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

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

Sponsors

Lead Sponsor: Millennium Pharmaceuticals, Inc.

Source Takeda
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).

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.

Overall Status Recruiting
Start Date October 30, 2020
Completion Date May 31, 2023
Primary Completion Date September 1, 2022
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
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 16
Secondary Outcome
Measure Time Frame
Percentage of Participants with Platelet Response Up to Week 16
Percentage of Participants with Complete Platelet Response Up to Week 16
Percentage of Participants with Clinically Meaningful Platelet Response Up to Week 16
Percentage of Participants with Hemostatic Platelet Response Up to Week 16
Enrollment 54
Condition
Intervention

Intervention Type: Drug

Intervention Name: Placebo

Description: TAK-079 placebo-matching injection.

Intervention Type: Drug

Intervention Name: TAK-079

Description: TAK-079 SC injection

Eligibility

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.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Medical Director Study Director Millennium Pharmaceuticals, Inc.
Overall Contact

Last Name: Takeda Study Registration Call Center

Phone: +1-866-835-2233

Email: [email protected]

Location
Facility: Status: Bleeding and Clotting Disorders Institute
Location Countries

United States

Verification Date

August 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 8
Arm Group

Label: Part A: Double Blind, Placebo

Type: Placebo Comparator

Description: TAK-079 placebo-matching injection subcutaneously (SC) once weekly (QW) for 8 weeks.

Label: Part A: Double Blind, TAK-079 Dose 1

Type: Experimental

Description: TAK-079 Dose 1, SC injection QW for 8 weeks.

Label: Part A: Double Blind, TAK-079 Dose 2

Type: Experimental

Description: TAK-079 Dose 2, SC injection QW for 8 weeks.

Label: Part A: Open-label Extension (OLE) Phase, TAK-079 Dose 1

Type: Experimental

Description: 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.

Label: Part A: OLE Phase, TAK-079 Dose 2

Type: Experimental

Description: 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.

Label: Part B: Double Blind, Placebo

Type: Placebo Comparator

Description: TAK-079 placebo-matching injection SC, QW for 8 weeks.

Label: Part B: Double Blind, TAK-079 Dose 3

Type: Experimental

Description: TAK-079 Dose 3, SC injection QW for 8 weeks.

Label: Part B: OLE Phase, TAK-079 Dose 3

Type: Experimental

Description: 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.

Patient Data Yes
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov