Trial Outcomes & Findings for Relapsed Malignant Blood Cancer After Allogeneic Hematopoietic Stem Cell Transplantation (NCT NCT01326728)

Last Updated: 2018-06-06

Results Overview

Biological response to agents and or treatments that can lead to bone marrow suppression/ cytopenias and sometimes death.

Recruitment status

TERMINATED

Target enrollment

56 participants

Primary outcome timeframe

up to 100 days or more following transplant

Results posted on

2018-06-06

Participant Flow

Participant milestones

Participant milestones
Measure	Recipients Recipient-Subjects will have clinical and research evaluations at baseline and three and six months post-allotransplant, at six-month intervals through three years post-allotransplant, then yearly. Evaluation after relapse treatment response and for new protocol options is permitted.	Donors Donor-Subjects will be enrolled at the time of their clinical evaluation and cell collection for Recipient-Subject therapy. Return evaluation for additional clinical product collection is permitted.
Overall Study STARTED	47	9
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	47	9

Reasons for withdrawal

Reasons for withdrawal
Measure	Recipients Recipient-Subjects will have clinical and research evaluations at baseline and three and six months post-allotransplant, at six-month intervals through three years post-allotransplant, then yearly. Evaluation after relapse treatment response and for new protocol options is permitted.	Donors Donor-Subjects will be enrolled at the time of their clinical evaluation and cell collection for Recipient-Subject therapy. Return evaluation for additional clinical product collection is permitted.
Overall Study Death	13	0
Overall Study Early closure of study	34	9

Baseline Characteristics

Relapsed Malignant Blood Cancer After Allogeneic Hematopoietic Stem Cell Transplantation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Recipients n=47 Participants Recipient-Subjects will have clinical and research evaluations at baseline and three and six months post-allotransplant, at six-month intervals through three years post-allotransplant, then yearly. Evaluation after relapse treatment response and for new protocol options is permitted.	Donors n=9 Participants Donor-Subjects will be enrolled at the time of their clinical evaluation and cell collection for Recipient-Subject therapy. Return evaluation for additional clinical product collection is permitted.	Total n=56 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	39 Participants n=93 Participants	9 Participants n=4 Participants	48 Participants n=27 Participants
Age, Categorical >=65 years	8 Participants n=93 Participants	0 Participants n=4 Participants	8 Participants n=27 Participants
Age, Continuous	49.34 years STANDARD_DEVIATION 15.46 • n=93 Participants	50.33 years STANDARD_DEVIATION 14.54 • n=4 Participants	49.83 years STANDARD_DEVIATION 15.0 • n=27 Participants
Sex: Female, Male Female	20 Participants n=93 Participants	3 Participants n=4 Participants	23 Participants n=27 Participants
Sex: Female, Male Male	27 Participants n=93 Participants	6 Participants n=4 Participants	33 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=93 Participants	1 Participants n=4 Participants	5 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	40 Participants n=93 Participants	8 Participants n=4 Participants	48 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=93 Participants	0 Participants n=4 Participants	3 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Asian	2 Participants n=93 Participants	1 Participants n=4 Participants	3 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	4 Participants n=93 Participants	2 Participants n=4 Participants	6 Participants n=27 Participants
Race (NIH/OMB) White	38 Participants n=93 Participants	5 Participants n=4 Participants	43 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=93 Participants	1 Participants n=4 Participants	3 Participants n=27 Participants
Region of Enrollment United States	47 participants n=93 Participants	9 participants n=4 Participants	56 participants n=27 Participants
Count of Participants with Presence or Absence of GVHD Prior to Study Presence of GVHD	0 Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants
Count of Participants with Presence or Absence of GVHD Prior to Study Absence of GVHD	47 Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants
Count of Participants with Disease Present Prior to Stem Cell Transplant Conditioning	47 Participants n=93 Participants	NA Participants n=4 Participants	NA Participants n=27 Participants

PRIMARY outcome

Timeframe: up to 100 days or more following transplant

Population: Protocol specified data were not to be summarized or analyzed unless 30-40 total pts in both grps (no relapse, relapse) are available (to permit a given comparison to have approx. 80% power for a test with an effect size of 1.0 and 0.05 two-sided alpha level). PI of the study left the NIH before this number was reached and the study was terminated.

Biological response to agents and or treatments that can lead to bone marrow suppression/ cytopenias and sometimes death.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: first day of treatment to day 100 after allotransplant

Time to Progression is the time between the first day of treatment to day 100 after allotransplant.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: first day of treatment to the day of death

Overall Survival is the time between the first day of treatment to the day of death.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: up to 100 days or more following allotransplant

Number of days for a participant to reach engraftment.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 100 days or more post allotransplant

Acute GVHD is defined as GVHD that presents with signs and symptoms typical of acute GVHD but presenting after day 100 post allotransplant. Clinical Staging Grade 2 ((+) to (+++) Skin; (+) Liver; and (+) Gut) involvement, Grade 3 ((++) to (+++) Skin; (++ to +++) Liver; and (++ to +++) Gut) involvement, and Grade 4 ((++) to (++++) Skin; (++ to ++++) Liver; and (++ to ++++) Gut) involvement.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 100 days post allotransplant

Mild chronic GVHD involves only 1 or 2 organs or sites with no clinically significant functional impairment (max. score of 1 in all affected organs or sites). Moderate GVHD involves at least 1 organ or site with clinically significant but no major disability (max. score of 2 in any affected organ or site), or 3 or more organs or sites with no clinically significant functional impairment (max. score of 1 in all affected organs or sites), and a lung score of 1 will also be considered moderate chronic GVHD. Severe chronic GVHD indicates major disability caused by chronic GVHD (score of 3 in any organ or site). A lung score of 2 or greater will also be considered severe chronic GVHD.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: up to 100 days or more post allotransplant

Count of Participants with Infection After Allotransplant.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 5 years

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Recipients n=47 Participants Recipient-Subjects will have clinical and research evaluations at baseline and three and six months post-allotransplant, at six-month intervals through three years post-allotransplant, then yearly. Evaluation after relapse treatment response and for new protocol options is permitted.
Number of Participants With Serious and Non-Serious Adverse Events	13 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 100 days or more following allotransplant

Count of participants with clinical blood markers of inflammation. Normal to low blood markers indicate relapse. Falling blood marker levels indicate possible imminent relapse.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 100 days or more following allotransplant

Regimen-specific sensitivity are new or renewed sensitivity to therapies following allotransplant.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 100 days or more following allotransplant

GVL is a donor anti-tumor response following transplant.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: After Day 100 or Following Treatment of GVHD

Participants who were initially in remission.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: up to 100 days or more following allotransplant

Improved serologic responses after allotransplant.

Outcome measures

Outcome data not reported

Adverse Events

Recipients

Serious events: 13 serious events

Other events: 0 other events

Deaths: 13 deaths

Donors

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Recipients n=47 participants at risk Recipient-Subjects will have clinical and research evaluations at baseline and three and six months post-allotransplant, at six-month intervals through three years post-allotransplant, then yearly. Evaluation after relapse treatment response and for new protocol options is permitted.	Donors n=9 participants at risk Donor-Subjects will be enrolled at the time of their clinical evaluation and cell collection for Recipient-Subject therapy. Return evaluation for additional clinical product collection is permitted.
Respiratory, thoracic and mediastinal disorders Hypoxia	2.1% 1/47 • Number of events 1 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.	0.00% 0/9 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.
Infections and infestations Sepsis	2.1% 1/47 • Number of events 1 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.	0.00% 0/9 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Treatment Secondary Malignancy	23.4% 11/47 • Number of events 11 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.	0.00% 0/9 • 5 years This protocol only captured grade 5 adverse events. it is a natural history study. All of the adverse events were captured on the subject's treatment/main protocol.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Ronald Gress

National Cancer Institute

Phone: 301-496-1791

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place