Trial Outcomes & Findings for Stem Cell Transplantation for Fanconi Anemia (NCT NCT00167206)

Results Overview

Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10\^9/Liter.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

16 participants

Primary outcome timeframe

Day 42 after hematopoietic cell transplant

Results posted on

2019-08-26

Participant Flow

Participant milestones

Participant milestones
Measure	Intent-To-Treat All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Overall Study STARTED	16
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplantation for Fanconi Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Age, Categorical <=18 years	16 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	9.8 years STANDARD_DEVIATION 4.5 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Region of Enrollment United States	16 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 42 after hematopoietic cell transplant

Calculated from Day 1 of hematopoietic cell transplant to Day 42 post-transplant. Hematopoietic recovery and engraftment is defined as the first of three consecutive days the patient's absolute neutrophil count is greater than or equal to 0.5X10\^9/Liter.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment	15 Participants

SECONDARY outcome

Timeframe: Day 100 after hematopoietic cell transplant

Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. A complication after Bone Marrow Transplant in which the transplanted stem cells do not grow in the recipient's bone marrow and thus do not produce new blood cells.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients Who Exhibited Secondary Graft Failure	1 Participants

SECONDARY outcome

Timeframe: Day 100 after hematopoietic cell transplant

Calculated from Day 1 of hematopoietic cell transplant to Day 100 after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients With Acute Graft Versus-Host Disease (aGVHD)	8 Participants

SECONDARY outcome

Timeframe: 1 year after hematopoietic cell transplant

Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. GVHD is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients With Chronic Graft Versus-Host Disease (GVHD)	2 Participants

SECONDARY outcome

Timeframe: 1 year after hematopoietic cell transplant

Calculated from Day 1 of hematopoietic cell transplant to 1 year after transplant. Regimen-related toxicity involves harmful effects in an organism through exposure to the treatment given.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients Who Exhibited Regimen-related Toxicity (RRT)	5 Participants

SECONDARY outcome

Timeframe: 1 year post-transplant.

Calculated mean value of patient CD4 values collected at intervals from Day 30 through 1 year post-transplant.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Immune Reconstitution - Mean Value (1 Year)	860 Number of CD4 cells per microliter Standard Deviation 870

SECONDARY outcome

Timeframe: at 2 years after transplant

Calculated mean value of patient CD4 values collected at intervals from Day 30 through 2 years post-transplant.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Immune Reconstitution - Mean Value (2 Years)	1100 Number of CD4 cells per microliter Standard Deviation 510

SECONDARY outcome

Timeframe: 1 year after transplant

Calculated from Day 1 of hematopoietic cell transplant to 1 year post-transplant.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients Alive at 1 Year	11 Participants

SECONDARY outcome

Timeframe: 2 years after transplant

Calculated from Day 1 of hematopoietic cell transplant to 2 years post-transplant.

Outcome measures

Outcome measures
Measure	Intent-To-Treat n=16 Participants All patients with Fanconi anemia who received thymic shielding during total body irradiation (450 cGy).
Number of Patients Alive at 2 Years	10 Participants

Adverse Events

Intent-To-Treat

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Margaret MacMillan, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2778

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place