Trial Outcomes & Findings for Partially HLA Mismatched (Haploidentical) Allogeneic Bone Marrow Transplantation (NCT NCT01749293)

Results Overview

To estimate the number of participants that had engraftment rates and the number of participants that had full donor chimerism at Day 60 in patients undergoing an HLA haploidentical stem cell transplant with post transplant high dose cyclophosphamide.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Up to Day 60 post-transplant.

Results posted on

2020-02-25

Participant Flow

Participant milestones

Participant milestones
Measure	Haploidentical Transplant All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial will receive total body irradiation (2Gy fractionated) from Day -2 or -1. Cyclophosphamide: Subjects in this trial will receive Cyclophosphamide 50 mg/kg IV QD on Days 3 and 4 post-transplant.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Partially HLA Mismatched (Haploidentical) Allogeneic Bone Marrow Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Haploidentical Transplant n=3 Participants All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	41.8 Years STANDARD_DEVIATION 2 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Day 60 post-transplant.

To estimate the number of participants that had engraftment rates and the number of participants that had full donor chimerism at Day 60 in patients undergoing an HLA haploidentical stem cell transplant with post transplant high dose cyclophosphamide.

Outcome measures

Outcome measures
Measure	Haploidentical Transplant n=3 Participants All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial will receive total body irradiation (2Gy fractionated) from Day -2 or -1. Cyclophosphamide: Subjects in this trial will receive Cyclophosphamide 50 mg/kg IV QD on Days 3 and 4 post-transplant.
Number of Participants That Engrafted and the Number of Participants That Had Full Donor Chimerism at Day 60	0 Participants

SECONDARY outcome

Timeframe: Up to one year post-transplant.

To estimate the number of participants that had an overall survival (OS) rate

Outcome measures

Outcome measures
Measure	Haploidentical Transplant n=3 Participants All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial will receive total body irradiation (2Gy fractionated) from Day -2 or -1. Cyclophosphamide: Subjects in this trial will receive Cyclophosphamide 50 mg/kg IV QD on Days 3 and 4 post-transplant.
Number of Participants That Had an Overall Survival Rate	1 Participants

SECONDARY outcome

Timeframe: Up to 1 year post-transplant

To estimate the number of participants who had an event free survival rate

Outcome measures

Outcome measures
Measure	Haploidentical Transplant n=3 Participants All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial will receive total body irradiation (2Gy fractionated) from Day -2 or -1. Cyclophosphamide: Subjects in this trial will receive Cyclophosphamide 50 mg/kg IV QD on Days 3 and 4 post-transplant.
Number of Participants That Had an Event Free Survival Rate	0 Participants

Adverse Events

Haploidentical Transplant

Serious events: 1 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Haploidentical Transplant n=3 participants at risk All subjects will be dosed with pre-transplant Fludarabine (180mg/m2)and Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg. Subjects will then undergo total body irradiation 2Gy. Subjects will undergo haploidentical allogeneic bone marrow transplant, followed by Cyclophosphamide, Tacrolimus and MMF based GVHD prophylaxis. Fludarabine: Subjects in this trial will receive Fludarabine 30 mg/m2 IV QD, adjusted for CrCl from Days -8 through -3. Busulfan: Subjects in this trial will receive Busulfan total AUC 2400 μmol\min/L or 6.4mg/kg in 4 doses with seizure prophylaxis from Days -6 through -3. Total Body Irradiation: Subjects in this trial will receive total body irradiation (2Gy fractionated) from Day -2 or -1. Cyclophosphamide: Subjects in this trial will receive Cyclophosphamide 50 mg/kg IV QD on Days 3 and 4 post-transplant.
Cardiac disorders Serious	33.3% 1/3 • Number of events 1 • 1 year

Other adverse events

Adverse event data not reported

Additional Information

Dr. Pritesh Patel

University of Illinois

Phone: 312-996-5762

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place