A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)

Summary

The goal of this clinical research study is to determine whether it is safe and practical to give CK0801 (a Cord blood-derived T-regulatory cell product) to patients with bone marrow failure syndrome. Researchers want to determine the highest possible dose that is safe to be given. Researchers also want to learn if CK0801 may improve the symptoms of bone marrow failure syndrome. Patients enrolled in this study will all have been diagnosed with treatment refractory bone marrow failure syndrome (which includes aplastic anemia, myelodysplastic syndrome, or myelofibrosis). Participants eligible to participate in this study are unable or unwilling to be treated with standard therapy or have failed standard therapy.

Timeline

Start

2019-05-30

Primary completion

2027-05-25

Completion

2027-05-30

Publications

• Kadia TM, Huang M, Pemmaraju N, Abbas HA, Ly C, Masarova L, Yilmaz M, Lyu MA, Zeng K, Sadeghi T, Cook R, DiNardo CD, Daver N, Issa GC, Jabbour E, Borthakur G, Jain N, Garcia-Manero G, Parmar S, Flowers C, Kantarjian H, Verstovsek S. Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes. NEJM Evid. 2024 Jun;3(6):EVIDoa2300362. doi: 10.1056/EVIDoa2300362. Epub 2024 May 28.

Drugs

Indications