A Study to Evaluate Safety, Tolerability and Efficacy of TAK-079 in Patients with Immune Thrombocytopenia
- Conditions
- Primary immune thrombocytopeniaMedDRA version: 23.0Level: LLTClassification code 10050245Term: Autoimmune thrombocytopeniaSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2019-004103-12-BG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 54
Each patient must meet all the following inclusion criteria to be to be randomized to treatment:
1. Age 18 years or older and able and willing to comply with study procedures.
2. 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.
3. 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
4.Diagnosis of ITP supported by a prior response to an ITP therapy (other than a TPO-RA)
that achieved a platelet count of =50,000/µL.
5. 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. High-dose pulse steroid therapy is not
allowed within 14 days before Day 1.
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.
6. Female patients of childbearing potential are required to have a negative pregnancy test. Both female patients of childbearing potential and male patients must practice an effective, reliable,
and approved contraceptive regimen during the study and for up to 90 days or 5 half-lives, whichever is longer, after discontinuation of treatment.
7. Voluntary written consent must be given before performance of any study-related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future medical care.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 36
Patients meeting any of the following exclusion criteria are not to be randomized to treatment:
1. Use of anticoagulants or any drug with antiplatelet effect (such as aspirin) within 3 weeks before screening.
2. History of any thrombotic or embolic event within 12 months before screening.
3. 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 (eg, 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.
Note: FEV1 testing is required for patients suspected of having COPD or asthma.
6. Use of rituximab or any mAb for immunomodulation within 4 months before first dosing.
Note: Patients 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. Diagnosed with myelodysplastic syndrome.
9. Has received a live vaccine within 4 weeks before screening or has any live vaccines planned during the study.
10. Currently experiencing any medical condition that, in the opinion of the investigator, might interfere with the patient’s participation in the study (such as significant ocular, cardiovascular, pulmonary, hematologic, gastrointestinal, endocrinologic, hepatic, renal, neurologic, malignancy, or infectious disease), that poses an added risk for the patient, or could confound the assessment of the patient.
11. Pregnancy or lactation during screening period or on Day 1 before first dose of study drug.
12. Participation in any other investigational drug study (including vaccine study) or exposure to other investigational agent within 4 weeks or 5 half-lives, whichever is longer, before Day 1.
13. 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. A mild, localized herpes simplex infection within 12 weeks before study treatment is initiated is allowed, as long as the lesion has resolved without systemic therapy before Day 1.
14. Exhibits clinically significant laboratory abnormalities at screening:
a) Alanine aminotransferase or aspartate aminotransferase >3 times the upper limit of normal (ULN).
b) Total bilirubin >1.5 × ULN (Note: Patients with a confirmed diagnosis of Gilbert syndrome that is documented in the patient’s medical record are not excluded based on this criterion).
c) Absolute neutrophil count <1500/mm3.
d) Hemoglobin <8 g/dL.
e) Immunoglobulin (Ig) G <500 mg/dL.
f) Lymphocyte count <500/mm3.
g) Creatinine clearance, CrCl, <30 mL/min (ie, CrCl =30 mL/min is allowed).
15. Has a positive T-cell interferon-? release assay (TIGRA) (results through QuantiFERON TB Gold test or T-Spot/Elispot) at the screening visit, noting the following:
a) A purified protein derivative (PPD) skin test may be used as a replacement, if TIGRA testing is not available.
b) Patients with an indeterminate TIGRA result must mee
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective is to evaluate the safety and tolerability of TAK-079 in patients with persistent/chronic primary ITP.;Secondary Objective: The secondary objective is to assess the effects of TAK-079 administration on platelet counts in patients with persistent/chronic primary ITP.;Primary end point(s): The primary endpoint is the percentage of patients with TEAEs including Grade 3 or higher events, SAEs, and AEs leading to TAK-079 discontinuation.;Timepoint(s) of evaluation of this end point: TEAEs that occur after administration of the first dose of TAK-079 and through to the end of the SFP period will be tabulated and followed until resolution.
- Secondary Outcome Measures
Name Time Method