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

Primary Immune Thrombocytopenia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04278924
• Other study ID #: TAK-079-1004
• Secondary ID: 2019-004103-12jR
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: November 9, 2020
• Est. completion date: August 1, 2024

Study information

• Verified date: January 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Active, not recruiting
• Enrollment: 41
• Est. completion date: August 1, 2024
• Est. primary completion date: August 1, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Placebo
TAK-079 placebo-matching SC injection.
• TAK-079
TAK-079 SC injection.

Locations

Country	Name	City	State
Bulgaria	University Multiprofile Hospital for Active Treatment - Dr. Georgi Stranski EAD	Pleven
Bulgaria	University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD	Plovdiv
Bulgaria	Acibadem City Clinic Multiprofile Hospital for Active Treatment Tokuda	Sofia	Sofia-Grad
Bulgaria	Military Medical Academy Multiprofile Hospital for Active Treatment - Sofia	Sofia
Bulgaria	University Multiprofile Hospital for Active Treatment Sofiamed OOD	Sofia	Sofia-Grad
Bulgaria	University Multiprofile Hospital for Active Treatment Sv Ivan Rilski EAD	Sofia
China	Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences	Tianjin	Tianjin
China	Union Hospital Tongji Medical College Huazhong University of Science and Technology	Wuhan	Hubei
Croatia	Clinical Hospital Centre Osijek	Osijek
Croatia	Klinicki bolnicki centar Zagreb	Zagreb
Croatia	University Hospital Merkur	Zagreb
Germany	Onkologische Schwerpunktpraxis Kurfurstendamm	Berlin
Germany	Universitatsklinikum Frankfurt	Frankfurt am Main	Hessen
Germany	OnkoNet Marburg GmbH	Marburg
Germany	Rotkreuzklinikum Munchen	Munchen
Greece	General Hospital of Athens - George Gennimatas	Athens	Attiki
Greece	University General Hospital of Patras	Patra	Achaia
Greece	Georgios Papanikolaou General Hospital of Thessaloniki	Thessaloniki
Italy	Azienda Ospedaliero Universitaria Di Bologna - Policlinico S Orsola Malpighi	Bologna
Italy	Azienda Ospedaliera Di Rilievo Nazionale E Di Alta Specializzazione Garibaldi	Catania	Sicilia
Italy	Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milano	Lombardia
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli
Italy	A.O.U. Maggiore della Carita	Novara
Italy	Azienda Policlinico Umberto I	Roma
Italy	Azienda Sanitaria Universitaria Giuliano Isontina (ASUGI)	Trieste	Friuli-Venezia Giulia
Japan	Nihon University Itabashi Hospital	Itabashi	Tokyo
Japan	Saiseikai Central Hospital	Minato-Ku	Tokyo
Slovenia	Univerzitetni klinicni Center Ljubljana	Ljubljana
Slovenia	University Clinical Centre Maribor	Maribor
Spain	Hospital de La Santa Creu i Sant Pau	Barcelona
Spain	Hospital del Mar	Barcelona
Spain	C.A.U de Burgos - Hospital Universitario de Burgos	Burgos
Spain	Hospital General Universitario Gregorio Maranon	Madrid
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario Quironsalud Madrid	Madrid
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital Universitario Virgen del Rocio - PPDS	Malaga
Spain	Hospital Universitario Principe de Asturias	Meco	Madrid
Spain	Hospital Universitario Central de Asturias	Oviedo	Asturias
Spain	Corporacio Sanitaria Parc Tauli	Sabadell	Barcelona
Spain	Hospital Universitari i Politecnic La Fe de Valencia	Valencia
Ukraine	Municipal Non-profit Enterprise "City Clinical Hospital # 4" of Dnipro City Council - PPDS	Dnipro
Ukraine	CNE Kyiv City Clinical Hospital #9 of Exec. Body of Kyiv City Council Kyiv City State Admin	Kyiv
Ukraine	Medical Center OK!Clinic+LLC International Institute of Clinical Research	Kyiv
Ukraine	State Institution Institute of Blood Pathology and Transfusion Medicine of NAMS of Ukraine	Lviv
Ukraine	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's'ka Oblast
Ukraine	MNE Regional Clinical Hospital n a O F Herbachevskyi of Zhytomyr Regional Council	Zhytomyr	Zhytomyrs'ka Oblast
United States	Boston Medical Center	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Virginia	Charlottesville	Virginia
United States	University of Florida	Gainesville	Florida
United States	Leo W. Jenkins Cancer Center	Greenville	North Carolina
United States	Bleeding and Clotting Disorders Institute	Peoria	Illinois
United States	Arizona Clinical Research Center - Hunt - PPDS	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Percentage of Participants with Platelet Response	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.	Up to Week 32
Secondary	Percentage of Participants with Complete Platelet Response	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.	Up to Week 32
Secondary	Percentage of Participants with Clinically Meaningful Platelet Response	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.	Up to Week 32
Secondary	Percentage of Participants with Hemostatic Platelet Response	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.	Up to Week 32

External Links

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