Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Primary Objective:
To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at the time of CK00801 infusion
Secondary Objective:
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CK0801 | Experimental | All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one intravenous dose of CK0801 (Treg cells) on study Day 0. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CK0801 | Biological | CK0801 (a Cord blood-derived T-regulatory cell product) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 | Number of Participants with Severe (Grade 3 or 4) Toxicity | 24 hours post-intervention |
| Number of Participants with Regimen Related Death | Number of Participants with Regimen Related Death | 30 days post-intervention |
| Number of Participants with Severe Cytokine Release Syndrome (CRS) | Number of Participants with Severe (Grade 3 or 4) CRS | 30 days post-intervention |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Tapan M Kadia, MD | M.D. Anderson Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sarcoma Oncology Research Center, Cancer Center of Southern California | Santa Monica | California | 90403 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38804782 | Derived | 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. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| ID | Term |
|---|---|
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| The University of Texas MD Anderson Cancer Center |
| Houston |
| Texas |
| 77030 |
| United States |