Trial Outcomes & Findings for A Two-Step Approach to Bone Marrow Transplant Using Cells From Two Partially-Matched Relatives (NCT NCT01532635)
NCT ID: NCT01532635
Last Updated: 2025-05-04
Results Overview
To assess one year relapse-free survival (RFS) in patients undergoing HSCT (hematopoietic stem cell transplantation) using the TJU 2 step-approach with two donors. Survival will be estimated by the Kaplan-Meier method. All estimates of rates will be presented with corresponding confidence intervals. For 1 year RFS rates, the method of Atkinson and Brown will be used to allow for the two-stage design; otherwise the method of Conover.
TERMINATED
PHASE2
4 participants
1 year
2025-05-04
Participant Flow
Participant milestones
| Measure |
Allogeneic HSCT Using Two Related Donors
CONDITIONING: 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.
TRANSPLANTATION: Patients undergo CD34+ selected allogeneic HSCT on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and mycophenolate mofetil IV or PO BID on days -1 to 28.
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
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 Two Partially-Matched Relatives
Baseline characteristics by cohort
| Measure |
Allogeneic HSCT Using Two Related Donors
n=4 Participants
CONDITIONING: 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.
TRANSPLANTATION: Patients undergo CD34+ selected allogeneic HSCT on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and mycophenolate mofetil IV or PO BID on days -1 to 28.
|
|---|---|
|
Age, Continuous
|
50.35 years
STANDARD_DEVIATION 20.64 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 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
|
4 Participants
n=5 Participants
|
|
Ethnicity (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 yearTo assess one year relapse-free survival (RFS) in patients undergoing HSCT (hematopoietic stem cell transplantation) using the TJU 2 step-approach with two donors. Survival will be estimated by the Kaplan-Meier method. All estimates of rates will be presented with corresponding confidence intervals. For 1 year RFS rates, the method of Atkinson and Brown will be used to allow for the two-stage design; otherwise the method of Conover.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearTo assess chimerism to ascertain whether one donor is emerging as dominant at regular intervals beginning at the time of engraftment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearIf dominance is observed, to compare the 2 donors with regard to degree of HLA mismatch, KIR types, CD 34+ cell doses, infusion order, donor age, and donor alloreactivity points in an effort to identify potential biologic factors that predict for dominance. To determine if trends toward dominance occur in T cell, NK cell, or other cellular subsets prior to emerging in the graft as a whole.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearTo assess if establishment of a dominant donor versus persistent chimerism of both donors is associated with a lower relapse rate.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearTo assess the consistency and pace of engraftment of both donors.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearAssess T and B cell Reconstitution
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 yearAssessment of regimen related toxicity, GVHD incidence and severity, and overall survival.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2-6 days prior to transplantAssessment of the tolerance of the period of fever, diarrhea, and rash after the introduction of second donor and qualitatively compare it to prior patient groups or concurrent patient groups
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 1 year post transplantTo test for loss of one or both HLA haplotypes in patients who relapse post-transplant and examine the relapse in the context of the characteristics of the 2 donors
Outcome measures
Outcome data not reported
Adverse Events
Allogeneic HSCT Using Two Related Donors
Serious adverse events
| Measure |
Allogeneic HSCT Using Two Related Donors
n=4 participants at risk
CONDITIONING: 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.
TRANSPLANTATION: Patients undergo CD34+ selected allogeneic HSCT on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and mycophenolate mofetil IV or PO BID on days -1 to 28.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Fevers
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Hyperbilirubinemia
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypoxia
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Left ventricular systolic dysfunction
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Pericardial effusion
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Dehydration
|
25.0%
1/4 • Number of events 1
|
Other adverse events
| Measure |
Allogeneic HSCT Using Two Related Donors
n=4 participants at risk
CONDITIONING: 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.
TRANSPLANTATION: Patients undergo CD34+ selected allogeneic HSCT on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and mycophenolate mofetil IV or PO BID on days -1 to 28.
|
|---|---|
|
General disorders
Abdominal pain
|
50.0%
2/4 • Number of events 4
|
|
Psychiatric disorders
Anxiety
|
50.0%
2/4 • Number of events 2
|
|
General disorders
Arm pain
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Atrial fibrillation
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Back pain
|
50.0%
2/4 • Number of events 2
|
|
Blood and lymphatic system disorders
Bacteremia
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Balance problems
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchitis
|
25.0%
1/4 • Number of events 1
|
|
Eye disorders
Change in vision
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Chest pain
|
75.0%
3/4 • Number of events 4
|
|
General disorders
Chest tightness
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Chills
|
50.0%
2/4 • Number of events 2
|
|
Gastrointestinal disorders
Clostridium difficile
|
25.0%
1/4 • Number of events 1
|
|
Immune system disorders
CMV reactivation
|
50.0%
2/4 • Number of events 2
|
|
Gastrointestinal disorders
Constipation
|
50.0%
2/4 • Number of events 4
|
|
Renal and urinary disorders
Decreased urine output
|
50.0%
2/4 • Number of events 4
|
|
General disorders
Dehydration
|
25.0%
1/4 • Number of events 1
|
|
Psychiatric disorders
Depression
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
4/4 • Number of events 9
|
|
General disorders
Dizziness
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Dry eyes
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Dyspepsea
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
50.0%
2/4 • Number of events 2
|
|
Renal and urinary disorders
Dysuria
|
50.0%
2/4 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
Edema
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Electrolyte imbalance
|
75.0%
3/4 • Number of events 3
|
|
General disorders
Fatigue
|
50.0%
2/4 • Number of events 2
|
|
General disorders
Feet pain
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Fevers
|
100.0%
4/4 • Number of events 11
|
|
Blood and lymphatic system disorders
Fluid overload
|
50.0%
2/4 • Number of events 3
|
|
General disorders
Generalized pain
|
50.0%
2/4 • Number of events 2
|
|
General disorders
Generalized weakness
|
25.0%
1/4 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
GVHD rash
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Hand pain
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Headache
|
75.0%
3/4 • Number of events 3
|
|
Gastrointestinal disorders
Heartburn
|
50.0%
2/4 • Number of events 2
|
|
Renal and urinary disorders
Hematuria
|
50.0%
2/4 • Number of events 2
|
|
Blood and lymphatic system disorders
HHV-6 reactivation
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Hiccups
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Hyperbilirubinemia
|
100.0%
4/4 • Number of events 6
|
|
Blood and lymphatic system disorders
Hypertension
|
50.0%
2/4 • Number of events 3
|
|
Blood and lymphatic system disorders
Hypokalemia
|
25.0%
1/4 • Number of events 2
|
|
Blood and lymphatic system disorders
Hyponatremia
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypotension
|
75.0%
3/4 • Number of events 5
|
|
General disorders
Hypothermia
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypoxia
|
50.0%
2/4 • Number of events 3
|
|
General disorders
Increased alkaline phosphatase
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Increased creatinine
|
75.0%
3/4 • Number of events 4
|
|
Hepatobiliary disorders
Increased LFTs
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Knee pain
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Leg pain
|
25.0%
1/4 • Number of events 1
|
|
Metabolism and nutrition disorders
Malnutrition
|
25.0%
1/4 • Number of events 1
|
|
Psychiatric disorders
Mental status change
|
25.0%
1/4 • Number of events 1
|
|
Gastrointestinal disorders
Mucositis
|
100.0%
4/4 • Number of events 4
|
|
General disorders
Neck pain
|
25.0%
1/4 • Number of events 1
|
|
Renal and urinary disorders
Nocturia
|
50.0%
2/4 • Number of events 2
|
|
Blood and lymphatic system disorders
Orthostatic hypotension
|
50.0%
2/4 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Parotitis
|
25.0%
1/4 • Number of events 1
|
|
Blood and lymphatic system disorders
Platelet transfusion reaction
|
25.0%
1/4 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
50.0%
2/4 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
25.0%
1/4 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
100.0%
4/4 • Number of events 9
|
|
General disorders
Rectal pain
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Rigors
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Shoulder pain
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Swollen ankles
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Tachycardia
|
50.0%
2/4 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnea
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Toenail bleeding
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Trouble sleeping
|
50.0%
2/4 • Number of events 2
|
|
General disorders
Upper extremity swelling
|
25.0%
1/4 • Number of events 1
|
|
Renal and urinary disorders
Urinary retention
|
50.0%
2/4 • Number of events 2
|
|
General disorders
Vaginal discomfort/itching
|
25.0%
1/4 • Number of events 1
|
|
Cardiac disorders
Volume overload
|
25.0%
1/4 • Number of events 1
|
|
General disorders
Weight loss
|
25.0%
1/4 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place