A Study of TAK-079 in Adults With Persistent/Chronic Primary Immune Thrombocytopenia
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Drug: TAK-079Drug: Placebo
- Registration Number
- NCT04278924
- Lead Sponsor
- Takeda
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 41
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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.
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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.
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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.
- 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.
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Part A: Double Blind, TAK-079 Dose 2 TAK-079 TAK-079 Dose 2, SC injection QW for 8 weeks. Part A: Open-label Extension (OLE) Phase, TAK-079 Dose 1 TAK-079 Participants who received placebo in double-blind Part A and opt to receive further treatment will be randomized to receive TAK-079 Dose 1, SC injection QW for 8 weeks in OLE Phase of Part A. Part A: OLE Phase, TAK-079 Dose 2 TAK-079 Participants who received placebo in double-blind Part A and opt to receive further treatment will be randomized to receive TAK-079 Dose 2, SC injection QW for 8 weeks in OLE Phase of Part A. Part B: Double Blind, Placebo Placebo TAK-079 placebo-matching injection SC, QW for 8 weeks. Part B: Double Blind, TAK-079 Dose 3 TAK-079 TAK-079 Dose 3, SC injection QW for 8 weeks. Part B: OLE Phase, TAK-079 Dose 3 TAK-079 Participants who received placebo in double-blind Part B and opt to receive further treatment will receive TAK-079 Dose 3, SC injection QW for 8 weeks in OLE Phase of Part B. Part A: Double Blind, Placebo Placebo TAK-079 placebo-matching injection subcutaneously (SC) once weekly (QW) for 8 weeks. Part A: Double Blind, TAK-079 Dose 1 TAK-079 TAK-079 Dose 1, SC injection QW for 8 weeks.
- Primary Outcome Measures
Name Time Method Percentage of Participants with at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event (AE) Leading to TAK-079 Discontinuation From the first dose of study drug up to Week 32
- Secondary Outcome Measures
Name Time Method Percentage of Participants with Platelet Response Up to Week 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.
Percentage of Participants with Complete Platelet Response Up to Week 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.
Percentage of Participants with Clinically Meaningful Platelet Response Up to Week 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.
Percentage of Participants with Hemostatic Platelet Response Up to Week 32 A hemostatic platelet response is defined for participants with a baseline platelet count of \<15,000/μL who achieve 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.
Trial Locations
- Locations (55)
Acibadem City Clinic Multiprofile Hospital for Active Treatment Tokuda
🇧🇬Sofia, Sofia-Grad, Bulgaria
Univerzitetni klinicni Center Ljubljana
🇸🇮Ljubljana, Slovenia
University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
🇧🇬Plovdiv, Bulgaria
General Hospital of Athens - George Gennimatas
🇬🇷Athens, Attiki, Greece
University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD
🇧🇬Sofia, Bulgaria
Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia
🇧🇬Sofia, Bulgaria
University General Hospital of Patras
🇬🇷Patra, Achaia, Greece
Saiseikai Central Hospital
🇯🇵Minato-Ku, Tokyo, Japan
University of Florida
🇺🇸Gainesville, Florida, United States
University Multiprofile Hospital for Active Treatment - Dr. Georgi Stranski EAD
🇧🇬Pleven, Bulgaria
Hospital Universitario Central de Asturias
🇪🇸Oviedo, Asturias, Spain
University of Virginia
🇺🇸Charlottesville, Virginia, United States
Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi
🇮🇹Catania, Sicilia, Italy
Hospital Universitario Virgen del Rocio - PPDS
🇪🇸Malaga, Spain
C.A.U de Burgos - Hospital Universitario de Burgos
🇪🇸Burgos, Spain
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
Arizona Clinical Research Center - Hunt - PPDS
🇺🇸Tucson, Arizona, United States
Bleeding and Clotting Disorders Institute
🇺🇸Peoria, Illinois, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University Multiprofile Hospital for Active Treatment Sofiamed OOD
🇧🇬Sofia, Sofia-Grad, Bulgaria
Leo W. Jenkins Cancer Center
🇺🇸Greenville, North Carolina, United States
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, China
Klinicki bolnicki centar Zagreb
🇭🇷Zagreb, Croatia
Onkologische Schwerpunktpraxis Kurfurstendamm
🇩🇪Berlin, Germany
University Hospital Merkur
🇭🇷Zagreb, Croatia
OnkoNet Marburg GmbH
🇩🇪Marburg, Germany
Universitatsklinikum Frankfurt
🇩🇪Frankfurt am Main, Hessen, Germany
Rotkreuzklinikum Munchen
🇩🇪Munchen, Germany
Georgios Papanikolaou General Hospital of Thessaloniki
🇬🇷Thessaloniki, Greece
Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI)
🇮🇹Trieste, Friuli-Venezia Giulia, Italy
Azienda Policlinico Umberto I
🇮🇹Roma, Italy
A.O.U. Maggiore della Carita
🇮🇹Novara, Italy
Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi
🇮🇹Bologna, Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
🇮🇹Milano, Lombardia, Italy
Azienda Ospedaliera Universitaria Federico II
🇮🇹Napoli, Italy
University Clinical Centre Maribor
🇸🇮Maribor, Slovenia
Nihon University Itabashi Hospital
🇯🇵Itabashi, Tokyo, Japan
Corporacio Sanitaria Parc Tauli
🇪🇸Sabadell, Barcelona, Spain
Hospital del Mar
🇪🇸Barcelona, Spain
Hospital de La Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitario Principe de Asturias
🇪🇸Meco, Madrid, 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
MNE Regional Clinical Hospital n a O F Herbachevskyi of Zhytomyr Regional Council
🇺🇦Zhytomyr, Zhytomyrs'ka Oblast, Ukraine
Municipal Non-profit Enterprise Mykolayiv Regional Clinical Hospital the Mykolayiv Regional Council
🇺🇦Mykolaiv, Mykolaivs'ka Oblast, Ukraine
Hospital Universitari i Politecnic La Fe de Valencia
🇪🇸Valencia, Spain
Municipal Non-profit Enterprise Ternopil Regional Clinical Hospital of Ternopil Regional Council
🇺🇦Ternopil, Ternopil's'ka 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
Clinical Hospital Centre Osijek
🇭🇷Osijek, Croatia