Trial Outcomes & Findings for Off-the-shelf NK Cells + SCT for Myeloid Malignancies (NCT NCT05115630)
NCT ID: NCT05115630
Last Updated: 2025-12-19
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
28 days post-transplant
Results posted on
2025-12-19
Participant Flow
All participants were registered in MD Anderson Cancer Center
Participant milestones
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Overall Study
Due to COVID19
|
1
|
|
Overall Study
Due to Bacteremia
|
1
|
|
Overall Study
Due to viral infection
|
1
|
Baseline Characteristics
Off-the-shelf NK Cells + SCT for Myeloid Malignancies
Baseline characteristics by cohort
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=24 Participants
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=8 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=8 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=8 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=8 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=8 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: 28 days post-transplantPrimary 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.
Outcome measures
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=21 Participants
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Number of Participants Who Experienced Graft Failure
|
0 Participants
|
SECONDARY outcome
Timeframe: 100 days post-transplantNumber of participants died from any cause other than relapse disease.
Outcome measures
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=21 Participants
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Number of Participants Experienced Non-relapsed Mortality at 100 Day Post Transplant
|
4 Participants
|
SECONDARY outcome
Timeframe: On the day of study consentNumber participants had prior allogeneic transplants before study enrollment
Outcome measures
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=21 Participants
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Number of Participants in Subsequent Transplant Prior to NK Cell Infusions/Stem Cell Transplant
|
10 Participants
|
Adverse Events
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
Serious events: 5 serious events
Other events: 20 other events
Deaths: 12 deaths
Serious adverse events
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=21 participants at risk
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
ARDS
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Infections and infestations
Bacterial
|
23.8%
5/21 • Number of events 6 • 100 days
|
|
Gastrointestinal disorders
Diarrhea
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Nervous system disorders
Encephalopathy
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Infections and infestations
Fungal
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Gastrointestinal bleeding
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Nervous system disorders
Guillain-Barre Syndrome
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Blood and lymphatic system disorders
HSCT related microangiopathy (TA-TMA)
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Blood and lymphatic system disorders
Low granulocyte
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Cardiac disorders
Pericardial effusion
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Cardiac disorders
Pericarditis
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Investigations
Platelet count decreased
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Blood and lymphatic system disorders
Secondary graft failure
|
14.3%
3/21 • Number of events 4 • 100 days
|
|
Infections and infestations
Viral
|
9.5%
2/21 • Number of events 2 • 100 days
|
Other adverse events
| Measure |
NK Cell Infusion in Combination With Fludarabine Melphalan and TBI for Participants Undergoing SCT
n=21 participants at risk
D-7 to D-6 Melphalan Administration: Patients \< age 60 receive melphalan 140 mg/m2 IV split into two doses (70 mg/m2 IV each day).
Patients age 60-70 receive melphalan 100 mg/m2 IV (infused per package insert) split into two doses (50 mg/m2 IV each day).
D-7 to D-4 Fludarabine Administration. Fludarabine will be administered at the dose of 40 mg/m2 IV daily for four doses on days -7 to -4.
D-3 TBI 200 cGy on D-3. D-2 NK cell (KDS-1001) administration D+3 and D+4 Post Transplant D+5 GvHD prophylaxis with Tacrolimus and Mycophenolate Mofetil
|
|---|---|
|
Infections and infestations
Viral
|
42.9%
9/21 • Number of events 14 • 100 days
|
|
Hepatobiliary disorders
VOD
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Abdominal pain
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Immune system disorders
Allergic reaction
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Investigations
ALT increased
|
33.3%
7/21 • Number of events 7 • 100 days
|
|
Metabolism and nutrition disorders
Anorexia
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Investigations
AST increased
|
28.6%
6/21 • Number of events 6 • 100 days
|
|
Infections and infestations
Bacterial
|
66.7%
14/21 • Number of events 25 • 100 days
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Cardiac disorders
Chest pain
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Constipation
|
14.3%
3/21 • Number of events 3 • 100 days
|
|
Investigations
Creatinine increased
|
47.6%
10/21 • Number of events 10 • 100 days
|
|
Renal and urinary disorders
Cystitis noninfective
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Gastrointestinal disorders
Diarrhea
|
66.7%
14/21 • Number of events 15 • 100 days
|
|
Gastrointestinal disorders
Dyspepsia
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Cardiac disorders
Ejection fraction decreased
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Nervous system disorders
Encephalopathy
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
42.9%
9/21 • Number of events 9 • 100 days
|
|
General disorders
Fluid overload
|
81.0%
17/21 • Number of events 17 • 100 days
|
|
Infections and infestations
Fungal
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Nervous system disorders
Headache
|
19.0%
4/21 • Number of events 4 • 100 days
|
|
Renal and urinary disorders
Hemorrhagic Cystitis
|
47.6%
10/21 • Number of events 10 • 100 days
|
|
Gastrointestinal disorders
Hemorrhoids
|
33.3%
7/21 • Number of events 7 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
19.0%
4/21 • Number of events 4 • 100 days
|
|
Blood and lymphatic system disorders
HSCT related microangiopathy (TA-TMA)
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Vascular disorders
Hypertension
|
42.9%
9/21 • Number of events 9 • 100 days
|
|
Metabolism and nutrition disorders
Hypokalemia
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Vascular disorders
Hypotension
|
28.6%
6/21 • Number of events 6 • 100 days
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Nausea
|
95.2%
20/21 • Number of events 28 • 100 days
|
|
Eye disorders
Ocular GvHD
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Oral GvHD
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
Oral mucositis
|
42.9%
9/21 • Number of events 9 • 100 days
|
|
Cardiac disorders
Pericardial effusion
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
4.8%
1/21 • Number of events 1 • 100 days
|
|
Skin and subcutaneous tissue disorders
Rash
|
57.1%
12/21 • Number of events 16 • 100 days
|
|
Investigations
T bilirubin increased
|
14.3%
3/21 • Number of events 3 • 100 days
|
|
Cardiac disorders
Tachycardia
|
9.5%
2/21 • Number of events 2 • 100 days
|
|
Vascular disorders
Thromboembolic event
|
19.0%
4/21 • Number of events 4 • 100 days
|
|
Nervous system disorders
Tremor
|
14.3%
3/21 • Number of events 3 • 100 days
|
Additional Information
Jeremy Ramdial, MD./ Stem Cell Transplantation Department
University of Texas MD Anderson Cancer Center
Phone: 713-745-0146
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place