A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03773393|
Recruitment Status : Not yet recruiting
First Posted : December 12, 2018
Last Update Posted : December 13, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Bone Marrow Disease||Biological: CK0801||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||18 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||
Open-label, Phase I study designed to evaluate the safety of CK0801 in the treatment of bone marrow failure syndromes.
This study will use a Simon 3 + 3 design.
Three patients will be treated at dose level 1: 1x106/kg. If no dose limiting toxicity (DLT) is observed, then the dose will be escalated to dose level 2: 3x106/kg for the next cohort of 3 patients. If no DLT is observed, then the dose will be escalated to dose level 3: 1x107/kg.
If 1 DLT is observed at a dose level, then 3 additional patients will be treated at that level. If no additional DLTs, then that dose level will be defined as MTD.
If ≥ 2 DLTs at dose level 2 or 3, then prior dose level is defined as MTD. If ≥ 2 DLTs at dose level 1, then DSMB will review and evaluate for study continuation. MTD decided when 6 patients are treated at a dose level with < 2 DLTs.
|Masking:||None (Open Label)|
|Official Title:||Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome|
|Estimated Study Start Date :||January 2019|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2020|
Experimental: CK0801, 50ml
All subjects will receive adoptive therapy with an infusion of unrelated cord blood-derived regulatory T cells: CK0801. Subjects will receive one 50mL intravenous dose of CK0801 (Treg cells) on study Day 0. A total of three cohorts will be evaluated.
Cohort dosing will be as follows:
Dose level 1 = 1x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 2 = 3x10e6/kg Treg cells per kg recipient ideal body weight (IBW); Dose level 3 = 1x10e7/kg Treg cells per kg recipient ideal body weight (IBW).
CK0801 (a Cord blood-derived T-regulatory cell product)
- Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 [ Time Frame: 24 hours post-intervention ]Number of Participants with Severe (Grade 3 or 4) Toxicity
- Number of Participants with Regimen Related Death [ Time Frame: 30 days post-intervention ]
- Number of Participants with Severe Cytokine Release Syndrome (CRS) [ Time Frame: 30 days post-intervention ]Number of Participants with Severe (Grade 3 or 4) CRS
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03773393
|Contact: Erin Horneemail@example.com|
|Contact: Tara Sadeghifirstname.lastname@example.org|
|United States, Texas|
|The University of Texas MD Anderson Cancer Center||Not yet recruiting|
|Houston, Texas, United States, 77030|
|Contact: Tapan M Kadia, MD 713-563-3534 email@example.com|
|Principal Investigator: Tapan M Kadia, MD|
|Principal Investigator:||Tapan M Kadia, MD||The University of Texas MD Anderson Cancer Center|