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

NCT03773393 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CK0801-101-1
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Bone Marrow Disease

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (1)

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.

Biological: CK0801

Interventions

CK0801 BIOLOGICAL

CK0801 (a Cord blood-derived T-regulatory cell product)

CK0801

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who fulfill the diagnostic criteria of bone marrow failure syndrome including: aplastic anemia, myelodysplastic syndrome, or myelofibrosis.
• HLA matched (≥ 3/6) cord blood unit available for CK0801 generation.
• Subjects age ≥ 18 years.
• Bilirubin ≤ 2 x ULN and SGPT (ALT) ≤ 2 x ULN (unless Gilbert's syndrome is documented).
• Calculated creatinine clearance of \> 50mL/min using the Cockcroft-Gault equation.
• Zubrod performance status ≤ 2.
• Female subjects of child bearing potential (FPCP) must have a negative urine or serum pregnancy test. NOTE: FPCP is defined as premenopausal and not surgically sterilized. FPCP must agree to use maximally effective birth control or to abstain from heterosexual activity throughout the study. Effective contraceptive methods include intra-uterine device, oral and/or injectable hormonal; contraception, or 2 adequate barrier methods (e.g., cervical cap with spermicide, diaphragm with spermicide).
• Subject has agreed to abide by all protocol required procedures including study-related assessments, visits and long term follow up.
• Subject is willing and able to provide written informed consent.

You may not qualify if:

• Subject has received an investigational agent within 4 weeks prior to CK0801 infusion.
• Subject has received radiation or chemotherapy within 21 days prior to CK0801 infusion.
• Subject has received prior cord blood-derived T-regulatory therapy.
• HIV seropositivity.
• Subject has uncontrolled infection, not responding to appropriate antimicrobial agents after seven days of therapy. The Protocol PI is the final arbiter of eligibility.
• Subjects with uncontrolled inter-current illness that in the opinion of the investigator would place the patient at greater risk of severe toxicity and/or impair the activity of CK0801
• Subjects is pregnant or breastfeeding.
• Bone marrow failure caused by stem cell transplantation.
• Subjects who are unable to provide consent or who, in the opinion of the Investigator will be unlikely to fully comply with protocol requirements.

Sponsors & Collaborators

• Cellenkos, Inc.: lead

Study Sites (2)

Sarcoma Oncology Research Center, Cancer Center of Southern California

Santa Monica, California, 90403, United States

Location

The University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

• 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.

  PMID: 38804782 DERIVED

MeSH Terms

Conditions

Bone Marrow Diseases

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Tapan M Kadia, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2018
• First Posted: December 12, 2018
• Study Start: May 30, 2019
• Primary Completion (Estimated): May 25, 2027
• Study Completion (Estimated): May 30, 2027
• Last Updated: April 13, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)