Trial Outcomes & Findings for A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia (NCT NCT03600909)
NCT ID: NCT03600909
Last Updated: 2022-04-12
Results Overview
Primary non-engraftment is diagnosed when the patient fails to achieve an ANC ≥500/µl at any time in the first 28 days post-transplant. If (1) after achievement of an ANC ≥500/mm\^3, the ANC declines to \<500/mm\^3 for more than 3 consecutive days in the absence of relapse, or, (2) there is absence of donor cells in the marrow and/or blood as demonstrated by chimerism assay in the absence of relapse, a diagnosis of secondary graft failure is made. The patient is not evaluable for graft failure or rejection if recurrence of host MDS is detected concurrently.
TERMINATED
PHASE2
3 participants
5 years
2022-04-12
Participant Flow
Participant milestones
| Measure |
Good Risk Patients
Patients 18 years old or younger with marrow aplasia or single lineage cytopenias (Arm A) will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.6-0.8 mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
Intermediate Risk Patients
Patients 18 years old or younger with MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8-1.0mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
High Risk Patients
Patients 19 years old or older with marrow aplasia or MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.4mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
|---|---|---|---|
|
Overall Study
STARTED
|
0
|
3
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
3
|
0
|
Reasons for withdrawal
| Measure |
Good Risk Patients
Patients 18 years old or younger with marrow aplasia or single lineage cytopenias (Arm A) will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.6-0.8 mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
Intermediate Risk Patients
Patients 18 years old or younger with MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8-1.0mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
High Risk Patients
Patients 19 years old or older with marrow aplasia or MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.4mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
|---|---|---|---|
|
Overall Study
Not treated
|
0
|
3
|
0
|
Baseline Characteristics
A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia
Baseline characteristics by cohort
| Measure |
Good Risk Patients
Patients 18 years old or younger with marrow aplasia or single lineage cytopenias (Arm A) will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.6-0.8 mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
Intermediate Risk Patients
n=3 Participants
Patients 18 years old or younger with MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8-1.0mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
High Risk Patients
Patients 19 years old or older with marrow aplasia or MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.4mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
—
|
18 years
n=107 Participants
|
—
|
18 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
—
|
2 Participants
n=107 Participants
|
—
|
2 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
—
|
1 Participants
n=107 Participants
|
—
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
—
|
3 Participants
n=107 Participants
|
—
|
3 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
—
|
2 Participants
n=107 Participants
|
—
|
2 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
—
|
1 Participants
n=107 Participants
|
—
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
—
|
0 Participants
n=107 Participants
|
—
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
—
|
3 Participants
n=107 Participants
|
—
|
3 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: 5 yearsPopulation: Data were not collected. No participants received treatment and study terminated due to slow accrual
Primary non-engraftment is diagnosed when the patient fails to achieve an ANC ≥500/µl at any time in the first 28 days post-transplant. If (1) after achievement of an ANC ≥500/mm\^3, the ANC declines to \<500/mm\^3 for more than 3 consecutive days in the absence of relapse, or, (2) there is absence of donor cells in the marrow and/or blood as demonstrated by chimerism assay in the absence of relapse, a diagnosis of secondary graft failure is made. The patient is not evaluable for graft failure or rejection if recurrence of host MDS is detected concurrently.
Outcome measures
Outcome data not reported
Adverse Events
Good Risk Patients
Intermediate Risk Patients
High Risk Patients
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Dr. Farid Boulad, MD
Memorial Sloan Kettering Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place