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Exploratory clinical study on the tolerability, safety and efficacy of iPS cell-derived platelets (MEG-002) in patients with thrombocytopenia

MEG-002: Exploratory clinical study in thrombocytopenic patients

1

Thrombocytopenia due to aplastic anemia.

Number of subject enrolled: 1 Number of subject completed the study: 1 (This trial was discontinued due to administrative reasons after one subject completed the study.)

No adverse event was reported.

In the subject infused with 3 units of MEG-002, no adverse event was reported and no clinically relevant abnormalities were observed in vital signs, laboratory tests etc. Corrected count increment (CCI) and count increment (CI) of platelets increased transiently after infusion of MEG-002. No bleeding was observed througout the study period.

The results of infusing 3 units of MEG-002 to one subject showed no safety issue, and increase in the platelet counts in the blood.

Jan. 10, 2024

No

https://jrct.niph.go.jp/latest-detail/jRCT2053210068

Takao Kazumasa

Megakaryon Corporation

Kyoto Research Park, 134 Chudoji Minamimachi, Shimogyo-ku, Kyoto

megakaryon_clinicalstudy_office@megakaryon.com

Takao Kazumasa

Megakaryon Corporation

Kyoto Research Park, 134 Chudoji Minamimachi, Shimogyo-ku, Kyoto

+81-75-754-7426

megakaryon_clinicalstudy_office@megakaryon.com

Complete

Aug. 19, 2021

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1) Thrombocytopenic patients under stable medical conditions who have been diagnosed with any of the following diseases ,
- Aplastic anemia
- Myelodysplastic syndrome
- Leukemia (excluding induction therapy, early cytopenia after consolidation therapy and acute promyelocytic leukemia)
- Solid cancer after chemotherapy (prolonged thrombocytopenia)
2) Patients whose platelet count is 20,000 / microL or less, or expected to be 20,000 / microL or less without blood transfusion, or patients who are at a risk of bleeding at 30,000 / microL or less
3) Patients who can wash out for 3 days or more after blood donation platelet transfusion before administration of MEG-002
4) Aged 18 years or older at the time of the informed consent
5) Gender: Not specified
6) Patients with written informed consent (and a surrogate in case of under 20)

1) Patients with platelet transfusion refractoriness
2) Patients with a history of severe blood transfusion allergies such as anaphylaxis in the past
3) Patients with hemophilia or other blood coagulation disorders
4) Patients with symptomatic thrombosis or at risk of thrombosis
5) Patients with a history of pulmonary embolism and central hepatic vein occlusion
6) Patients with a history of thrombotic thrombocytopenic purpura, hemolytic uremic syndrome, or immune thrombocytopenic purpura
7) Patients with disseminated intravascular coagulation or sepsis
8) Splenomegaly (maximum ultrasound image diameter of 18 cm or more)
9) Patients requiring anticoagulant / antithrombotic therapy with anticoagulants and antiplatelet agents
10) Patients with moderate or sever renal dysfunction and/or hepatic dysfunction

Both

Thrombocytopenia

Platelet transfusion (Intravenously infused at 3 or 10 units using a blood transfusion set.)

Safety (AE, ADR), Effecacy (CCI)

+81-75-751-4389

none