Trial Outcomes & Findings for Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy (NCT NCT00303719)

Last Updated: 2020-05-12

Results Overview

Successful sustained engraftment is defined as primary neutrophil engraftment by day 42 and e90% donor cells at day 100, with or without DLI. Engraftment based on absolute neutrophil count of donor origin \> 0.5 x 10e9 /L for 3 days by day 42

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

342 participants

Primary outcome timeframe

Day 42 and Day 100

Results posted on

2020-05-12

Participant Flow

Participant milestones

Participant milestones
Measure	High Risk Patients Patients with refractory leukemia or MDS	Standard Risk Patients Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Overall Study STARTED	13	329
Overall Study COMPLETED	13	292
Overall Study NOT COMPLETED	0	37

Reasons for withdrawal

Reasons for withdrawal
Measure	High Risk Patients Patients with refractory leukemia or MDS	Standard Risk Patients Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Overall Study Not evaluable, prior therapies	0	30
Overall Study Lost to Follow-up	0	7

Baseline Characteristics

Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder	Total n=305 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=39 Participants	184 Participants n=41 Participants	188 Participants n=35 Participants
Age, Categorical >=65 years	9 Participants n=39 Participants	108 Participants n=41 Participants	117 Participants n=35 Participants
Sex: Female, Male Female	4 Participants n=39 Participants	112 Participants n=41 Participants	116 Participants n=35 Participants
Sex: Female, Male Male	9 Participants n=39 Participants	180 Participants n=41 Participants	189 Participants n=35 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=39 Participants	3 Participants n=41 Participants	4 Participants n=35 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=39 Participants	250 Participants n=41 Participants	260 Participants n=35 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=39 Participants	39 Participants n=41 Participants	41 Participants n=35 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=39 Participants	2 Participants n=41 Participants	2 Participants n=35 Participants
Race (NIH/OMB) Asian	0 Participants n=39 Participants	4 Participants n=41 Participants	4 Participants n=35 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=39 Participants	0 Participants n=41 Participants	0 Participants n=35 Participants
Race (NIH/OMB) Black or African American	0 Participants n=39 Participants	5 Participants n=41 Participants	5 Participants n=35 Participants
Race (NIH/OMB) White	12 Participants n=39 Participants	259 Participants n=41 Participants	271 Participants n=35 Participants
Race (NIH/OMB) More than one race	0 Participants n=39 Participants	6 Participants n=41 Participants	6 Participants n=35 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=39 Participants	16 Participants n=41 Participants	17 Participants n=35 Participants

PRIMARY outcome

Timeframe: Day 42 and Day 100

Outcome measures

Outcome measures
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Neutrophil and Donor Cell Engraftment	12 Participants	289 Participants

SECONDARY outcome

Timeframe: Day 100

Safety by development of severe adverse events within 100 days of transplant

Outcome measures

Outcome measures
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Serious Adverse Events	0 Participants	47 Participants

SECONDARY outcome

Timeframe: Day 100

\> 30% transplant related mortality at 100 days (non-relapse).

Outcome measures

Outcome measures
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Transplant Related Mortality	2 Participants	40 Participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Outcome measures
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Overall Survival	8 Participants	181 Participants

SECONDARY outcome

Timeframe: Day 100

Grade III-IV graft versus host disease

Outcome measures

Outcome measures
Measure	High Risk Patients n=13 Participants Patients with refractory leukemia or MDS	Standard Risk Patients n=292 Participants Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Acute Graft-Versus-Host Disease	2 Participants	79 Participants

Adverse Events

High Risk Patients

Serious events: 1 serious events

Other events: 12 other events

Deaths: 6 deaths

Standard Risk Patients

Serious events: 53 serious events

Other events: 272 other events

Deaths: 124 deaths

Serious adverse events

Serious adverse events
Measure	High Risk Patients n=13 participants at risk Patients with refractory leukemia or MDS	Standard Risk Patients n=292 participants at risk Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Immune system disorders Graft versus Host disease	0.00% 0/13 • 2 years	9.6% 28/292 • 2 years
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Relapse	7.7% 1/13 • 2 years	7.2% 21/292 • 2 years
Infections and infestations Infection	0.00% 0/13 • 2 years	1.4% 4/292 • 2 years
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Progressive disease	0.00% 0/13 • 2 years	0.00% 0/292 • 2 years

Other adverse events

Other adverse events
Measure	High Risk Patients n=13 participants at risk Patients with refractory leukemia or MDS	Standard Risk Patients n=292 participants at risk Patients with Disease criteria: Acute myelogenous leukemia , Acute lymphocytic leukemia, Chronic myelogenous leukemia , NHL, Hodgkins, chronic lymphocytic leukemia, multiple myeloma, Acquired bone marrow failure syndromes, Myelodysplastic syndrome, Renal cell cancer,Chronic myeloproliferative disorder
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome (ARDS)	0.00% 0/13 • 2 years	5.8% 17/292 • Number of events 18 • 2 years
Infections and infestations Bacterial infection	38.5% 5/13 • Number of events 15 • 2 years	43.8% 128/292 • Number of events 334 • 2 years
Gastrointestinal disorders Bleeding	15.4% 2/13 • Number of events 4 • 2 years	15.4% 45/292 • Number of events 56 • 2 years
Renal and urinary disorders Cystitis	7.7% 1/13 • Number of events 1 • 2 years	5.1% 15/292 • Number of events 18 • 2 years
Renal and urinary disorders Hemodialysis	15.4% 2/13 • Number of events 2 • 2 years	6.2% 18/292 • Number of events 21 • 2 years
Infections and infestations Infection (Etiology unknown)	30.8% 4/13 • Number of events 8 • 2 years	32.9% 96/292 • Number of events 219 • 2 years
Cardiac disorders Organ failure	15.4% 2/13 • Number of events 4 • 2 years	5.8% 17/292 • Number of events 21 • 2 years
Infections and infestations Fungal infection	30.8% 4/13 • Number of events 15 • 2 years	41.8% 122/292 • Number of events 269 • 2 years
Nervous system disorders Neuropathy	7.7% 1/13 • Number of events 1 • 2 years	14.7% 43/292 • Number of events 51 • 2 years
Nervous system disorders Neurotoxicity	0.00% 0/13 • 2 years	9.9% 29/292 • Number of events 37 • 2 years
Respiratory, thoracic and mediastinal disorders perihilar opacity	7.7% 1/13 • Number of events 1 • 2 years	4.8% 14/292 • Number of events 20 • 2 years
General disorders Other disorders	69.2% 9/13 • Number of events 25 • 2 years	61.3% 179/292 • Number of events 489 • 2 years
Infections and infestations Pnemonia	61.5% 8/13 • Number of events 15 • 2 years	51.0% 149/292 • Number of events 351 • 2 years
Ear and labyrinth disorders Ear disorder	15.4% 2/13 • Number of events 2 • 2 years	3.4% 10/292 • Number of events 10 • 2 years
Respiratory, thoracic and mediastinal disorders Increased oxygen requirement	7.7% 1/13 • Number of events 1 • 2 years	20.9% 61/292 • Number of events 73 • 2 years
Infections and infestations Viral infection	61.5% 8/13 • Number of events 15 • 2 years	49.0% 143/292 • Number of events 291 • 2 years
Hepatobiliary disorders Abnormal liver	0.00% 0/13 • 2 years	6.2% 18/292 • Number of events 20 • 2 years
Cardiac disorders Decrease cardiac function	0.00% 0/13 • 2 years	9.6% 28/292 • Number of events 33 • 2 years
Cardiac disorders pericardial effusion	15.4% 2/13 • Number of events 3 • 2 years	15.4% 45/292 • Number of events 51 • 2 years
Eye disorders Eye disorder	0.00% 0/13 • 2 years	10.6% 31/292 • Number of events 48 • 2 years
Respiratory, thoracic and mediastinal disorders Pulmonary hemorrhage	0.00% 0/13 • 2 years	8.6% 25/292 • Number of events 25 • 2 years

Additional Information

Erica Warlick, MD

Masonic Cancer Center, University of Minnesota

Phone: 612-625-8942

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place