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

JRCT ID: jRCT2031220408

Registered date:25/10/2022

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Primary Immune Thrombocytopenia
• Date of first enrollment: 09/11/2020
• Target sample size: 41
• Countries of recruitment: USA,Japan,Bulgaria,Japan,Croatia,Japan,Germany,Japan,Italy,Japan,Slovenia,Japan,Ukraine,Japan,Spain,,Japan,Greece,Japan,China,Japan
• Study type: Interventional

Intervention(s)

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

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

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

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

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

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.

Outcome(s)

Primary Outcome

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 32

Secondary Outcome

1.Percentage of Participants with Platelet Response
Time Frame: Up to Week 32
Platelet response is defined as a platelet count >=50,000/microliter (mcrL) and >=20,000/mcrL 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 32
Complete platelet response is defined as a platelet count >=100,000/mcrL 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 32
A clinically meaningful platelet response is defined as a platelet count >=20,000/mcrL 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 32
A hemostatic platelet response is defined for participants with a baseline platelet count of <15,000/mcrL who achieve a platelet count of >=30,000/mcrL and >=20,000/mcrL 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.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include 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/mcrL (and individually =< 35,000/mcrL) 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/mcrL.
4.If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least 4 weeks before dosing.
a.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.
b.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.

Exclude 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 Information

• Primary Sponsor: Nishizawa Atsushi
• Secondary ID(s): NCT04278924,2019-004103-12