Trial Outcomes & Findings for Preventing Stem Cell Transplant Complications With a Blood Separator Machine (NCT NCT01866839)
NCT ID: NCT01866839
Last Updated: 2020-07-21
Results Overview
Determine the rate of overall survival at 200 day
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
41 participants
Primary outcome timeframe
200 days
Results posted on
2020-07-21
Participant Flow
1 participant signed consent but did not start study
Participant milestones
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Overall Study
STARTED
|
40
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Preventing Stem Cell Transplant Complications With a Blood Separator Machine
Baseline characteristics by cohort
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=39 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
32 Participants
n=39 Participants
|
|
Age, Categorical
>=65 years
|
6 Participants
n=39 Participants
|
|
Sex: Female, Male
Female
|
21 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
21 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
4 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
15 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: 200 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Determine the rate of overall survival at 200 day
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Overall Survival
|
36 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Disease Free Survival at 2 years
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Disease Free Survival at 2 Years
|
60.8 percentage of participant
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Incidence of relapse / disease progression
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Disease Progression
|
11 Participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Participants who develop acute GVHD
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Incidence of Acute GVHD
|
26 Participants
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Participants who develop chronic GVHD
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Incidence of Chronic GVHD
|
14 Participants
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Time in days
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Median Time to ANC>500/mm3
|
12 Days
Interval 9.0 to 28.0
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Time in days
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Median Time to Platelets>20K/mm3
|
15 Days
Interval 14.0 to 28.0
|
SECONDARY outcome
Timeframe: 100 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Grade I
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Severity of Acute GVHD
|
10 Participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Grade II
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Severity of Acute GVHD
|
7 Participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Grade III
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
|
|---|---|
|
Severity of Acute GVHD
|
8 Participants
|
SECONDARY outcome
Timeframe: 100 daysPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Grade IV
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Severity of Acute GVHD
|
1 Participants
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Extensive
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Severity of Chronic GVHD
|
10 Participants
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Subjects for whom the Miltenyi CliniMACS CD34 selection system was used for graft manipulation in HLA-matched sibling allogeneic stem cell transplantation
Limited
Outcome measures
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 Participants
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
|
Severity of Chronic GVHD
|
4 Participants
|
Adverse Events
CD34+ Cell Positively Selected Graft Stem Cell Recipient
Serious events: 38 serious events
Other events: 0 other events
Deaths: 11 deaths
Serious adverse events
| Measure |
CD34+ Cell Positively Selected Graft Stem Cell Recipient
n=40 participants at risk
Recipients received a myeloablative conditioning regimen of cyclophosphamide (120 mg/kg total), fludarabine (125 mg/m2 total) and total body irradiation (1200 cGy with lung shielding to 600 cGy), followed by an infusion of a stem cell product selected for CD34+ progenitors using the Miltenyi CliniMACS® system. Older subjects will receive a lower dose of irradiation (800 or 600 cGy based on age) to reduce the regimen intensity.
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|---|---|
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Blood and lymphatic system disorders
Anaemia
|
2.5%
1/40 • 3 years
|
|
Blood and lymphatic system disorders
Engraftment syndrome
|
2.5%
1/40 • 3 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
2.5%
1/40 • 3 years
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
2.5%
1/40 • 3 years
|
|
Cardiac disorders
Cardiac failure congestive
|
2.5%
1/40 • 3 years
|
|
Cardiac disorders
Cardiomyopathy acute
|
2.5%
1/40 • 3 years
|
|
Cardiac disorders
Diastolic dysfunction
|
2.5%
1/40 • 3 years
|
|
Cardiac disorders
Fluid overload
|
5.0%
2/40 • 3 years
|
|
Cardiac disorders
Tachycardia
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Abdominal discomfort
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Appendicitis
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Clostridium difficile colitis
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Constipation
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Cytomegalovirus gastrointestinal infection
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Diarrhoea
|
7.5%
3/40 • 3 years
|
|
Gastrointestinal disorders
Gastritis
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Gastroenteritis astroviral
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Nausea
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Viral diarrhoea
|
2.5%
1/40 • 3 years
|
|
Gastrointestinal disorders
Vomiting
|
2.5%
1/40 • 3 years
|
|
General disorders
Adult failure to thrive
|
2.5%
1/40 • 3 years
|
|
General disorders
Catheter site pain
|
2.5%
1/40 • 3 years
|
|
General disorders
Drug withdrawal convulsions
|
2.5%
1/40 • 3 years
|
|
General disorders
Drug withdrawal syndrome
|
2.5%
1/40 • 3 years
|
|
General disorders
Incarcerated hernia
|
2.5%
1/40 • 3 years
|
|
General disorders
Pain
|
2.5%
1/40 • 3 years
|
|
Immune system disorders
Acute graft versus host disease in intestine
|
17.5%
7/40 • 3 years
|
|
Immune system disorders
Acute graft versus host disease in skin
|
7.5%
3/40 • 3 years
|
|
Immune system disorders
Chronic graft versus host disease
|
2.5%
1/40 • 3 years
|
|
Immune system disorders
Chronic graft versus host disease in intestine
|
7.5%
3/40 • 3 years
|
|
Immune system disorders
Food allergy
|
2.5%
1/40 • 3 years
|
|
Immune system disorders
Reaction to preservatives
|
2.5%
1/40 • 3 years
|
|
Immune system disorders
Transplant rejection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Adenovirus infection
|
5.0%
2/40 • 3 years
|
|
Infections and infestations
Bacteraemia
|
15.0%
6/40 • 3 years
|
|
Infections and infestations
BK virus infection
|
5.0%
2/40 • 3 years
|
|
Infections and infestations
Candida infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Cellulitis
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Clostridium difficile infection
|
12.5%
5/40 • 3 years
|
|
Infections and infestations
Cystitis
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Cytomegalovirus infection
|
17.5%
7/40 • 3 years
|
|
Infections and infestations
Epstein-Barr viraemia
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Escherichia bacteraemia
|
7.5%
3/40 • 3 years
|
|
Infections and infestations
Escherichia sepsis
|
5.0%
2/40 • 3 years
|
|
Infections and infestations
Fungaemia
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Influenza
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Klebsiella infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Lung infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Pneumonia
|
12.5%
5/40 • 3 years
|
|
Infections and infestations
Pneumonia fungal
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Pyelonephritis
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Salmonella sepsis
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Sepsis
|
10.0%
4/40 • 3 years
|
|
Infections and infestations
Septic shock
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Stenotrophomonas infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
suspected infection
|
12.5%
5/40 • 3 years
|
|
Infections and infestations
Upper respiratory tract infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Urinary tract infection
|
7.5%
3/40 • 3 years
|
|
Infections and infestations
Varicella zoster virus infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Vulvovaginal human papilloma virus infection
|
2.5%
1/40 • 3 years
|
|
Infections and infestations
Wound abscess
|
2.5%
1/40 • 3 years
|
|
Injury, poisoning and procedural complications
Delayed engraftment
|
2.5%
1/40 • 3 years
|
|
Injury, poisoning and procedural complications
Engraft failure
|
2.5%
1/40 • 3 years
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
2.5%
1/40 • 3 years
|
|
Injury, poisoning and procedural complications
Procedural headache
|
2.5%
1/40 • 3 years
|
|
Investigations
Anion gap
|
2.5%
1/40 • 3 years
|
|
Investigations
Body temperature increased
|
30.0%
12/40 • 3 years
|
|
Investigations
Hyperkalaemia
|
2.5%
1/40 • 3 years
|
|
Nervous system disorders
Headache
|
2.5%
1/40 • 3 years
|
|
Nervous system disorders
Orthostatic hypotension
|
2.5%
1/40 • 3 years
|
|
Nervous system disorders
Posterior reversible encephalopathy syndrome
|
2.5%
1/40 • 3 years
|
|
Psychiatric disorders
Altered state of consciousness
|
2.5%
1/40 • 3 years
|
|
Psychiatric disorders
Mental status changes
|
2.5%
1/40 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
2.5%
1/40 • 3 years
|
|
Surgical and medical procedures
Mucositis management
|
2.5%
1/40 • 3 years
|
|
Vascular disorders
Orthostatic hypotension
|
2.5%
1/40 • 3 years
|
|
Vascular disorders
Syncope
|
2.5%
1/40 • 3 years
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place