Aplastic AnemiaPhase 2NCT07499102
CK0801 for Treatment of Aplastic Anemia
A Phase 2 study testing CK0801 in people with Aplastic Anemia, run by Cellenkos, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- CK0801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- 3 Dec 2026to 3 Aug 2029
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
About this study
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.
Read the detailed description
Aplastic anemia is a rare, life-threatening bone marrow failure disorder caused by immune-mediated destruction of hematopoietic stem and progenitor cells. Regulatory T cells (Tregs), which help maintain immune homeostasis, are decreased and functionally impaired in aplastic anemia. CK0801 is an allogeneic cord blood-derived Treg cell therapy being studied as a potential treatment to restore immune balance and improve hematopoiesis. Prior clinical experience described in the protocol showed a favorable safety profile and early signs of activity, including reduced transfusion requirements in patients with bone marrow failure.
This Phase 2, multicenter, open-label, single-agent study will evaluate the safety and efficacy of intravenous CK0801 in adults with aplastic anemia who remain transfusion dependent after at least one prior line of therapy or who are intolerant to standard-of-care treatment. A total of 12 participants are planned. CK0801 will be administered as a weight-based intravenous infusion, with protocol-defined premedication before treatment.
The primary endpoint is overall response at Day 180. Secondary endpoints include response at Days 28, 56, 100, and 365, time to best response, time to transfusion independence, duration of transfusion independence, immunosuppression-free survival, overall survival, progression-free survival, clonal evolution, patient-reported outcomes, and safety. Exploratory endpoints include donor-specific antibodies, immunosuppressive medication discontinuation, cytokine and biomarker changes, pharmacokinetics, immunogenicity, and immune reconstitution.
Enrollment will proceed using Simon's two-stage design. In Stage 1, 6 participants will be enrolled. If predefined activity criteria are met, the study will proceed to Stage 2 and enroll 6 additional participants, for a total of 12 participants. Participants will undergo protocol-defined clinical, laboratory, disease, safety, and correlative assessments. The overall study duration is expected to be approximately 24 months.
- Conditions
- Aplastic Anemia
- Being tested
- CK0801
- 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
- Medical subject headings
- Anemia, Aplastic, Bone Marrow Failure Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CK0801Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: CK0801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Day +180
Overall Response Rate at Additional Timepoints
Proportion of subjects achieving CR or PR at earlier and later timepoints following treatment
Measured: Days 28, 56, 100, and 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2026
- Study start3 Dec 2026 (estimated)
- Primary completion3 Aug 2028 (estimated)
- Study completion3 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellenkos, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07499102
- Sponsor's protocol ID
- CK0801-301-01
- Status last verified
- Oct 2026
- First submitted
- 24 Mar 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No