Bone Marrow DiseasePhase 1NCT03773393
A Clinical Trial of CK0801 (a New Drug) in Patients With Bone Marrow Failure Syndrome (BMF)
A Phase 1 study testing CK0801 in people with Bone Marrow Disease, run by Cellenkos, Inc. at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Bone Marrow Disease
- 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
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 30 May 2019to 30 May 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- 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.
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
Sarcoma Oncology Research Center, Cancer Center of Southern California
Santa Monica, California, 90403
About this study
Official title: Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome
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.
Read the detailed description
Primary Objective:
To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at the time of CK00801 infusion
- Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0)
- Regimen related death within 30 days
- Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days
Secondary Objective:
- Preliminary assessment of disease-specific response
- Duration of disease-specific response
- Conditions
- Bone Marrow Disease
- Being tested
- CK0801
- Medical subject headings
- Bone Marrow Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CK0801Biological
CK0801 (a Cord blood-derived T-regulatory cell product)
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: CK0801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0
Number of Participants with Severe (Grade 3 or 4) Toxicity
Measured: 24 hours post-intervention
Number of Participants with Regimen Related Death
Number of Participants with Regimen Related Death
Measured: 30 days post-intervention
Number of Participants with Severe Cytokine Release Syndrome (CRS)
Number of Participants with Severe (Grade 3 or 4) CRS
Measured: 30 days post-intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2018
- Study start30 May 2019
- Primary completion25 May 2027 (estimated)
- Study completion30 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Bone Marrow DiseaseStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellenkos, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Tapan M Kadia, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03773393
- Sponsor's protocol ID
- CK0801-101-1
- Status last verified
- Oct 2026
- First submitted
- 23 Oct 2018
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 38804782
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Trial to Evaluate the Safety and Feasibility of CK0801 in Treatment of Bone Marrow Failure Syndrome
- ClinicalTrials.gov ID
- NCT03773393
- Acronym / sponsor ID
- CK0801-101-1
- Conditions
- Bone Marrow Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Cellenkos, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 May 2019 (actual)
- Primary completion
- 25 May 2027 (estimated)
- Study completion
- 30 May 2027 (estimated)
- First posted
- 12 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Primary Objective:
To determine dose-limiting toxicity of CK0801 as defined as any of the events each start at the time of CK00801 infusion
- Severe (grade 3 or 4) infusion toxicity within 24 hours (NCI-CTCAE V4.0)
- Regimen related death within 30 days
- Severe (grade 3 or 4) Cytokine Release Syndrome within 30 days
Secondary Objective:
- Preliminary assessment of disease-specific response
- Duration of disease-specific response
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CK0801 | Experimental | Biological: CK0801All 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CK0801CK0801 (a Cord blood-derived T-regulatory cell product) | Biological | CK0801 |
Primary outcome
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 Regimen Related Death
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
- Derived from this study: PubMed 38804782Kadia 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tapan M Kadia, MD principal investigator
M.D. Anderson Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CK0801 in people with Bone Marrow Disease, run by Cellenkos, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Severe Infusion Toxicity as Assessed by CTCAE v4.0 (time frame: 24 hours post-intervention). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03773393, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.