A Study of TAK-226 for Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes
ConditionMyelodysplastic Syndromes
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorTakeda
About this trial
The main aim of the study is to evaluate how TAK-226 improves symptoms of transfusion-dependent anemia in Japanese patients with lower-risk myelodysplastic syndromes.
The study consists of Screening Period (up to 6 weeks), Treatment Period, Safety Follow-Up Period (8 weeks), and Long-Term Follow-Up Period (approximately 5 years from the first dose of the study treatment).
Participants of this study will be administered TAK-226 during Treatment Period. Subsequently, the participants will be monitored for side effects related to the study treatment during Safety Follow-Up Period and Long-Term Follow-Up Period. The approximate duration of participation for a participant is up to approximately 6 years.
During the study period, participants will visit the study clinic/hospital multiple times as per the study schedule. During Treatment Period, the participants will come to the clinic/hospital approximately every two to four weeks.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Participants or their legally authorized representative must be willing and able to sign the ICF and to adhere to the protocol requirements.
Japanese adult male or female participant greater than or equal to (>=) 18 years of age at the time of signing informed consent.
Diagnosis of MDS with or without ring sideroblasts (RS) (as determined in an evaluable bone marrow sample collected at screening to confirm diagnosis) according to WHO 2016 classification that meets the International Prognostic Scoring System-Revised (IPSS-R) classification of very low-, low-, or intermediate-risk MDS.
Low-transfusion burden (LTB), defined as 4 to 7 RBC units per 16 weeks; or
Disqualifiers
Del(5q) MDS or secondary MDS.
Anemia due to any other known cause (eg, thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and/or folate).
Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks in TD cohort or 8 weeks in NTD cohort before enrollment.
New York Heart Association heart disease class III or IV;
Trial design
Design model
Single group
Treatments tested in this trial
TAK-226
DrugTAK-226 subcutaneous injection
Treatment groups
42 Participants
are divided into 1 treatment groupGroup A: TAK-226Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Transfusion dependent (TD) cohort: Percentage of Participants Achieving Transfusion Independence (TI) for Greater Than or Equal to (>=) 8 Weeks from Baseline through Week 24
Transfusion independence is defined as the absence of any red blood cells (RBC) transfusions in a period of at least 8 weeks after the first dose of the study treatment through week 24.
Time frame
Baseline, Up to Week 242
Non-transfusion dependent (NTD) cohort: Percentage of Participants Achieving Mean Hgb Increase of >=1.5 grams per deciliter (g/dL) for >=8 Weeks from Baseline through Week 24 And No RBC Transfusion during the Same >=8 Weeks Period
Time frame
Baseline, Up to Week 24Secondary outcomes
1
TD cohort: Percentage of Participants Achieving TI for >=24 Weeks from Baseline through Week 48
Transfusion independence is defined as the absence of any red blood cells (RBC) transfusions in a period of at least 24 weeks after the first dose of the study treatment through week 48.
Time frame
Baseline, Up to Week 482
TD cohort: Percentage of Participants with High Transfusion Burden (HTB) Achieving TI for >=8 Weeks from Baseline through Week 24
Transfusion independence is defined as the absence of any RBC transfusions in a period of at least 8 weeks after the first dose of the study treatment through week 24.
Time frame
Baseline, Up to Week 243
TD cohort: Percentage of Participants Achieving Mean Hemoglobin (Hgb) Increase of >=1.5 g/dL for >=8 Weeks from Baseline through Week 24
Time frame
Baseline, Up to Week 244
TD cohort: Number of Participants with Treatment-Emergent Adverse Event (TEAEs) and Serious Adverse Event (SAEs)
An AE is any untoward medical occurrence in a clinical trial participant, temporally associated with the use of the trial intervention, whether or not the occurrence is considered related to the trial intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of the trial intervention. A TEAE is defined as an AE that commences on or after the first dose of the study treatment and within 60 days after the last dose of the study treatment, or analysis cutoff date, whichever is earlier. An SAE is any untoward medical occurrence that, at any dose: results in death, is life threatening, requires in-patient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event.
Time frame
Up to approximately 6 years Source ClinicalTrials.gov