Trial Outcomes & Findings for Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia (NCT NCT00630253)
NCT ID: NCT00630253
Last Updated: 2021-10-12
Results Overview
graft failure = absolute neutrophil count (ANC) \<5 x 10\^8/L and an acellular bone marrow aspirate/biopsy
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
31 participants
Primary outcome timeframe
From Day 1 to event, assessed up to100 days
Results posted on
2021-10-12
Participant Flow
Participant milestones
| Measure |
Marrow Isolex
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
Umbilical Cord Blood (UCB) Arm
No processing
|
Marrow Clinimax
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Overall Study
STARTED
|
16
|
9
|
6
|
|
Overall Study
COMPLETED
|
16
|
9
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
Baseline characteristics by cohort
| Measure |
Marrow Isolex
n=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
Total
n=31 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
13 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 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
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
16 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
30 Participants
n=4 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
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
4 Participants
n=4 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
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
9 participants
n=7 Participants
|
6 participants
n=5 Participants
|
31 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: From Day 1 to event, assessed up to100 daysgraft failure = absolute neutrophil count (ANC) \<5 x 10\^8/L and an acellular bone marrow aspirate/biopsy
Outcome measures
| Measure |
Marrow Isolex
n=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Number of Participants Experiencing Graft Failure
|
0 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=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Number of Participants With Acute Graft-Versus-Host Disease (GVHD)
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 1 YearThe percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate. Overall survival will be defined as time from enrollment to date of death or censored at the date of last documented contact for patients still alive.
Outcome measures
| Measure |
Marrow Isolex
n=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Number of Participants Experiencing Overall Survival
|
15 Participants
|
8 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: 1 YearChronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Outcome measures
| Measure |
Marrow Isolex
n=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Number of Participants With Chronic Graft-Versus-Host Disease (GVHD)
|
2 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100In the field of transplantation, toxicity is high and all deaths without previous relapse or progression are usually considered as related to transplantation
Outcome measures
| Measure |
Marrow Isolex
n=16 Participants
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 Participants
No processing
|
Marrow Clinimax
n=6 Participants
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Number of Participants With Transplant Related Deaths
|
1 Participants
|
0 Participants
|
1 Participants
|
Adverse Events
Marrow Isolex
Serious events: 0 serious events
Other events: 9 other events
Deaths: 1 deaths
UCB Arm
Serious events: 0 serious events
Other events: 6 other events
Deaths: 1 deaths
Marrow Clinimax
Serious events: 1 serious events
Other events: 5 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Marrow Isolex
n=16 participants at risk
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 participants at risk
No processing
|
Marrow Clinimax
n=6 participants at risk
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Gastrointestinal disorders
Hemorrhage
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Marrow Isolex
n=16 participants at risk
bone marrow processed using Isolex 300i (for patients enrolled through April 2010)
|
UCB Arm
n=9 participants at risk
No processing
|
Marrow Clinimax
n=6 participants at risk
bone marrow processed using CliniMACS (for patients enrolled beginning with the August 2010 protocol version)
|
|---|---|---|---|
|
Nervous system disorders
Brain Infarction
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Cardiac disorders
Cardiac arrect
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Cardiac disorders
Cardiopumonary shock
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Infections and infestations
Cystitis
|
12.5%
2/16 • Number of events 2 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Investigations
Elevaed ALT
|
12.5%
2/16 • Number of events 2 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Investigations
Elevaed bilirubin
|
6.2%
1/16 • Number of events 1 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Gastrointestinal disorders
GI bleeding
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
General disorders
Graft failure
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Renal and urinary disorders
Hematuria
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
Hemolysis
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Infections and infestations
Hepatitis
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/16 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Vascular disorders
Hypertension
|
6.2%
1/16 • Number of events 1 • 1 year
|
22.2%
2/9 • Number of events 2 • 1 year
|
50.0%
3/6 • Number of events 3 • 1 year
|
|
Nervous system disorders
Hypoxic ischemic encephalopathy
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Infections and infestations
Infection
|
43.8%
7/16 • Number of events 27 • 1 year
|
55.6%
5/9 • Number of events 13 • 1 year
|
66.7%
4/6 • Number of events 10 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Intubation
|
0.00%
0/16 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Hepatobiliary disorders
Liver Ischemia
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Nervous system disorders
Neurotoxicity
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Ear and labyrinth disorders
Ototoxicity
|
0.00%
0/16 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Cardiac disorders
Pericardial effusion
|
6.2%
1/16 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
0.00%
0/6 • 1 year
|
|
Infections and infestations
Pneumonia
|
0.00%
0/16 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
33.3%
2/6 • Number of events 2 • 1 year
|
|
Infections and infestations
Pneumonitis
|
0.00%
0/16 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
0.00%
0/6 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hemorrhage
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory acidosis
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/16 • 1 year
|
0.00%
0/9 • 1 year
|
16.7%
1/6 • Number of events 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