Trial Outcomes & Findings for Haploidentical (Half-matched) Related Donor Stem Cell Transplantation Using Killer Immunoglobulin-like Receptors in Addition to Normal Selection Factors to Determine the Best Donor (NCT NCT02880293)
NCT ID: NCT02880293
Last Updated: 2024-12-06
Results Overview
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
44 participants
Primary outcome timeframe
1 year
Results posted on
2024-12-06
Participant Flow
Participant milestones
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Overall Study
STARTED
|
44
|
|
Overall Study
COMPLETED
|
39
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Overall Study
Inevaluable
|
5
|
Baseline Characteristics
Haploidentical (Half-matched) Related Donor Stem Cell Transplantation Using Killer Immunoglobulin-like Receptors in Addition to Normal Selection Factors to Determine the Best Donor
Baseline characteristics by cohort
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
n=44 Participants
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Age, Continuous
|
62 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
32 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
25 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
44 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
n=44 Participants
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Proportion of Patients Undergoing an Allo HCT Transplant Who Have a KIR Favorable Donor.
Pts with KIR favorable donor
|
23 Participants
|
|
Proportion of Patients Undergoing an Allo HCT Transplant Who Have a KIR Favorable Donor.
Pts without KIR favorable donor
|
21 Participants
|
Adverse Events
Patients Will Undergo Donor/Recipient Bone Marrow
Serious events: 10 serious events
Other events: 43 other events
Deaths: 17 deaths
Serious adverse events
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
n=44 participants at risk
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
6.8%
3/44 • 1 year
|
|
Immune system disorders
Allergic reaction
|
2.3%
1/44 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.5%
2/44 • 1 year
|
|
Infections and infestations
Bronchial infection
|
2.3%
1/44 • 1 year
|
|
Psychiatric disorders
Delirium
|
2.3%
1/44 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
2.3%
1/44 • 1 year
|
|
Nervous system disorders
Encephalopathy
|
2.3%
1/44 • 1 year
|
|
Ear and labyrinth disorders
Hearing impaired
|
2.3%
1/44 • 1 year
|
|
Metabolism and nutrition disorders
Hyponatremia
|
2.3%
1/44 • 1 year
|
|
Gastrointestinal disorders
Ileus
|
2.3%
1/44 • 1 year
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.3%
1/44 • 1 year
|
|
Infections and infestations
Kidney infection
|
2.3%
1/44 • 1 year
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
2.3%
1/44 • 1 year
|
|
Infections and infestations
Lung infection
|
9.1%
4/44 • 1 year
|
|
Cardiac disorders
Myocardial infarction
|
2.3%
1/44 • 1 year
|
|
Investigations
Neutrophil count decreased
|
6.8%
3/44 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
4.5%
2/44 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
4.5%
2/44 • 1 year
|
|
Infections and infestations
Sepsis
|
11.4%
5/44 • 1 year
|
|
Infections and infestations
Small intestine infection
|
2.3%
1/44 • 1 year
|
|
Ear and labyrinth disorders
Tinnitus
|
2.3%
1/44 • 1 year
|
|
Ear and labyrinth disorders
Vestibular disorder
|
2.3%
1/44 • 1 year
|
Other adverse events
| Measure |
Patients Will Undergo Donor/Recipient Bone Marrow
n=44 participants at risk
All patients will undergo haploidentical, allogeneic hematopoietic cell transplantation. Conditioning will consist of fludarabine, melphalan, and thiotepa. Graft versus host disease prophylaxis will be with post-transplant cyclophosphamide in addition to standard tacrolimus and mycophenolate mofetil. Donors will undergo HLA and KIR geno- and allotyping to determine the best donor.
melphalan: melphalan (140 mg/m2 IV on day -7)
fludarabine: fludarabine (40 mg/m2/d on days -5 through -2)
thiotepa: thiotepa (5 mg/kg IV on day -67)
Cyclophosphamide: cyclophosphamide (50 mg/kg IV on day +3 and +4)
Mesna
Mycophenolate Mofetil: (15 mg/kg PO/IV TID)
Filgrastim
Tacrolimus
|
|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
2.3%
1/44 • 1 year
|
|
Investigations
Activated partial thromboplastin time prolonged
|
2.3%
1/44 • 1 year
|
|
Renal and urinary disorders
Acute Kidney Injury
|
2.3%
1/44 • 1 year
|
|
Investigations
Alanine aminotransferase increased
|
9.1%
4/44 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
61.4%
27/44 • 1 year
|
|
Metabolism and nutrition disorders
Anorexia
|
2.3%
1/44 • 1 year
|
|
Investigations
Aspartate aminotransferase increased
|
6.8%
3/44 • 1 year
|
|
Investigations
Blood bilirubin increased
|
6.8%
3/44 • 1 year
|
|
Gastrointestinal disorders
Colitis
|
2.3%
1/44 • 1 year
|
|
Renal and urinary disorders
Cystitis noninfective
|
2.3%
1/44 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
2.3%
1/44 • 1 year
|
|
Nervous system disorders
Encephalopathy
|
2.3%
1/44 • 1 year
|
|
Gastrointestinal disorders
Esophagitis
|
2.3%
1/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
2.3%
1/44 • 1 year
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
52.3%
23/44 • 1 year
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
9.1%
4/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
6.8%
3/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
4.5%
2/44 • 1 year
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
6.8%
3/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
9.1%
4/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
34.1%
15/44 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
38.6%
17/44 • 1 year
|
|
Investigations
Lymphocyte count decreased
|
97.7%
43/44 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
2.3%
1/44 • 1 year
|
|
Investigations
Neutrophil count decreased
|
63.6%
28/44 • 1 year
|
|
General disorders
Pain
|
6.8%
3/44 • 1 year
|
|
Investigations
Platelet count decreased
|
97.7%
43/44 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
2.3%
1/44 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.3%
1/44 • 1 year
|
|
Infections and infestations
Sepsis
|
2.3%
1/44 • 1 year
|
|
Ear and labyrinth disorders
Vertigo
|
2.3%
1/44 • 1 year
|
|
Investigations
White blood cell decreased
|
97.7%
43/44 • 1 year
|
Additional Information
Dr. Brian Shaffer, MD
Memorial Sloan Kettering Cancer Center
Phone: 646-608-3737
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place