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

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ClinicalTrials.gov Identifier: NCT04278924
Recruitment Status : Recruiting
First Posted : February 20, 2020
Last Update Posted : December 14, 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. 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 study drug after a safety follow-up period and will be randomized to one of the two open-label TAK-079 treatment arms.

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 after the 8 weeks of treatment.

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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
Actual Study Start Date : November 9, 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 SC 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 opt to receive further treatment who have been 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 opt to receive further treatment who have been 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 SC 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 opt to receive further treatment will 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 ]

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 without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous 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 without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous 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 without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous 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 without a dosing period-permitted rescue treatment in the previous 4 weeks and without any other previous rescue therapy.



Information from the National Library of Medicine

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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:

  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 patient'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 minute (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 CD19 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.


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 +1-877-825-3327 medinfoUS@takeda.com

Locations
Show Show 31 study locations
Sponsors and Collaborators
Millennium Pharmaceuticals, Inc.
Investigators
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Study Director: Medical Director Clinical Science 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: December 14, 2020
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (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.
URL: https://vivli.org/ourmember/takeda/

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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