Phase 2
N=41
A Study of TAK-079 in Adults With Persistent/Chronic Primary Immune Thrombocytopenia
Primary Immune Thrombocytopenia
Bottom Line
View on ClinicalTrials.gov: NCT04278924 ↗Enrolled (actual)
41
Serious AEs
11.3%
Results posted
Jun 2025
Primary outcome: Primary: Percentage of Participants With at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation — 23.1; 22.2; 0; 27.3 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Placebo (Drug); TAK-079 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Takeda
- Primary completion
- Apr 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With at Least One Grade 3 or Higher Treatment Emergent Adverse Event (TEAE), Treatment Emergent Serious Adverse Event (SAE), and TEAEs Leading to TAK-079 Discontinuation |
23.1; 22.2; 0; 27.3; 0; 0 | — |
| SECONDARY Percentage of Participants With Platelet Response at Weeks 16 and 32 |
23.08; 66.67; 62.50; 90.91; 50.00; 50.00 | =0.056 |
| SECONDARY Percentage of Participants With Complete Platelet Response at Weeks 16 and 32 |
0; 55.56; 50.00; 81.82; 0; 25.00 | =0.001 sig |
| SECONDARY Percentage of Participants With Clinically Meaningful Platelet Response at Weeks 16 and 32 |
30.77; 66.67; 75.00; 90.91; 75.00; 50.00 | =0.120 |
| SECONDARY Percentage of Participants With Hemostatic Platelet Response at Weeks 16 and 32 |
0; 40.00; 25.00; 100.00; 100.00; 100.00 | =0.187 |
Summary
Primary immune thrombocytopenia (ITP) is a rare disease that results in low levels of platelets - the cells that help blood clot.
The main aim of the study is to check for side effects from taking TAK-079 at three different dose levels. Another aim is to learn if TAK-079 can increase the platelet count in people with ITP.
In addition to receiving stable background therapy for ITP, participants will receive an injection of either TAK-079 or a placebo once a week for 2 months. A placebo looks like TAK-079 but will not have any medicine in it. After treatment, all participants will be followed-up for another 2 months.
Then, participants who received TAK-079 will continue to be followed-up for an extra 4 months. Participants who received the placebo and would like to receive TAK-079 may be able to do this in an extension period in the study.
Eligibility Criteria
Inclusion Criteria
- 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 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.
- 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.
- If receiving standard background treatment for ITP, treatment should be stable in dose and frequency for at least 4 weeks before dosing.
- 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.
- 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
- Use of 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.
- 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.
- Diagnosed with chronic obstructive pulmonary disease (COPD) or asthma, and a prebronchodilatory forced expiratory volume in 1 second (FEV1) <50% of predicted normal.
- 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.
- Use of immunosuppressants (such as cyclophosphamide, vincristine) other than permitted oral immunosuppressants within 6 months before first dosing.
- Has been diagnosed with myelodysplastic syndrome.
- 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.
Data sourced from ClinicalTrials.gov (NCT04278924). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.