Off-the-shelf NK Cells + SCT for Myeloid Malignancies

NCT05115630 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: 2021-0329NCI-2021-12094
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief 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). KDS-1001 is a study product created using certain immune cells called natural killer (NK) cells collected from a third-party donor.

Conditions

Myeloid Malignancies; Acute Myeloid Leukemia; Myelodysplastic Syndrome; Chronic Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• 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.

  28 days post-transplant

Secondary Outcomes (2)

• Number of Participants Experienced Non-relapsed Mortality at 100 Day Post Transplant

  100 days post-transplant

• Number of Participants in Subsequent Transplant Prior to NK Cell Infusions/Stem Cell Transplant

  On the day of study consent

Study Arms (8)

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

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.

Drug: Mesna

Filgrastim

EXPERIMENTAL

Starting on Day 7, you will begin to receive filgrastim as an injection under the skin 1 time a day.

Drug: Filgrastim

Melphalan

EXPERIMENTAL

On Day -7, you will receive melphalan by vein over about 30 minutes.

Drug: Melphalan

Fludarabine phosphate

EXPERIMENTAL

On Days -7, -6, -5, and -4, you will receive fludarabine by vein about 1 hour.

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.

Drug: Tacrolimus

Mycophenolate mofetil

EXPERIMENTAL

by mouth 3 times a day for 90 days or longer.

Drug: Mycophenolate mofetil

Total Body Irradiation One Dose

EXPERIMENTAL

on Day -2, you may receive 1 dose of total body irradiation (TBI).

Drug: Total Body Irradiation One Dose

Interventions

Melphalan DRUG

Given by IV

Melphalan

Fludarabine phosphate DRUG

Given by IV

Fludarabine phosphate

Tacrolimus DRUG

Given by IV

Tacrolimus

Mycophenolate mofetil DRUG

Given by IV

Mycophenolate mofetil

Total Body Irradiation One Dose DRUG

Given by IV

Total Body Irradiation One Dose

Cyclophosphamide DRUG

Given by IV

Cyclophosphamide

Mesna DRUG

Given by IV

Mesna

Filgrastim DRUG

Given by IV

Filgrastim

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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):
• a. 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);
• 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)
• or del(5q); -7; -17/abn(17p)
• Complex karyotype, monosomal karyotype
• Wild-type NPM1 and FLT3-ITDhigh

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

M D Anderson Caner Center

Houston, Texas, 77030, United States

Location

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Interventions

Cyclophosphamide; Mesna; Filgrastim; Melphalan; fludarabine phosphate; Tacrolimus; Mycophenolic Acid; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfhydryl Compounds; Sulfur Compounds; Sulfonic Acids; Sulfur Acids; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Radiotherapy; Therapeutics; Investigative Techniques

Results Point of Contact

• Title: Jeremy Ramdial, MD./ Stem Cell Transplantation Department
• Organization: University of Texas MD Anderson Cancer Center

JLRamdial@mdanderson.org

713-745-0146

Study Officials

• Jeremy Ramdial, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

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

Study Record Dates

• First Submitted: October 11, 2021
• First Posted: November 10, 2021
• Study Start: April 8, 2022
• Primary Completion: May 15, 2025
• Study Completion: May 15, 2025
• Last Updated: December 19, 2025
• Results First Posted: December 19, 2025

Record last verified: 2025-12

Locations

US (1)