Trial Outcomes & Findings for Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies (NCT NCT01745913)

Last Updated: 2018-07-11

Results Overview

No patients completed this study and are therefore inevaluable. Subject data not evaluable for the outcome measure time frame. Subject data only evaluable up to month 3.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

2 participants

Primary outcome timeframe

estimation of 24 months to determine engraftment rates for all subjects

Results posted on

2018-07-11

Participant Flow

Participant milestones

Participant milestones
Measure	Haplo-Cord SCT The UCB unit must supply a minimum of 1.0 x107/kg pre-cryopreserved nucleated cell dose. The unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1 CliniMACS® CD34 Reagent System: If a subject is randomized to the haplo-cord transplant group, their family member will undergo a stem cell collection. The stem cells from the haplo-identical donor will be purified by a procedure called CD34 selection before they are given to the subject. A special device called the CliniMACS® CD34 Reagent System, which is not FDA approved, will be used for this purpose.	UCB SCT For the standard arm, UCB units will be selected using the Minnesota strategy and the strategy followed in a recent CTN study.17;19Each unit must supply a minimum of 1.5 x107/kg pre-cryopreserved nucleated cell dose. Subjects must have two partially HLA-matched UCB units. Each unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1 Fludarabine: Fludarabine: 30 mg/m2 /day intravenously x 5 days total dose 150 mg/m2. Fludarabine will be dosed according to actual body weight Melphalan: Melphalan: 70mg/m2/day intravenously x 2 days. Melphalan will be dosed according to actual body weight.
Overall Study STARTED	1	1
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Haplo-Cord SCT n=1 Participants The UCB unit must supply a minimum of 1.0 x107/kg pre-cryopreserved nucleated cell dose. The unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1.	UCB SCT n=1 Participants For the standard arm, UCB units will be selected using the Minnesota strategy and the strategy followed in a recent CTN study.17;19Each unit must supply a minimum of 1.5 x107/kg pre-cryopreserved nucleated cell dose. Subjects must have two partially HLA-matched UCB units. Each unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1.	Total n=2 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants

PRIMARY outcome

Timeframe: estimation of 24 months to determine engraftment rates for all subjects

Population: Data not collected. Patients died.

No patients completed this study and are therefore inevaluable. Subject data not evaluable for the outcome measure time frame. Subject data only evaluable up to month 3.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: estimation of 24 months to determine platelet recovery for all subjects

Population: Data not collected. Patients died.

No patients completed this study and are therefore inevaluable

Outcome measures

Outcome data not reported

Serious events: 0 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Haplo-Cord SCT n=1 participants at risk The UCB unit must supply a minimum of 1.0 x107/kg pre-cryopreserved nucleated cell dose. The unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1.	UCB SCT n=1 participants at risk For the standard arm, UCB units will be selected using the Minnesota strategy and the strategy followed in a recent CTN study.17;19Each unit must supply a minimum of 1.5 x107/kg pre-cryopreserved nucleated cell dose. Subjects must have two partially HLA-matched UCB units. Each unit must match at a minimum of 4 of 6 at HLA-A, -B, -DRB1 loci with the recipient. This may include 0-2 antigen mismatches at each A or B (at the antigen level) or DRB1 (at the allele level) loci. All typing will be done using molecular typing. Though molecular level typing will be available, a match is defined at intermediate resolution for HLA-A and -B and at high resolution for -DRB1.
General disorders chills	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Metabolism and nutrition disorders Hypomagnesemia	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
General disorders rigors	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders nausea	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Psychiatric disorders insomnia	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Musculoskeletal and connective tissue disorders generalized muscle weakness	— 0/0 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Nervous system disorders headache	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
General disorders fever	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders abdominal pain	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders abdominal cramping	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders Diarrhea	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
General disorders Fatigue	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
General disorders Decreased Appetite	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Skin and subcutaneous tissue disorders Papulopustular rash	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
General disorders Dizziness	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders Stomach Pain	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
General disorders lower left extremity Edema	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders Bloating	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Psychiatric disorders Mood Changes	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders Nausea	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
Skin and subcutaneous tissue disorders Night Sweats	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
Gastrointestinal disorders Vomiting	0.00% 0/1 • 3 months	100.0% 1/1 • Number of events 1 • 3 months
General disorders Nosebleed	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
Eye disorders Decrease in vision	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
General disorders Foot Pain	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
Gastrointestinal disorders Intermittent: Vomiting	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months
Skin and subcutaneous tissue disorders Pruritus	100.0% 1/1 • Number of events 1 • 3 months	0.00% 0/1 • 3 months

Additional Information

Koen van Besien, MD

Weill Cornell Medical College

Phone: 2127462646

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place