerpunktpraxis Kurfurstendamm

Berlin, 10707, Germany

Location

OnkoNet Marburg GmbH

Marburg, 35037, Germany

Location

Rotkreuzklinikum Munchen

München, 80634, Germany

Location

University General Hospital of Patras

Pátrai, Achaia, 26500, Greece

Location

General Hospital of Athens - George Gennimatas

Athens, Attica, 115 27, Greece

Location

Georgios Papanikolaou General Hospital of Thessaloniki

Thessaloniki, 57010, Greece

Location

Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI)

Trieste, Friuli Venezia Giulia, 34149, Italy

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico

Milan, Lombardy, 20122, Italy

Location

Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi

Catania, Sicily, 95122, Italy

Location

Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliera Universitaria Federico II

Napoli, 80131, Italy

Location

A.O.U. Maggiore della Carita

Novara, 20100, Italy

Location

Azienda Policlinico Umberto I

Roma, 161, Italy

Location

Saiseikai Central Hospital

Minato-Ku, Tokyo, 108-0073, Japan

Location

Nihon University Itabashi Hospital

tabashi City, Tokyo, 173-8610, Japan

Location

Univerzitetni klinicni Center Ljubljana

Ljubljana, 1000, Slovenia

Location

University Clinical Centre Maribor

Maribor, 2000, Slovenia

Location

Corporacio Sanitaria Parc Tauli

Sabadell, Barcelona, 8208, Spain

Location

Hospital Universitario Principe de Asturias

Meco, Madrid, 28880, Spain

Location

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33006, Spain

Location

Hospital del Mar

Barcelona, 08003, Spain

Location

Hospital de La Santa Creu i Sant Pau

Barcelona, 8041, Spain

Location

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

Burgos, 9005, Spain

Location

Hospital Universitario Quironsalud Madrid

Madrid, 28006, Spain

Location

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28026, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario Virgen del Rocio - PPDS

Málaga, 29010, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

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

Mykolaiv, Mykolaivs'ka Oblast, 54058, Ukraine

Location

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

Ternopil, Ternopil Oblast, 46002, Ukraine

Location

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

Zhytomyr, Zhytomyr Oblast, 10002, Ukraine

Location

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

Dnipro, 49102, Ukraine

Location

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

Kyiv, 2091, Ukraine

Location

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

Kyiv, 4060, Ukraine

Location

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

Lviv, 79044, Ukraine

Location

Related Links

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

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Takeda

TrialDisclosures@takeda.com

+1-877-825-3327

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2020
• First Posted: February 20, 2020
• Study Start: November 9, 2020
• Primary Completion: April 29, 2024
• Study Completion: April 29, 2024
• Last Updated: June 15, 2025
• Results First Posted: June 15, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

BU (6); UA (7); CN (2); CR (3); JP (2); US (7); SL (2); ES (12); DE (4); GR (3); IT (7)