Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia (NCT NCT00258427)
NCT ID: NCT00258427
Last Updated: 2021-12-02
Results Overview
Graft failure is defined as absolute neutrophil count( ANC ) \<5 x 10\^8/L by day 30.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
Day 30
Results posted on
2021-12-02
Participant Flow
Participant milestones
| Measure |
Marrow Isolex
Bone marrow processed using Isolex300i
|
USB Arm
No processing
|
Marrow Clinimacs
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
8
|
2
|
1
|
|
Overall Study
COMPLETED
|
3
|
8
|
2
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Baseline characteristics by cohort
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
Total
n=14 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
5 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
9 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
1 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
3 Participants
n=21 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
8 Participants
n=21 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
2 Participants
n=21 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
14 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Day 30Graft failure is defined as absolute neutrophil count( ANC ) \<5 x 10\^8/L by day 30.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Graft Failure
|
1 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 42Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Chronic Graft-Versus-Host Disease
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearChronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Chronic Graft-Versus-Host Disease
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 yearAcute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Acute Graft-Versus-Host Disease
|
0 Participants
|
1 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 42Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Acute Graft-Versus-Host Disease
|
0 Participants
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 YearPatients with leukemia will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate.
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Relapse
|
0 Participants
|
2 Participants
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 YearOverall Survival - Number of patients alive at 1 year post transplant
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Overall Survival
|
1 Participants
|
4 Participants
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day 1 through 1 year post-transplantNumber of participants experiencing Major Infections by the end of treatment
Outcome measures
| Measure |
Marrow Isolex
n=3 Participants
Bone marrow processed using Isolex300i
|
USB Arm
n=8 Participants
No processing
|
Marrow Clinimacs
n=2 Participants
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 Participants
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Number of Participants Experiencing Major Infections
|
3 Participants
|
8 Participants
|
2 Participants
|
1 Participants
|
Adverse Events
Marrow Isolex
Serious events: 2 serious events
Other events: 3 other events
Deaths: 2 deaths
USB Arm
Serious events: 2 serious events
Other events: 7 other events
Deaths: 4 deaths
Marrow Clinimacs
Serious events: 1 serious events
Other events: 2 other events
Deaths: 1 deaths
Sibling withoutCliniMACS
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Marrow Isolex
n=3 participants at risk
Bone marrow processed using Isolex300i
|
USB Arm
n=8 participants at risk
No processing
|
Marrow Clinimacs
n=2 participants at risk
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 participants at risk
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Death
|
66.7%
2/3 • Number of events 2 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
diffuse alveolar hemorrhage
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
General disorders
Primary Graft Failure
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Relapse
|
0.00%
0/3 • 1 year
|
0.00%
0/8 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/1 • 1 year
|
Other adverse events
| Measure |
Marrow Isolex
n=3 participants at risk
Bone marrow processed using Isolex300i
|
USB Arm
n=8 participants at risk
No processing
|
Marrow Clinimacs
n=2 participants at risk
Bone marrow processed using CliniMACS
|
Sibling withoutCliniMACS
n=1 participants at risk
sibling donor without use of CliniMACS system
|
|---|---|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Acute hypoxic respiratory failure
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/3 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
acute respiratory distress syndrome
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Surgical and medical procedures
Chest Tube Placement
|
33.3%
1/3 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Surgical and medical procedures
Chest Xray
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Blood and lymphatic system disorders
Coagulopathy
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Renal and urinary disorders
Dialysis
|
33.3%
1/3 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Blood and lymphatic system disorders
Disseminated intravascular coagulation
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
General disorders
Drug Rash with Eosinophilia
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
General disorders
Engraftment syndrome
|
0.00%
0/3 • 1 year
|
25.0%
2/8 • Number of events 3 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Gastrointestinal disorders
Eosinophilic gut
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Gastrointestinal disorders
GI bleeding
|
33.3%
1/3 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Ear and labyrinth disorders
hearing loss
|
0.00%
0/3 • 1 year
|
0.00%
0/8 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/1 • 1 year
|
|
Vascular disorders
Hypertension
|
0.00%
0/3 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
100.0%
1/1 • Number of events 1 • 1 year
|
|
Infections and infestations
Infection
|
100.0%
3/3 • Number of events 18 • 1 year
|
75.0%
6/8 • Number of events 18 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
100.0%
1/1 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Intubation
|
66.7%
2/3 • Number of events 2 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Cardiac disorders
pericardial effusion
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 2 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Surgical and medical procedures
Pleuracentesis
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Infections and infestations
Pneumonia
|
33.3%
1/3 • Number of events 1 • 1 year
|
37.5%
3/8 • Number of events 10 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/1 • 1 year
|
|
Gastrointestinal disorders
Pneumoperitoneum
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
General disorders
Primary Graft Failure
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary hemorrhage
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Renal and urinary disorders
Renal failure
|
33.3%
1/3 • Number of events 1 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress syndrome
|
0.00%
0/3 • 1 year
|
12.5%
1/8 • Number of events 1 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Infections and infestations
Thrush
|
0.00%
0/3 • 1 year
|
0.00%
0/8 • 1 year
|
50.0%
1/2 • Number of events 1 • 1 year
|
0.00%
0/1 • 1 year
|
|
Nervous system disorders
Unresponsive
|
33.3%
1/3 • Number of events 1 • 1 year
|
0.00%
0/8 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
|
Hepatobiliary disorders
Veno occlusive disease
|
33.3%
1/3 • Number of events 1 • 1 year
|
25.0%
2/8 • Number of events 2 • 1 year
|
0.00%
0/2 • 1 year
|
0.00%
0/1 • 1 year
|
Additional Information
Margaret L. MacMillan, M.D.
Masonic Cancer Center, University of Minnesota
Phone: 612-273-2800
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place