Trial Outcomes & Findings for Haploidentical Donor Hematopoietic Stem Cell Transplant in Treating Patients With Hematologic Malignancies (NCT NCT01871441)
NCT ID: NCT01871441
Last Updated: 2025-05-16
Results Overview
Disease free survival (DFS), defined as the time to death, relapse or disease progression.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
1 year
Results posted on
2025-05-16
Participant Flow
Participant milestones
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
Total-body irradiation: Undergo TBI
Donor lymphocytes infusion (DLI): Undergo DLI
Cyclophosphamide: Given IV
Allogeneic hematopoietic stem cell transplantation (HSCT): Undergo haploidentical allogeneic HSCT
Tacrolimus: Given IV
Mycophenolate mofetil: Given IV
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Haploidentical Donor Hematopoietic Stem Cell Transplant in Treating Patients With Hematologic Malignancies
Baseline characteristics by cohort
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
n=4 Participants
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
Total-body irradiation: Undergo TBI
Donor lymphocytes infusion (DLI): Undergo DLI
Cyclophosphamide: Given IV
Allogeneic hematopoietic stem cell transplantation (HSCT): Undergo haploidentical allogeneic HSCT
Tacrolimus: Given IV
Mycophenolate mofetil: Given IV
|
|---|---|
|
Age, Continuous
|
55 years
STANDARD_DEVIATION 11.35 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 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
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearDisease free survival (DFS), defined as the time to death, relapse or disease progression.
Outcome measures
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
n=4 Participants
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
|
|---|---|
|
Number of Participants With Disease-free Survival (DFS)
|
1 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearRelapse of Disease is defined as the return of a disease or the signs and symptoms of a disease after a period of improvement. Relapse is almost always associated with the immunological failure of the donor immune system to recognize and/or respond to reemergence of a tumor. The number of participants with relapse of disease will be collected.
Outcome measures
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
n=4 Participants
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
|
|---|---|
|
Number of Participants With Relapse of Disease
|
2 Participants
|
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: No data were collected or analyzed.
The rates of grade III-IV GVHD in female recipients with male donors will be computed with corresponding exact binomial 95% confidence intervals.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 1 yearPopulation: No data were collected or analyzed.
The difference in DFS in recipient-donor combinations in which there is at least 1 KIR ligand mismatch versus those without a KIR ligand mismatch will be tested using log-rank test.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (Haploidentical Allogeneic HSCT)
Serious events: 4 serious events
Other events: 4 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
n=4 participants at risk
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
Total-body irradiation: Undergo TBI
Donor lymphocytes infusion (DLI): Undergo DLI
Cyclophosphamide: Given IV
Allogeneic hematopoietic stem cell transplantation (HSCT): Undergo haploidentical allogeneic HSCT
Tacrolimus: Given IV
Mycophenolate mofetil: Given IV
|
|---|---|
|
Hepatobiliary disorders
increased LFTs
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Colitis - infectious diarrhea
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Immune system disorders
Fever
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Immune system disorders
CMV reactivation
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
UTI
|
25.0%
1/4 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Treatment (Haploidentical Allogeneic HSCT)
n=4 participants at risk
Patients undergo TBI BID on days -9 to -6, undergo DLI on day -6, and receive cyclophosphamide IV over 2 hours on days -3 and -2.
TRANSPLANT: Patients undergo haploidentical allogeneic hematopoietic stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV beginning on day -1 with taper beginning on day 42, and mycophenolate mofetil IV BID from day -1 to day 28.
Total-body irradiation: Undergo TBI
Donor lymphocytes infusion (DLI): Undergo DLI
Cyclophosphamide: Given IV
Allogeneic hematopoietic stem cell transplantation (HSCT): Undergo haploidentical allogeneic HSCT
Tacrolimus: Given IV
Mycophenolate mofetil: Given IV
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Abdominal pain
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Acid reflux
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Nervous system disorders
Anxiety
|
75.0%
3/4 • Number of events 3 • 1 year
|
|
Cardiac disorders
Arrhythmia
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Awakening to Urinate
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Eye disorders
Blurred vision
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Infections and infestations
C. Difficile
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Immune system disorders
CMV reactivation
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Reproductive system and breast disorders
Constipation
|
75.0%
3/4 • Number of events 3 • 1 year
|
|
General disorders
Dehydration
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Nervous system disorders
Depression
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
75.0%
3/4 • Number of events 5 • 1 year
|
|
General disorders
Dizziness
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Dry Hands
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
Dysuria
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
General disorders
Dysgeusia
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Electrolyte Abnormalities
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
Elevated transaminase
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Fatigue
|
75.0%
3/4 • Number of events 3 • 1 year
|
|
Infections and infestations
Fever
|
100.0%
4/4 • Number of events 8 • 1 year
|
|
General disorders
Fluid overload
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
GERD
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Eye disorders
Glaucoma
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Nervous system disorders
Hallucinations
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Hives - related to injection
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Hyperpigmentation
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Cardiac disorders
Hypertension
|
75.0%
3/4 • Number of events 3 • 1 year
|
|
Cardiac disorders
Hypotension
|
50.0%
2/4 • Number of events 3 • 1 year
|
|
Blood and lymphatic system disorders
Increased Creatinine
|
25.0%
1/4 • Number of events 2 • 1 year
|
|
Renal and urinary disorders
Increased LFTs
|
50.0%
2/4 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Indigestion
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Nervous system disorders
Insomnia
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
Iron overload
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Itching
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Knee pain
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Swelling
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Lightheadedness
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Mucositis
|
75.0%
3/4 • Number of events 3 • 1 year
|
|
Nervous system disorders
Numbness
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Parotiditis
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
Pericatheter Thrombus
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pruitis
|
50.0%
2/4 • Number of events 5 • 1 year
|
|
General disorders
Pronatriuretic Peptide elevated
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Rigors
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Runny nose
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Nervous system disorders
Short term memory loss
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Sweating
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Swollen hands
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
SOB
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Stomach cramping
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Cardiac disorders
Tachycardia
|
50.0%
2/4 • Number of events 3 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Tachypenea
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
General disorders
Trouble sleeping
|
50.0%
2/4 • Number of events 3 • 1 year
|
|
Nervous system disorders
Twitching
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
Urinary incontinence
|
25.0%
1/4 • Number of events 1 • 1 year
|
|
Eye disorders
Watery Eyes
|
25.0%
1/4 • Number of events 1 • 1 year
|
Additional Information
Dr. Neal Flomenberg
Sidney Kimmel Cancer Center at Thomas Jefferson University
Phone: 215 955-0182
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place