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Study to Evaluate Safety, Tolerability, and Efficacy of TAK-079 in Participants With Persistent/Chronic Primary Immune Thrombocytopenia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04278924
Recruitment Status : Recruiting
First Posted : February 20, 2020
Last Update Posted : November 4, 2020
Sponsor:
Information provided by (Responsible Party):
Takeda ( Millennium Pharmaceuticals, Inc. )

Brief Summary:
The purpose of this study is to evaluate the safety and tolerability of TAK-079 in participants with persistent/chronic primary immune thrombocytopenia (ITP).

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia Drug: Placebo Drug: TAK-079 Phase 2

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 54 patients. Participants will be randomly assigned (by chance, like flipping a coin) to one of the three treatment groups in the Part A double blind treatment period. Those who received placebo in this period will have choice to receive study drug after a safety follow-up period and upon randomization to either of the two open-label treatment arms.

Upon investigators decision, Part B will randomly assign participants to one of two treatment groups in Part B double blind treatment period. Those who received placebo in this period will have the choice to receive study drug after safety follow-up period in a single open-label treatment arm.

This multi-center trial will be conducted worldwide.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Estimated Study Start Date : November 30, 2020
Estimated Primary Completion Date : September 1, 2022
Estimated Study Completion Date : May 31, 2023


Arm Intervention/treatment
Placebo Comparator: Part A: Double Blind, Placebo
TAK-079 placebo-matching injection subcutaneously (SC) once weekly (QW) for 8 weeks.
Drug: Placebo
TAK-079 placebo-matching injection.

Experimental: Part A: Double Blind, TAK-079 Dose 1
TAK-079 Dose 1, SC injection QW for 8 weeks.
Drug: TAK-079
TAK-079 SC injection

Experimental: Part A: Double Blind, TAK-079 Dose 2
TAK-079 Dose 2, SC injection QW for 8 weeks.
Drug: TAK-079
TAK-079 SC injection

Experimental: Part A: Open-label Extension (OLE) Phase, TAK-079 Dose 1
Participants who received placebo in double-blind Part A and opted 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.
Drug: TAK-079
TAK-079 SC injection

Experimental: Part A: OLE Phase, TAK-079 Dose 2
Participants who received placebo in double-blind Part A and opted 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.
Drug: TAK-079
TAK-079 SC injection

Placebo Comparator: Part B: Double Blind, Placebo
TAK-079 placebo-matching injection SC, QW for 8 weeks.
Drug: Placebo
TAK-079 placebo-matching injection.

Experimental: Part B: Double Blind, TAK-079 Dose 3
TAK-079 Dose 3, SC injection QW for 8 weeks.
Drug: TAK-079
TAK-079 SC injection

Experimental: Part B: OLE Phase, TAK-079 Dose 3
Participants who received placebo in double-blind Part B and opted to receive further treatment will be randomized to receive TAK-079 Dose 3, SC injection QW for 8 weeks in OLE phase of Part B.
Drug: TAK-079
TAK-079 SC injection




Primary Outcome Measures :
  1. 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 [ Time Frame: From the first dose of study drug up to Week 16 ]
    An AE is any untoward medical occurrence in a participant administered a medicinal investigational drug. It does not necessarily have to have a causal relationship with treatment. An SAE is any untoward medical occurrence that results in death; is life-threatening; requires inpatient hospitalization or prolongation of present hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect or is a medically important event that may not be immediately life-threatening or result in death or hospitalization, but may jeopardize the participant or may require intervention to prevent one of other outcomes listed in definition above, or involves suspected transmission via a medicinal product of an infectious agent. TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Severity grade for AE will be determined using National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 4.03.


Secondary Outcome Measures :
  1. Percentage of Participants with Platelet Response [ Time Frame: Up to Week 16 ]
    Platelet response is defined as a platelet count ≥50,000/μL and ≥20,000/μL above baseline on at least 2 visits in the absence of any concomitant rescue therapy.

  2. Percentage of Participants with Complete Platelet Response [ Time Frame: Up to Week 16 ]
    Complete platelet response is defined as a platelet count ≥100,000/μL on at least 2 visits in the absence of any concomitant rescue therapy.

  3. Percentage of Participants with Clinically Meaningful Platelet Response [ Time Frame: Up to Week 16 ]
    A clinically meaningful platelet response is defined as a platelet count ≥20,000/μL above baseline on at least 2 visits in the absence of any concomitant rescue therapy.

  4. Percentage of Participants with Hemostatic Platelet Response [ Time Frame: Up to Week 16 ]
    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 in the absence of any concomitant rescue therapy.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has been 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 mean platelet count of <30,000/μL for the 4 weeks before the first study dose. The mean platelet count is based on at least 2 platelet counts within 4 weeks of dosing, including the value obtained at screening. No individual platelet count >35,000/μL during these times is allowed.
  • Has had 1 prior platelet response to any standard of care treatment to achieve a platelet count of ≥50,000/μL. Any standard treatment in this context may include therapies that are not permitted concomitant therapies during the study.
  • 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 thrombopoietin receptor agonist (TPO-RA) (romiplostim, eltrombopag, avatrombopag); ±fostamatinib. Corticosteroids, including dexamethasone, must be given as oral, daily therapy as opposed to pulse therapy. High-dose pulse steroid therapy is not allowed within 14 days before Day 1.
    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:

  • Uses 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.
  • Uses intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin or anti-D treatment within 4 weeks of screening.
  • Is diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 minute (FEV1) <50% of predicted normal.
  • Uses rituximab or any monoclonal antibody (mAb) for immunomodulation within 4 months before first dosing.
  • Uses immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing.
  • Is diagnosed with myelodysplastic syndrome.
  • Has received vaccinations within 4 weeks before screening or has any vaccinations 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04278924


Contacts
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Contact: Takeda Study Registration Call Center +1-866-835-2233 globaloncologymedinfo@takeda.com

Locations
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United States, Illinois
Bleeding and Clotting Disorders Institute Recruiting
Peoria, Illinois, United States, 61615
Sponsors and Collaborators
Millennium Pharmaceuticals, Inc.
Investigators
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Study Director: Medical Director Millennium Pharmaceuticals, Inc.
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Responsible Party: Millennium Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04278924    
Other Study ID Numbers: TAK-079-1004
2019-004103-12 ( EudraCT Number )
First Posted: February 20, 2020    Key Record Dates
Last Update Posted: November 4, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda makes patient-level, de-identified data sets and associated documents available for all interventional studies after applicable marketing approvals and commercial availability have been received (or program is completely terminated), an opportunity for the primary publication of the research and final report development has been allowed, and other criteria have been met as set forth in Takeda's Data Sharing Policy (see www.TakedaClinicalTrials.com for details). To obtain access, researchers must submit a legitimate academic research proposal for adjudication by an independent review panel, who will review the scientific merit of the research and the requestor's qualifications and conflict of interest that can result in potential bias. Once approved, qualified researchers who sign a data sharing agreement are provided access to these data in a secure research environment.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Takeda ( Millennium Pharmaceuticals, Inc. ):
Drug Therapy
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations