Trial Outcomes & Findings for GVHD Prophylaxis With Post Transplant Cyclophosphamide for Patients With Renal Insufficiency Undergoing a Conventional 8/8 HLA-matched Related or Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplant (NCT NCT02360111)

NCT ID: NCT02360111

Last Updated: 2019-07-24

Results Overview

Chronic GVHD will be diagnosed and graded according to the (NIH criteria) treated with standard or experimental immunosuppressive therapy.

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 3 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2019-07-24

Participant Flow

Participant milestones

Measure	Post Transplant Cyclophosphamide Melphalan 70 mg/m 2/d will be administered intravenously on d-6 and -5 Fludarabine 25 mg/m 2/d will be administered intravenously on d-6 thru -2 Day -1 will be a day or rest Cyclophosphamide and mesna will be given on d+3 and +4 Siro +/- MMF will be started in those patients who are to receive it on d+5. Neupogen will begin d+7. Cyclophosphamide
Overall Study STARTED	3
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Post Transplant Cyclophosphamide Melphalan 70 mg/m 2/d will be administered intravenously on d-6 and -5 Fludarabine 25 mg/m 2/d will be administered intravenously on d-6 thru -2 Day -1 will be a day or rest Cyclophosphamide and mesna will be given on d+3 and +4 Siro +/- MMF will be started in those patients who are to receive it on d+5. Neupogen will begin d+7. Cyclophosphamide
Overall Study Death	2

Baseline Characteristics

GVHD Prophylaxis With Post Transplant Cyclophosphamide for Patients With Renal Insufficiency Undergoing a Conventional 8/8 HLA-matched Related or Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplant

Baseline characteristics by cohort

Measure	Post Transplant Cyclophosphamide n=3 Participants Melphalan 70 mg/m 2/d will be administered intravenously on d-6 and -5 Fludarabine 25 mg/m 2/d will be administered intravenously on d-6 thru -2 Day -1 will be a day or rest Cyclophosphamide and mesna will be given on d+3 and +4 Siro +/- MMF will be started in those patients who are to receive it on d+5. Neupogen will begin d+7. Cyclophosphamide
Age, Continuous	70 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Protocol terminated prematurely due to low accrual

Chronic GVHD will be diagnosed and graded according to the (NIH criteria) treated with standard or experimental immunosuppressive therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Protocol terminated prematurely due to low accrual

DFS is defined as the minimum interval of time to relapse/recurrence, to death or to the last follow-up, from the time of transplant

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Protocol terminated prematurely due to low accrual

Overall survival is defined as time from transplant to death or last follow-up.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Protocol terminated prematurely due to low accrual

Renal insufficiency is defined as a calculated eGFR <60 ml/min/1.73m2. Those with a eGFR < 30 ml/min/1.73m2 will be considered ineligible.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Population: Protocol terminated prematurely due to low accrual

will be evaluated according to the criteria established by BMT CTN , and will be correlated with the level of immune recovery.

Outcome measures

Outcome data not reported

Adverse Events

Post Transplant Cyclophosphamide

Serious events: 3 serious events

Other events: 3 other events

Deaths: 2 deaths

Serious adverse events

Measure	Post Transplant Cyclophosphamide n=3 participants at risk Melphalan 70 mg/m 2/d will be administered intravenously on d-6 and -5 Fludarabine 25 mg/m 2/d will be administered intravenously on d-6 thru -2 Day -1 will be a day or rest Cyclophosphamide and mesna will be given on d+3 and +4 Siro +/- MMF will be started in those patients who are to receive it on d+5. Neupogen will begin d+7. Cyclophosphamide
Renal and urinary disorders Acute kidney injury	33.3% 1/3 • 24 months
Psychiatric disorders Delirium	33.3% 1/3 • 24 months
Gastrointestinal disorders Diarrhea	33.3% 1/3 • 24 months
General disorders Edema limbs	33.3% 1/3 • 24 months
Nervous system disorders Encephalopathy	33.3% 1/3 • 24 months
Infections and infestations Lung infection	66.7% 2/3 • 24 months
Cardiac disorders Pericardial effusion	33.3% 1/3 • 24 months
Respiratory, thoracic and mediastinal disorders Pleural effusion	33.3% 1/3 • 24 months
Skin and subcutaneous tissue disorders Purpura	33.3% 1/3 • 24 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	33.3% 1/3 • 24 months
Infections and infestations Salivary gland infection	33.3% 1/3 • 24 months
Infections and infestations Sepsis	100.0% 3/3 • 24 months
Renal and urinary disorders Urinary retention	33.3% 1/3 • 24 months

Other adverse events

Measure	Post Transplant Cyclophosphamide n=3 participants at risk Melphalan 70 mg/m 2/d will be administered intravenously on d-6 and -5 Fludarabine 25 mg/m 2/d will be administered intravenously on d-6 thru -2 Day -1 will be a day or rest Cyclophosphamide and mesna will be given on d+3 and +4 Siro +/- MMF will be started in those patients who are to receive it on d+5. Neupogen will begin d+7. Cyclophosphamide
Blood and lymphatic system disorders Anemia	100.0% 3/3 • 24 months
Investigations Lymphocyte count decreased	100.0% 3/3 • 24 months
Investigations Neutrophil count decreased	100.0% 3/3 • 24 months
Investigations Platelet count decreased	100.0% 3/3 • 24 months
Investigations White blood cell decreased	100.0% 3/3 • 24 months
Metabolism and nutrition disorders Hyperkalemia	66.7% 2/3 • 24 months
Metabolism and nutrition disorders Hypokalemia	66.7% 2/3 • 24 months
Renal and urinary disorders Acute kidney injury	33.3% 1/3 • 24 months
Investigations Aspartate aminotransferase increased	33.3% 1/3 • 24 months
Investigations Blood bilirubin increased	33.3% 1/3 • 24 months
Investigations Creatinine increased	33.3% 1/3 • 24 months
Psychiatric disorders Delirium	33.3% 1/3 • 24 months
Gastrointestinal disorders Diarrhea	33.3% 1/3 • 24 months
General disorders Edema limbs	33.3% 1/3 • 24 months
Nervous system disorders Encephalopathy	33.3% 1/3 • 24 months
Metabolism and nutrition disorders Hyperglycemia	33.3% 1/3 • 24 months
Metabolism and nutrition disorders Hypocalcemia	33.3% 1/3 • 24 months
Metabolism and nutrition disorders Hyponatremia	33.3% 1/3 • 24 months
Metabolism and nutrition disorders Hypophosphatemia	33.3% 1/3 • 24 months
Infections and infestations Lung infection	33.3% 1/3 • 24 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	33.3% 1/3 • 24 months
Infections and infestations Sepsis	33.3% 1/3 • 24 months
Nervous system disorders Syncope	33.3% 1/3 • 24 months
Renal and urinary disorders Urinary retention	33.3% 1/3 • 24 months

Additional Information

Dr. Ann Jakubowski, Ph.D., MD

Memorial Sloan Kettering Cancer Center

Phone: 212-639-5013

Email: jakubowa@mskcc.org

Results disclosure agreements

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