CK0801 for Treatment of Aplastic Anemia

ConditionAplastic Anemia

Trial statusNot yet recruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorCellenkos, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

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.

Treatment groups

12 Participants

are divided into 1 treatment group

Group A: CK0801Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

Primary Outcome-Overall Response Rate (ORR) at Day 180

Overall response rate (ORR), defined as the proportion of subjects achieving either a complete response (CR) or partial response (PR) following treatment with CK0801.

Time frame

Day +180

Overall Response Rate at Additional Timepoints

Proportion of subjects achieving CR or PR at earlier and later timepoints following treatment

Time frame

Days 28, 56, 100, and 365

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Cellenkos, Inc.

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: