CK0801 for Treatment of Aplastic Anemia

NCT07499102 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CK0801-301-01
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care treatment. CK0801 is an allogeneic cord blood-derived regulatory T-cell therapy administered intravenously. The study is designed to assess safety and clinical activity, including hematologic response, transfusion independence, duration of response, survival outcomes, and patient-reported outcomes. Exploratory assessments include immune reconstitution, biomarkers, pharmacokinetics, immunogenicity, and donor-specific antibodies. The primary endpoint is overall response at Day 180.

Conditions

Aplastic Anemia

Keywords

Aplastic Anemia; Bone Marrow Failure; CK0801; Regulatory T Cells; Treg Cell Therapy; Cord Blood-Derived Tregs; Allogeneic Cell Therapy; Transfusion Dependent; Immune Dysregulation; Open-Label Phase 2 Study; Cord Blood Unit

Outcome Measures

Primary Outcomes (2)

• Overall Response Rate (OR) at Day +180

  Overall response at Day +180 after treatment with CK0801. Response is based on transfusion improvement and includes complete response (CR) and partial response (PR), where CR is defined as transfusion independence for 4 consecutive weeks and PR is defined as a decrease in transfusion requirements.

  Day +180

• Overall Response Rate (OR) at Day +180

  Overall response at Day +180 after treatment with CK0801. Response is based on transfusion improvement and includes complete response (CR) and partial response (PR), where CR is defined as transfusion independence for 4 consecutive weeks and PR is defined as a decrease in transfusion requirements.

  Day+180

Study Arms (1)

CK0801

EXPERIMENTAL

All enrolled participants receive CK0801 administered intravenously according to the protocol. CK0801 is given as a weight-based dose of 1 x 10\^7 Tregs/kg using actual body weight, capped at 100 kg, on Study Day +1.

Drug: CK0801

Interventions

CK0801 DRUG

CK0801 is an investigational allogeneic, cord blood-derived regulatory T-cell product administered by intravenous infusion. The planned dose is 1 x 10\^7 CK0801 Tregs/kg using actual body weight, capped at 100 kg.

CK0801

Eligibility Criteria

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

You may qualify if:

• Age 18 years or older
• Diagnosis of aplastic anemia
• Transfusion dependent after at least one prior line of treatment, or intolerance to standard-of-care treatment
• Female subjects of childbearing potential must have a negative urine or serum pregnancy test
• Agrees to comply with all protocol-required procedures, including study-related assessments, visits, and long-term follow-up
• Willing and able to provide written informed consent

You may not qualify if:

• Known HIV seropositivity
• Uncontrolled infection not responding to appropriate antimicrobial agents after 7 days of therapy; the Protocol PI is the final arbiter of eligibility
• Uncontrolled intercurrent illness that, in the opinion of the investigator, would place the subject at greater risk of severe toxicity and/or impair the activity of CK0801
• Pregnant or breastfeeding
• Unable to provide consent or, in the opinion of the investigator, unlikely to fully comply with protocol requirements

Sponsors & Collaborators

• Cellenkos, Inc.: lead

MeSH Terms

Conditions

Anemia, Aplastic; Bone Marrow Failure Disorders

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single-group, open-label, multicenter Phase 2 study in which all enrolled participants receive CK0801. Participants are assigned to one treatment group and receive CK0801 as a single-agent intravenous infusion, with efficacy and safety assessed over protocol-defined follow-up visits.

Study Record Dates

• First Submitted: March 24, 2026
• First Posted: March 30, 2026
• Study Start (Estimated): August 3, 2026
• Primary Completion (Estimated): August 3, 2028
• Study Completion (Estimated): August 3, 2029
• Last Updated: March 30, 2026

Record last verified: 2026-03