Trial Outcomes & Findings for A Two-Step Approach to Bone Marrow Transplant Using Cells From A Partially-Matched Relative (NCT NCT00429143)

NCT ID: NCT00429143

Last Updated: 2025-05-04

Results Overview

To determine overall survival at 6 months post-transplant.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 27 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2025-05-04

Participant Flow

Patients presenting to Thomas Jefferson University with hematological malignancies requiring hematopoeitic stem cell transplantation without matched related donors. Opened January, 2006 through August, 2009

Participant milestones

Measure	Haploidentical Allogeneic Transplantation Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Study STARTED	27
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Two-Step Approach to Bone Marrow Transplant Using Cells From A Partially-Matched Relative

Baseline characteristics by cohort

Measure	Haploidentical Allogeneic Transplantation n=27 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	24 Participants n=5 Participants
Age, Categorical >=65 years	3 Participants n=5 Participants
Age, Continuous	50 years STANDARD_DEVIATION 12.9 • n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Region of Enrollment United States	27 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Population: 27 Patients undergoing haploidentical transplant at Thomas Jefferson University

To determine overall survival at 6 months post-transplant.

Outcome measures

Measure	Haploidentical Allogeneic Transplantation n=27 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Overall Survival of Participants	13 participants

PRIMARY outcome

Timeframe: 6 months

Population: Two patients died prior to expected day of engraftment

To determine the optimal dose of CD3+ donor lymphocytes required for consistent engraftment without the development of grade III/IV GVHD.

Measured as CD3+ donor lymphocytes given as n x 10^8/kg.

"n" was found to be 2 and was found to be the optimal dose and was the only dose given.

Outcome measures

Measure	Haploidentical Allogeneic Transplantation n=25 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD	2 lymphocytes x 10^8/kg

SECONDARY outcome

Timeframe: 6 months

Population: Two patients died prior to expected engraftment day

To assess hematopoietic engraftment rates.

Outcome measures

Measure	Haploidentical Allogeneic Transplantation n=25 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Engraftment Rates	23 participants

SECONDARY outcome

Timeframe: 6 months

Population: Two patients did not engraft, two patients died prior to expected day of engraftment

To assess the pace of lymphoid recovery in this patient population.

Outcome measures

Measure	Haploidentical Allogeneic Transplantation n=23 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Lymphoid Recovery	23 participants

SECONDARY outcome

Timeframe: 6 months

Population: Two patients died prior to expected engraftment. Two patients who rejected were retransplanted and were evaluable for GVHD.

To determine the incidence and severity of GVHD in these patients using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.'

Severity was graded using CTCAE 3.0 (1=mild, 2=moderate, 3=severe, 4=life threatening/disabling, 5=death)

Outcome measures

Measure	Haploidentical Allogeneic Transplantation n=25 Participants Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Incidence of Grades III-IV GVHD	2 participants

Adverse Events

Haploidentical Allogeneic Transplantation

Serious events: 23 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Measure	Haploidentical Allogeneic Transplantation n=27 participants at risk Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Hepatobiliary disorders Liver dysfunction/failure	3.7% 1/27 • Number of events 1 • 6 Months
General disorders Death - Multi-organ failure	11.1% 3/27 • Number of events 3 • 6 Months
Infections and infestations Opportunistic infection associated with >=Grade 2 Lymphopenia	29.6% 8/27 • Number of events 9 • 6 Months
Immune system disorders Cytokine release syndrome/acute infusion reaction	3.7% 1/27 • Number of events 1 • 6 Months
General disorders Death - Disease progression NOS	18.5% 5/27 • Number of events 5 • 6 Months
Infections and infestations Infection - Other	14.8% 4/27 • Number of events 4 • 6 Months
Gastrointestinal disorders Diarrhea	7.4% 2/27 • Number of events 2 • 6 Months
Infections and infestations Infection with normal ANC or Grade 1 or 2 neutrophils	7.4% 2/27 • Number of events 2 • 6 Months
Gastrointestinal disorders Nausea	3.7% 1/27 • Number of events 1 • 6 Months
Vascular disorders Thrombosis/embolism	3.7% 1/27 • Number of events 1 • 6 Months
Respiratory, thoracic and mediastinal disorders Pneumonitis/pulmonary infiltrates	3.7% 1/27 • Number of events 1 • 6 Months
Respiratory, thoracic and mediastinal disorders Hypoxia	3.7% 1/27 • Number of events 1 • 6 Months
Renal and urinary disorders Renal/Genitourinary - Other	3.7% 1/27 • Number of events 1 • 6 Months
General disorders Constitutional Symptoms - Other	3.7% 1/27 • Number of events 1 • 6 Months
General disorders Death - Death NOS	7.4% 2/27 • Number of events 2 • 6 Months

Other adverse events

Measure	Haploidentical Allogeneic Transplantation n=27 participants at risk Patients undergoing hematopoietic stem cell transplant from a partially matched related donor
Gastrointestinal disorders Mucositis/stomatitis	85.2% 23/27 • Number of events 23 • 6 Months
Renal and urinary disorders Renal/Genitourinary - Other	7.4% 2/27 • Number of events 2 • 6 Months
Metabolism and nutrition disorders Bilirubin	11.1% 3/27 • Number of events 3 • 6 Months

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