Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation (HSCT) Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit (NCT NCT01050946)

Results Overview

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

3 years

Results posted on

2015-11-20

Participant Flow

FDA approval was granted on 6/18/11 and went into effect on 7/18/11.Recruitment period started from the 7/18/11 until study closure on 3/28/13, All recruitment took place in the clinic setting. Only one patient was accrued.

Participant milestones

Participant milestones
Measure	Haploidentical/Cord Transplant Haploidentical/cord transplant with the preconditioning regimen at discretion of treating physician. Haploidentical/cord transplant : Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Hematopoietic Stem Cell Transplantation (HSCT) Using CD34 Selected Mismatched Related Donor and One Umbilical Cord Unit

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Haploidentical/Cord Transplant n=1 Participants Haploidentical/cord transplant with the precondition regimen at discretion of treating physician. Haploidentical/cord transplant : Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	61 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 years

Population: Only one patient enrolled on study. Patient died prior to time frame of 3 years. It is not possible to assess this outcome measure.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Patient did not live to 2 years, no relapse observed

NOT analyzed since there was only patient and no relapse was observed till patient passed away

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days

Population: Patient engrafted neutrophils and platelets but no statistical analysis possible

time to neutrophil recovery after transplant

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days

Population: No further analysis reported as only one patient enrolled

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days

Population: only one participant - no analysis done

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Population: only pt who was enrolled died of TRM

Outcome measures

Outcome measures
Measure	Haploidentical/Cord Transplant n=1 Participants Haploidentical/cord transplant with the precondition regimen at discretion of treating physician. Haploidentical/cord transplant: Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Transplant Related Mortality (TRM): TRM is Death Occurring in Patients in Continuous Complete Remission.	1 participants

SECONDARY outcome

Timeframe: 3 years

Outcome measures

Outcome measures
Measure	Haploidentical/Cord Transplant n=1 Participants Haploidentical/cord transplant with the precondition regimen at discretion of treating physician. Haploidentical/cord transplant: Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Disease-free Survival:Death or Relapse Will be Considered Events for This Endpoint.	0 participants

Adverse Events

Haploidentical/Cord Transplant

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Haploidentical/Cord Transplant n=1 participants at risk Haploidentical/cord transplant with the preconditioning regimen at discretion of treating physician. Haploidentical/cord transplant : Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Respiratory, thoracic and mediastinal disorders Acute Respiratory Distress Syndrome	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Renal and urinary disorders Acute Renal Injury	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Psychiatric disorders Confusion	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Infections and infestations Death	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.

Other adverse events

Other adverse events
Measure	Haploidentical/Cord Transplant n=1 participants at risk Haploidentical/cord transplant with the preconditioning regimen at discretion of treating physician. Haploidentical/cord transplant : Myeloablative preparative regimen of chemotherapy and radiation followed by mismatch related(haploidentical)donor and one unit umbilical cord blood transplantation. Conditioning Regimens Choice of regimen at the discretion of the treating physician 1. Fludarabine 30mg/m2(Days-7,-6,-5,-4,-3)-,Melphalan 70mg/m2(Day -3,-2), ATG 1.5mg/m2(Day-7,-5,-3,-1) 2. Fludarabine 50mg/m2(Day -6,-5,-4,-3,-2),Busulfan 3.2mg/kg(Day -5,-4,-3,-2),400cGY Total Body Irradiation(TBI)Day-1,ATG 1.5mg/kg(Day-7,-5,-3,-1) Day 0 -Haploidentical donor and one umbilical cord blood unit infusion
Respiratory, thoracic and mediastinal disorders Acute Respiratory Distress Syndrome	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Renal and urinary disorders Acute Renal Injury	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Psychiatric disorders Confusion	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.
Infections and infestations Death	100.0% 1/1 • Number of events 1 • From the time the patient signed consent until the patient's death.

Additional Information

Jeanne Palmer,MD

Medical College of Wisconsin

Phone: 414-805-6800

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place