Trial Outcomes & Findings for Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor (NCT NCT00582933)

NCT ID: NCT00582933

Last Updated: 2016-02-01

Results Overview

To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 96 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2016-02-01

Participant Flow

Participant milestones

Measure	Transplant Patients BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.
Overall Study STARTED	95
Overall Study COMPLETED	92
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Measure

Transplant Patients BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.

Overall Study Physician Decision

3

Baseline Characteristics

Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related or Unrelated Donor

Baseline characteristics by cohort

Measure	Transplant Patients n=92 Participants BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.
Age, Continuous	54.5 years n=5 Participants
Sex: Female, Male Female	44 Participants n=5 Participants
Sex: Female, Male Male	48 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD.

Outcome measures

Measure

Transplant Patients n=92 Participants BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.

Death From GVHD

4 participants

Adverse Events

Transplant Patients

Serious events: 16 serious events

Other events: 92 other events

Deaths: 0 deaths

Serious adverse events

Measure	Transplant Patients n=92 participants at risk BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.
Respiratory, thoracic and mediastinal disorders Adult respiratory disorder	4.3% 4/92 • Number of events 6
Metabolism and nutrition disorders Creatinine	1.1% 1/92 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	2.2% 2/92 • Number of events 2
General disorders Edema	1.1% 1/92 • Number of events 1
Blood and lymphatic system disorders Hemorrhage, other	1.1% 1/92 • Number of events 1
Respiratory, thoracic and mediastinal disorders Hypoxia	9.8% 9/92 • Number of events 9
Infections and infestations infection unknown neutropenia	1.1% 1/92 • Number of events 1
Infections and infestations infection without neutropenia	2.2% 2/92 • Number of events 2
Nervous system disorders Neuropathy-motor	2.2% 2/92 • Number of events 2
Eye disorders Ocular-Vision other	1.1% 1/92 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pneumonitis	2.2% 2/92 • Number of events 2
Renal and urinary disorders Renal failure	3.3% 3/92 • Number of events 3
Nervous system disorders Seizure	1.1% 1/92 • Number of events 1
Gastrointestinal disorders Stomatitis/BMT	1.1% 1/92 • Number of events 1

Other adverse events

Measure	Transplant Patients n=92 participants at risk BUSULFAN, MELPHALAN, FLUDARABINE, G-CSF: All research participants will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8- 1.0 mg/Kg/dose Q6H x 10 doses), melphalan (70 mg/m2/dose x 2 doses) and fludarabine (25 mg/m2/day x 5 doses). Doses of busulfan will be adjusted according to plasma levels. All research participants will also receive ATG (thymoglobulin®) prior to transplant to promote engraftment.All research participants will also receive G-CSF posttransplant to foster engraftment.
Metabolism and nutrition disorders Alkaline phosphatase	16.3% 15/92 • Number of events 15
Metabolism and nutrition disorders Bilirubin	18.5% 17/92 • Number of events 17
Metabolism and nutrition disorders Creatinine	15.2% 14/92 • Number of events 14
Blood and lymphatic system disorders Hemoglobin (Hgb)	96.7% 89/92 • Number of events 89
Metabolism and nutrition disorders Hyperglycemia	52.2% 48/92 • Number of events 48
Metabolism and nutrition disorders Hyperkalemia	19.6% 18/92 • Number of events 18
Metabolism and nutrition disorders Hypocalcemia	35.9% 33/92 • Number of events 33
Metabolism and nutrition disorders Hypokalemia	52.2% 48/92 • Number of events 48
Metabolism and nutrition disorders Hyponatremia	25.0% 23/92 • Number of events 23
Metabolism and nutrition disorders Hypophosphatemia	53.3% 49/92 • Number of events 49
Blood and lymphatic system disorders Leukocytes	100.0% 92/92 • Number of events 92
Blood and lymphatic system disorders Lymphopenia	100.0% 92/92 • Number of events 92
Blood and lymphatic system disorders Neutrophils	100.0% 92/92 • Number of events 92
Blood and lymphatic system disorders Platelets	100.0% 92/92 • Number of events 92
Blood and lymphatic system disorders Partial thromboplastin time (PTT)	19.6% 18/92 • Number of events 18
Blood and lymphatic system disorders SGOT (AST)	25.0% 23/92 • Number of events 23
Blood and lymphatic system disorders SGPT (ALT)	30.4% 28/92 • Number of events 28

Additional Information

Dr. Farid Boulad

Memorial Sloan Kettering Cancer Center

Phone: 212-639-2429

Email: bouladf@mskcc.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place