Myeloid MalignanciesPhase 2NCT05115630
Off-the-shelf NK Cells + SCT for Myeloid Malignancies
A Phase 2 study testing Cyclophosphamide, Mesna and Filgrastim in people with Myeloid Malignancies and Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to learn about the safety and effectiveness of giving KDS-1001 in combination with a standard stem cell transplant to patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myeloid leukemia (CML). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myeloid Malignancies, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- Cyclophosphamide, Mesna and Filgrastim
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Apr 2022to 15 May 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients ages 18 to 70 years old at the time of enrollment.
- Patients weighing at least 42 kg
- Patient with the hematologic malignancies described below, as well as an HLA matched related donor, HLA matched unrelated donor, a haploidentical related donor, or a one antigen mismatched unrelated donor. HLA matching includes HLA A, B, C, and DR-B1.
- Patients must have one of the following diseases:
- Acute myeloid leukemia (AML):
- With one or more high-risk features defined as: (i) Greater than 1 cycle of induction therapy required to achieve remission; (ii) Preceding myelodysplastic syndrome (MDS);
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- Presence of FLT3 mutations or internal tandem duplication or other mutations designated as adverse-risk by the ELN Leukemia Net AML Classification (see Appendix 2):
- Adverse:
- t(6;9)(p23;q34.1); DEK-NUP214
- t(v;11q23.3); KMT2A rearranged
- t(9;22)(q34.1;q11.2); BCR-ABL1
- inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2); GATA2,MECOM(EVI1)
- -5 or del(5q); -7; -17/abn(17p)
- Complex karyotype, monosomal karyotype
- Wild-type NPM1 and FLT3-ITDhigh
- Mutated RUNX1
- Mutated ASXL1
- Mutated TP53 (iv) FAB M6 or M7 classification; (v) Adverse cytogenetics: -5, del 5q, -7, del7q, abnormalities involving 3q, 9q, 11q, 20q, 21q, 17, +8 or complex karyotype [> 3 abnormalities]; or other mutations designated as adverse-risk by the ELN criteria; (vi) Treatment-related AML (vii) Primary induction failure with partial response to therapy who achieve adequate cytoreduction (viii) Aplastic/hypoplastic marrow with or without detectable persistent disease after induction chemotherapy or after salvage chemotherapy.
- Have minimal residual disease by flow cytometry, FISH, detection of disease related mutations or cytogenetic abnormality after first course of induction chemotherapy (x) Have relapsed after prior allogeneic hematopoietic transplant
- AND
- Patients must be in one of the following (i) CR: complete remission, (ii) CRi: CR with incomplete hematologic recovery, or (iii) MLFS: morphological leukemia-free state with less than 5% bone marrow blasts.
- If not in either of the above i-iii, then may be in either of the following:
- Primary induction failure with partial response to therapy who achieve adequate cytoreduction
- Aplastic/hypoplastic marrow with or without detectable persistent disease after induction chemotherapy or after salvage chemotherapy
- Myelodysplastic syndromes (MDS):
- De novo MDS with intermediate or high-risk IPSS scores, chronic myelomonocytic leukemia (CMML) or treatment-related MDS. Patients with intermediate-1 features should have failed to respond to hypomethylating agent therapy. .
- Patients must have less than 10% bone marrow blasts
- Chronic myeloid leukemia (CML):
- Failed to achieve cytogenetic remission or have cytogenetic relapse after treatment with at least 2 tyrosine kinase inhibitors, or
- Accelerated phase or blast phase at any time, or
- Intolerant of available TKIs
- Performance score of at least 70% by Karnofsky or 0 to 1 by ECOG.
- Adequate major non-hematopoietic organ system function as demonstrated by:
- Serum creatinine clearance equal or more than 50 ml/min (calculated with Cockcroft-Gault formula).
- Bilirubin equal or less than 1.5 mg/dL except for Gilbert's disease. ALT or AST equal or less than 200 U/L for adults. Conjugated (direct) bilirubin less than 2x upper limit of normal.
- Left ventricular ejection fraction equal or greater than 45%.
- Diffusing capacity for carbon monoxide (DLCO) equal or greater than 60% predicted corrected for hemoglobin.
- Ability to understand and willingness to sign the written informed consent document.
- Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator while on study.
Who cannot join
- HIV positive; active hepatitis B or C.
- Uncontrolled infections; PI is the final arbiter of this criterion.
- Liver cirrhosis.
- CNS involvement within 3 months prior to the transplant.
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Inability to comply with medical therapy or follow-up.
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- Patient with a known history of allergic reactions to any constituents of the product, including a known history of allergic reactions to cellular products or DMSO.
- Other malignancy/cancer diagnosis with active disease or in remission and <2 years ago, not including nonmelanoma skin cancer
- Requiring systemic corticosteroids with prednisone dose >10 mg or equivalent.
- KDS-1001 Donor specific antibodies (dsa) >3000 MFI units or C1q positive
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I/II Clinical Trial of "Off-the-shelf" NK Cell Administration in Combination With Allogeneic SCT to Decrease Disease Relapse in Patients With High-risk Myeloid Malignancies Undergoing Matched Related, Matched Unrelated, One Antigen Mismatched Unrelated, or Haploidentical Stem-cell Transplantation
The goal of this clinical research study is to learn about the safety and effectiveness of giving KDS-1001 in combination with a standard stem cell transplant to patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myeloid leukemia (CML). KDS-1001 is a study product created using certain immune cells called natural killer (NK) cells collected from a third-party donor.
Read the detailed description
Primary Objective
Assess the safety and effectiveness of "off the shelf" third party NK cells in combination with allogeneic SCT in patients with myeloid malignancies.
Secondary Objectives
To assess NK cell related toxicities To estimate the proportion of patients with engraftment/graft failure. To assess the rate of leukemia relapse, disease-free survival (DFS), overall survival (OS), and GVHD-free, Relapse-free survival (GRFS) after transplantation by one year.
To estimate the non-relapse mortality (NRM) at day 100, day 180 and 1 year post-transplant.
To estimate the cumulative incidence of grade 2-4 and grades 3-4 aGVHD at day 100. To assess the rate of chronic GVHD within the first-year post transplantation. To assess rate of BK, CMV, and Adenovirus infections. To assess MRD. To assess immune reconstitution post-transplant
- Conditions
- Myeloid Malignancies, Acute Myeloid Leukemia, Myelodysplastic Syndrome, Chronic Myeloid Leukemia
- Being tested
- Cyclophosphamide, Mesna, Filgrastim, Melphalan, Fludarabine phosphate
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Given by IV
MesnaDrug
Given by IV
FilgrastimDrug
Given by IV
MelphalanDrug
Given by IV
Given by IV
TacrolimusDrug
Given by IV
Given by IV
Total Body Irradiation One DoseDrug
Given by IV
| Study group | Role | What they receive |
|---|---|---|
| Cyclophosphamide | Experimental | On Days 3 and 4, you will receive cyclophosphamide by vein over about 3 hours to help lower the risk of graft-versus-host disease Receives: Drug: Cyclophosphamide |
| Mesna | Experimental | On Days 3 and 4, You will also receive mesna by vein over 30 minutes every 4 hours for a total of 10 mesna doses. Receives: Drug: Mesna |
| Filgrastim | Experimental | Starting on Day 7, you will begin to receive filgrastim as an injection under the skin 1 time a day. Receives: Drug: Filgrastim |
| Melphalan | Experimental | On Day -7, you will receive melphalan by vein over about 30 minutes. Receives: Drug: Melphalan |
| Fludarabine phosphate | Experimental | On Days -7, -6, -5, and -4, you will receive fludarabine by vein about 1 hour. Receives: Drug: Fludarabine phosphate |
| Tacrolimus | Experimental | Starting on Day 5, you will begin receiving tacrolimus to help lower the risk of GVHD. You will begin by receiving it nonstop by vein until you are able to take it by mouth. You will then take tacrolimus by mouth 2 times a day for about 3 months. Receives: Drug: Tacrolimus |
| Mycophenolate mofetil | Experimental | by mouth 3 times a day for 90 days or longer. Receives: Drug: Mycophenolate mofetil |
| Total Body Irradiation One Dose | Experimental | on Day -2, you may receive 1 dose of total body irradiation (TBI). Receives: Drug: Total Body Irradiation One Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Experienced Graft Failure
Primary Graft failure is defined as failure to achieve an ANC > 0.5 x 109/L for 3 consecutive days by day 28 post SC infusion, with no evidence of donor derived cells by bone marrow chimerism studies and no evidence of persistent or relapsing disease.
Measured: 28 days post-transplant
Secondary outcomes (2)
Number of Participants Experienced Non-relapsed Mortality at 100 Day Post Transplant
Number of participants died from any cause other than relapse disease.
Measured: 100 days post-transplant
Number of Participants in Subsequent Transplant Prior to NK Cell Infusions/Stem Cell Transplant
Number participants had prior allogeneic transplants before study enrollment
Measured: On the day of study consent
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2021
- Study start8 Apr 2022
- Primary completion15 May 2025
- Study completion15 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myeloid MalignanciesStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeremy Ramdial, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05115630
- Sponsor's protocol ID
- 2021-0329
- Other IDs
- NCI-2021-12094
- Status last verified
- Dec 2025
- First submitted
- 11 Oct 2021
- Last updated
- 19 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Clinical Trial of "Off-the-shelf" NK Cell Administration in Combination With Allogeneic SCT to Decrease Disease Relapse in Patients With High-risk Myeloid Malignancies Undergoing Matched Related, Matched Unrelated, One Antigen Mismatched Unrelated, or Haploidentical Stem-cell Transplantation
- ClinicalTrials.gov ID
- NCT05115630
- Acronym / sponsor ID
- 2021-0329
- Conditions
- Myeloid Malignancies, Acute Myeloid Leukemia, Myelodysplastic Syndrome, Chronic Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 8 Apr 2022 (actual)
- Primary completion
- 15 May 2025 (actual)
- Study completion
- 15 May 2025 (actual)
- First posted
- 10 Nov 2021 (actual)
- Results
- Posted 19 Dec 2025
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 learn about the safety and effectiveness of giving KDS-1001 in combination with a standard stem cell transplant to patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myeloid leukemia (CML). KDS-1001 is a study product created using certain immune cells called natural killer (NK) cells collected from a third-party donor.
Detailed description
Primary Objective
Assess the safety and effectiveness of "off the shelf" third party NK cells in combination with allogeneic SCT in patients with myeloid malignancies.
Secondary Objectives
To assess NK cell related toxicities To estimate the proportion of patients with engraftment/graft failure. To assess the rate of leukemia relapse, disease-free survival (DFS), overall survival (OS), and GVHD-free, Relapse-free survival (GRFS) after transplantation by one year.
To estimate the non-relapse mortality (NRM) at day 100, day 180 and 1 year post-transplant.
To estimate the cumulative incidence of grade 2-4 and grades 3-4 aGVHD at day 100. To assess the rate of chronic GVHD within the first-year post transplantation. To assess rate of BK, CMV, and Adenovirus infections. To assess MRD. To assess immune reconstitution post-transplant
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| Cyclophosphamide | Experimental | Drug: CyclophosphamideOn Days 3 and 4, you will receive cyclophosphamide by vein over about 3 hours to help lower the risk of graft-versus-host disease |
| Mesna | Experimental | Drug: MesnaOn Days 3 and 4, You will also receive mesna by vein over 30 minutes every 4 hours for a total of 10 mesna doses. |
| Filgrastim | Experimental | Drug: FilgrastimStarting on Day 7, you will begin to receive filgrastim as an injection under the skin 1 time a day. |
| Melphalan | Experimental | Drug: MelphalanOn Day -7, you will receive melphalan by vein over about 30 minutes. |
| Fludarabine phosphate | Experimental | Drug: Fludarabine phosphateOn Days -7, -6, -5, and -4, you will receive fludarabine by vein about 1 hour. |
| Tacrolimus | Experimental | Drug: TacrolimusStarting on Day 5, you will begin receiving tacrolimus to help lower the risk of GVHD. You will begin by receiving it nonstop by vein until you are able to take it by mouth. You will then take tacrolimus by mouth 2 times a day for about 3 months. |
| Mycophenolate mofetil | Experimental | Drug: Mycophenolate mofetilby mouth 3 times a day for 90 days or longer. |
| Total Body Irradiation One Dose | Experimental | Drug: Total Body Irradiation One Doseon Day -2, you may receive 1 dose of total body irradiation (TBI). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (39 listed)
- Patients ages 18 to 70 years old at the time of enrollment.
- Patients weighing at least 42 kg
- Patient with the hematologic malignancies described below, as well as an HLA matched related donor, HLA matched unrelated donor, a haploidentical related donor, or a one antigen mismatched unrelated donor. HLA matching includes HLA A, B, C, and DR-B1.
- Patients must have one of the following diseases:
- Presence of FLT3 mutations or internal tandem duplication or other mutations designated as adverse-risk by the ELN Leukemia Net AML Classification (see Appendix 2):
Key exclusion criteria (10 listed)
- HIV positive; active hepatitis B or C.
- Uncontrolled infections; PI is the final arbiter of this criterion.
- Liver cirrhosis.
- CNS involvement within 3 months prior to the transplant.
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
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 |
|---|---|---|
| CyclophosphamideGiven by IV | Drug | Cyclophosphamide |
| MesnaGiven by IV | Drug | Mesna |
| FilgrastimGiven by IV | Drug | Filgrastim |
| MelphalanGiven by IV | Drug | Melphalan |
| Fludarabine phosphateGiven by IV | Drug | Fludarabine phosphate |
| TacrolimusGiven by IV | Drug | Tacrolimus |
| Mycophenolate mofetilGiven by IV | Drug | Mycophenolate mofetil |
| Total Body Irradiation One DoseGiven by IV | Drug | Total Body Irradiation One Dose |
Primary outcome
Number of Participants Who Experienced Graft Failure
Time frame: 28 days post-transplant
Primary Graft failure is defined as failure to achieve an ANC > 0.5 x 109/L for 3 consecutive days by day 28 post SC infusion, with no evidence of donor derived cells by bone marrow chimerism studies and no evidence of persistent or relapsing disease.
2 secondary outcomes
Number of Participants Experienced Non-relapsed Mortality at 100 Day Post Transplant
Time frame: 100 days post-transplant
Number of Participants in Subsequent Transplant Prior to NK Cell Infusions/Stem Cell Transplant
Time frame: On the day of study consent
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Jeremy Ramdial, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide, Mesna and Filgrastim in people with Myeloid Malignancies and Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site 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 39 inclusion and 10 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: finished, as of the sponsor's last update on 19 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Who Experienced Graft Failure (time frame: 28 days post-transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Dec 2025.