Trial Outcomes & Findings for Study of Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Hematopoietic Malignancies Including Those That Are Challenging to Engraft (NCT NCT03187756)

Last Updated: 2020-03-27

Results Overview

Estimate the one year after transplantation event free survival (EFS) rate using a Kaplan-Meier curve with a 90% confidence interval. An event for EFS is defined as the first of any of the following failures: relapse or disease progression or death from any cause

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

One Year

Results posted on

2020-03-27

Participant Flow

Participant milestones

Participant milestones
Measure	Cyclophosphamide Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Overall Study STARTED	6
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Cyclophosphamide Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Overall Study Termination of study	4

Baseline Characteristics

Study of Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Hematopoietic Malignancies Including Those That Are Challenging to Engraft

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Age, Continuous	51 Years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 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) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: One Year

Population: 2 participants reached the 1 year time point but disease assessment was completed for only 1 subject (complete remission).

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=1 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Event Free Survival (EFS)	1 Participants

SECONDARY outcome

Timeframe: 1 year

Acute GVHD is graded by standard criteria, and all suspected cases of acute GVHD will be confirmed histologically by biopsy of an affected organ. The severity of acute GVHD is determined by an assessment of the degree of involvement of the skin, liver, and gastrointestinal tract. Grade I is characterized as mild disease (skin involvement alone), Grade II as moderate, Grade III as severe (involvement of any organ system), and Grade IV as life-threatening. The diagnosis of a chronic GVHD per NIH criteria requires a) at least 1 diagnostic manifestation or b) 1 distinctive manifestation confirmed by biopsy or testing of the same or other involved organ.

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Number of Participants With Chronic GVHD and Grades I-IV GVHD chronic GVHD	0 Participants
Number of Participants With Chronic GVHD and Grades I-IV GVHD acute grade IV GVHD	0 Participants
Number of Participants With Chronic GVHD and Grades I-IV GVHD acute grade III GVHD	0 Participants
Number of Participants With Chronic GVHD and Grades I-IV GVHD acute grade II GVHD	0 Participants
Number of Participants With Chronic GVHD and Grades I-IV GVHD acute grade I GVHD	2 Participants

SECONDARY outcome

Timeframe: 1 year

Population: While AE data was reported (see AE / SAE section), due to termination of the study, relationship of major AEs to transplant procedures was unable to be assessed. Therefore, this information cannot be reported.

Summarize major toxicities and complications associated with the transplantation procedure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year

Estimate the cumulative incidence of systemic steroid initiation, by 1 year after HSCT. This is will be reported as number of participants who started steroids over the course of the study.

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Cumulative Incidences of Systemic Steroid Initiation	6 Participants

SECONDARY outcome

Timeframe: 1 year

Graft failure and death, or graft failure, death and treatment of relapse/progressions. This will be reported as the number of participants with graft failures.

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Graft Failure Frequency	0 Participants

SECONDARY outcome

Timeframe: 1 year

Neutrophil recovery is defined as post-nadir ANC greater than or equal to 500/mm3 for three consecutive measurements on different days. The first of the three days will be designated as the day of neutrophil recovery.

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Time to Neutrophil Recovery	24.17 days Interval 17.0 to 31.0

SECONDARY outcome

Timeframe: 1 year

Platelet recovery is defined as sustained platelet count greater than or equal to 20,000/mm3 or greater than or equal to 50,000/mm3 with no platelet transfusions in the preceding seven days. The first of three consecutive measurements on different days will be designated as the day of initial platelet recovery.

Outcome measures

Outcome measures
Measure	Cyclophosphamide n=6 Participants Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Time to Platelet Recovery	9.4 days Interval 1.0 to 24.0

Adverse Events

Cyclophosphamide

Serious events: 6 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cyclophosphamide n=6 participants at risk Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Infections and infestations Cytomegalovirus	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Fever	66.7% 4/6 • Number of events 5 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Infections and infestations Enterocolitis Infectious	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Abdominal Pain	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Musculoskeletal and connective tissue disorders Generalized Muscle Weakness	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Gastritis	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Renal and urinary disorders Hematuria	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.

Other adverse events

Other adverse events
Measure	Cyclophosphamide n=6 participants at risk Cy 50 mg/kg IV, over approximately 1-2 hours (depending on volume), is given on Day 3 posttransplantation (ideally between 60 and 72 hours after marrow infusion) and on Day 4 (approximately 24 hours after Day 3 Cy).
Infections and infestations Adenovirus	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Metabolism and nutrition disorders Anorexia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Anemia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Back Pain	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Metabolism and nutrition disorders Vomiting	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Skin and subcutaneous tissue disorders Uticaria	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Renal and urinary disorders Urinary Retention	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Tract Infection (URI)	33.3% 2/6 • Number of events 2 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Upper GI Stage 1 Acute GVHD	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Hepatobiliary disorders Transaminitis MILD	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Cardiac disorders Tachycardia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders superficial thrombophlebitis (Left Arm)	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Sore Throat	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Skin and subcutaneous tissue disorders Skin Stage 1 Acute GVHD	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Infections and infestations Rhinovirus	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Rectal Mucositis	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Radiation Enteritis	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Skin and subcutaneous tissue disorders Pruritis	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Infections and infestations Parainfluenza	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Eye disorders Photophobia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Skin and subcutaneous tissue disorders Palmar Plantar Erythrodysesthesia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Pain	33.3% 2/6 • Number of events 2 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Infections and infestations Norovirus	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Non-Cardiac Chest Pain	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Nervous system disorders Neoropathy	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Neck Pain	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders Nausea	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Nasal Congestion	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Musculoskeletal and connective tissue disorders Myalgias	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Musculoskeletal and connective tissue disorders Muscle Weakness Lower Limb	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Skin and subcutaneous tissue disorders Maculopapular Rash	33.3% 2/6 • Number of events 2 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Leukopenia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Intermittent Tremor	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Hepatobiliary disorders Intermittent Increased Serum Creatinine	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Hypomagnesemia	66.7% 4/6 • Number of events 4 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Hypokalemia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Hypertension	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Blood and lymphatic system disorders Hyperglycemia	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
General disorders Headache	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.
Gastrointestinal disorders GERD	16.7% 1/6 • Number of events 1 • The time frame for adverse event data collection was 13 months. All adverse events occurring during the study period were reported. The clinical course of each event was followed up until resolution, stabilization, or until it was determined that the study treatment or participation was not the cause.

Additional Information

David Kaminetzky, MD

Perlmutter Cancer Center, NYU Langone Health

Phone: 212-731-5855

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place