Trial Outcomes & Findings for Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia (NCT NCT00590460)

Results Overview

Number of patients with engraftment of at least 65% of donor cells 100 days after transplantation

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

100 Days

Results posted on

2018-05-24

Participant Flow

Participant milestones

Participant milestones
Measure	Allo Stem Cell Transplant Allogeneic Stem Cell Transplant
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group1 n=5 Participants only one group
Age, Customized <=5 years	1 participants n=5 Participants
Age, Customized Between 5 and 9 years	2 participants n=5 Participants
Age, Customized Between 10 and 15 years	2 participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 Days

Number of patients with engraftment of at least 65% of donor cells 100 days after transplantation

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Number of Patients With Donor Engraftment	2 participants

SECONDARY outcome

Timeframe: 100 days

Graft failure is defined as engraftment of less than 65% of donor cells 100 days after transplantation.

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Number of Patients With Graft Failure	3 participants

SECONDARY outcome

Timeframe: 100 days

Number of patients with treated related death

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Patients With Treated Related Death	0 participants

SECONDARY outcome

Timeframe: 30 Days

Number of days to Absolute neutrophil count (ANC) of 500/mm3

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Days to Absolute Neutrophil Count (ANC) of 500/mm3	15 days Interval 14.0 to 16.0

SECONDARY outcome

Timeframe: 30 Days

Population: Participants achieved a platelet count of 20,000 / mm3 without transfusions.

Number of days to Platelet count of 20,000 / mm3 without transfusions

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=2 Participants Single Group: Allogeneic Stem Cell Transplant
Days to Platelet Count of 20,000/mm3 Without Transfusions	16 days Interval 14.0 to 18.0

SECONDARY outcome

Timeframe: 100 days

Number of patients with grade II - IV acute Graft versus Host Disease (GVHD)

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Patients With Grade II - IV Acute Graft Versus Host Disease (GVHD)	0 participants

SECONDARY outcome

Timeframe: 1 year

Number of patients alive at 1 year post allogeneic stem cell transplant

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Number of Patients Alive at 1 Year Post Transplant	5 participants

SECONDARY outcome

Timeframe: 365 days

Number of patients with limited chronic GVHD from day 100 to 365

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Patients With Limited Chronic GVHD From Day 100 to 365	0 participants

SECONDARY outcome

Timeframe: 365 days

Number of patients with extensive chronic GVHD from day 100 to 365.

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Patients With Extensive Chronic GVHD From Day 100 to 365	0 participants

SECONDARY outcome

Timeframe: 100 days

Number of patients with Grade III-IV acute GVHD

Outcome measures

Outcome measures
Measure	Allogeneic Stem Cell Transplant n=5 Participants Single Group: Allogeneic Stem Cell Transplant
Patients With Grade III - IV Acute GVHD	0 participants

Adverse Events

Group1

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Group1 n=5 participants at risk only one group
Nervous system disorders CNC hemorrhage	20.0% 1/5 • Number of events 1 • 1 year

Other adverse events

Other adverse events
Measure	Group1 n=5 participants at risk only one group
Cardiac disorders Hypertension	60.0% 3/5 • Number of events 3 • 1 year
Gastrointestinal disorders Anorexia	20.0% 1/5 • Number of events 1 • 1 year
Gastrointestinal disorders Diarrhea	20.0% 1/5 • Number of events 1 • 1 year
Hepatobiliary disorders AST	40.0% 2/5 • Number of events 4 • 1 year
Infections and infestations Picorna virus sinusitis	20.0% 1/5 • Number of events 1 • 1 year
Infections and infestations Herpes simplex type I	20.0% 1/5 • Number of events 1 • 1 year
Metabolism and nutrition disorders Hyperkalemia	40.0% 2/5 • Number of events 5 • 1 year
General disorders Jaw Pain	20.0% 1/5 • Number of events 1 • 1 year

Additional Information

Malcolm Brenner, MD

BAYLOR

Phone: 832-824-4663

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place