Trial Outcomes & Findings for Reduced Intensity Haploidentical Transplant for Hematological Malignancies (NCT NCT01162096)

NCT ID: NCT01162096

Last Updated: 2025-04-30

Results Overview

Number of patients alive at 6 months post-transplant

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 34 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2025-04-30

Participant Flow

Participant milestones

Measure	Transplantation
Overall Study STARTED	34
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced Intensity Haploidentical Transplant for Hematological Malignancies

Baseline characteristics by cohort

Measure	Transplantation n=34 Participants
Age, Continuous	59.23 years STANDARD_DEVIATION 15.57 • n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	14 Participants n=5 Participants
Age, Categorical >=65 years	20 Participants n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	21 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	6 Participants n=5 Participants
Race (NIH/OMB) White	27 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	33 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	34 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Number of patients alive at 6 months post-transplant

Outcome measures

Measure	Transplantation n=34 Participants
Overall Survival at 6 Months Post-transplant in Patients Receiving a Partially-matched Related Donor Allogeneic Transplant After Reduced-intensity Conditioning	22 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 32 patients are evaluable for engraftment

Outcome measures

Measure	Transplantation n=32 Participants
Number of Participants With Successful Engraftment	31 Participants

SECONDARY outcome

Timeframe: Up to 5 years

Outcome measures

Measure	Transplantation n=34 Participants
Immune Reconstitution CD3/4 count at day 28	35.945 cells/ul Interval 2.1 to 213.6
Immune Reconstitution CD3/4 count at day 90	104.540 cells/ul Interval 22.4 to 424.2
Immune Reconstitution CD3/8 count at day 28	26.50 cells/ul Interval 2.1 to 213.6
Immune Reconstitution CD3/8 count at day 90	119.2 cells/ul Interval 11.2 to 849.6

Adverse Events

Transplantation

Serious events: 14 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Transplantation n=34 participants at risk
General disorders Death - Multi-organ failure	8.8% 3/34 • Number of events 3
General disorders Death - Disease progression NOS	17.6% 6/34 • Number of events 6
General disorders Death - Death NOS	14.7% 5/34 • Number of events 5
Infections and infestations Opportunistic infection associated with >=Grade 2 Lymphopenia	26.5% 9/34 • Number of events 12
Infections and infestations Infection - Other	14.7% 5/34 • Number of events 5
Blood and lymphatic system disorders Blood/Bone Marrow - other	14.7% 5/34 • Number of events 5
Gastrointestinal disorders Diarrhea	5.9% 2/34 • Number of events 2
Cardiac disorders Cardiac General - other	5.9% 2/34 • Number of events 2
Cardiac disorders Hypotension	8.8% 3/34 • Number of events 3
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	5.9% 2/34 • Number of events 2
Immune system disorders Allergic reaction/ hypersensitivity	2.9% 1/34 • Number of events 1
Infections and infestations Febrile neutropenia	2.9% 1/34 • Number of events 1
Hepatobiliary disorders Cholecystitis	2.9% 1/34 • Number of events 1
General disorders Fatigue (asthenia, lethargy, malaise)	2.9% 1/34 • Number of events 1
Cardiac disorders Left ventricular diastolic dysfunction	2.9% 1/34 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	2.9% 1/34 • Number of events 1
Respiratory, thoracic and mediastinal disorders Adult Respiratory Distress Syndrome (ARDS)	2.9% 1/34 • Number of events 1
Gastrointestinal disorders Gastrointestinal - other	2.9% 1/34 • Number of events 1
Nervous system disorders Myelitis	2.9% 1/34 • Number of events 1
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - other	2.9% 1/34 • Number of events 1

Other adverse events

Adverse event data not reported

Additional Information

Neal Flomenberg, MD

Thomas Jefferson University

Phone: 215-955-4367

Email: neal.flomenberg@jefferson.edu

Results disclosure agreements

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