Trial Outcomes & Findings for Bone Marrow Transplant Using a Reduced Intensity Regimen That is Given in Two Steps (NCT NCT01350258)
NCT ID: NCT01350258
Last Updated: 2025-05-31
Results Overview
To define the dose of MEL required for the establishment of peripheral T cell tolerance with concomitant immune reconstitution.
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
8 participants
Primary outcome timeframe
100 days post-transplant
Results posted on
2025-05-31
Participant Flow
Participant milestones
| Measure |
Transplant Treatment Group
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate IV over 60 minutes on days -11 to -8 and thiotepa IV (cytarabine IV as of February 2012) over 2 hours on days -11 to -9. Patients undergo TBI on day -6. Patients receive melphalan IV over 20 minutes on days -3 and -2.
TRANSPLANTATION: Patients undergo DLI on day -6 and allogeneic CD-34+ peripheral blood stem cell transplantation on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and receive MMF IV BID on days -1 to day 28.
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bone Marrow Transplant Using a Reduced Intensity Regimen That is Given in Two Steps
Baseline characteristics by cohort
| Measure |
Transplant Treatment Group
n=8 Participants
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate IV over 60 minutes on days -11 to -8 and thiotepa IV (cytarabine IV as of February 2012) over 2 hours on days -11 to -9. Patients undergo TBI on day -6. Patients receive melphalan IV over 20 minutes on days -3 and -2.
TRANSPLANTATION: Patients undergo DLI on day -6 and allogeneic CD-34+ peripheral blood stem cell transplantation on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and receive MMF IV BID on days -1 to day 28.
|
|---|---|
|
Age, Continuous
|
60.43 years
STANDARD_DEVIATION 7.86 • n=99 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
2 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
8 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 100 days post-transplantTo define the dose of MEL required for the establishment of peripheral T cell tolerance with concomitant immune reconstitution.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 100 days post-treatmentTo evaluate the 100 day non-relapse mortality (NRM) rate in patients undergoing HSCT treated on this successor TJU 2 Step RIC haploidentical regimen and compare it with that of the initial regimen.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At 1 and 3 yearsTo compare relapse rates in patients undergoing HSCT treated on this successor TJU 2 Step RIC haploidentical regimen and compare it with that of the initial regimen.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At 1 and 3 yearsTo determine the incidence and severity of graft-versus-host disease (GVHD) in patients undergoing treatment on this regimen using MEL for T cell tolerization as well as tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 days post-transplantTo evaluate engraftment rates and lymphoid reconstitution in patients treated on this trial.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At 1 and 3 yearsTo assess overall survival in patients undergoing HSCT treated on this trial.
Outcome measures
Outcome data not reported
Adverse Events
Transplant Treatment Group
Serious events: 8 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Transplant Treatment Group
n=8 participants at risk
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate IV over 60 minutes on days -11 to -8 and thiotepa IV (cytarabine IV as of February 2012) over 2 hours on days -11 to -9. Patients undergo TBI on day -6. Patients receive melphalan IV over 20 minutes on days -3 and -2.
TRANSPLANTATION: Patients undergo DLI on day -6 and allogeneic CD-34+ peripheral blood stem cell transplantation on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and receive MMF IV BID on days -1 to day 28.
|
|---|---|
|
Immune system disorders
CMV reactivation
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Colitis/enteritis
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhea
|
62.5%
5/8 • Number of events 5
|
|
General disorders
Fever
|
25.0%
2/8 • Number of events 2
|
|
Blood and lymphatic system disorders
Gram negative sepsis
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
HHV-6 viremia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hyperbilirubinemia
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Hypoxia
|
12.5%
1/8 • Number of events 1
|
|
Hepatobiliary disorders
Increased LFTs
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Lacunar infarct in pons
|
12.5%
1/8 • Number of events 1
|
|
Hepatobiliary disorders
Liver failure
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Orthostatic hypotension
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
12.5%
1/8 • Number of events 1
|
|
Renal and urinary disorders
Renal failure
|
25.0%
2/8 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Sepsis
|
12.5%
1/8 • Number of events 1
|
Other adverse events
| Measure |
Transplant Treatment Group
n=8 participants at risk
REDUCED INTENSITY CONDITIONING: Patients receive fludarabine phosphate IV over 60 minutes on days -11 to -8 and thiotepa IV (cytarabine IV as of February 2012) over 2 hours on days -11 to -9. Patients undergo TBI on day -6. Patients receive melphalan IV over 20 minutes on days -3 and -2.
TRANSPLANTATION: Patients undergo DLI on day -6 and allogeneic CD-34+ peripheral blood stem cell transplantation on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV or PO beginning on day -1 with taper beginning on day 42 and receive MMF IV BID on days -1 to day 28.
|
|---|---|
|
Cardiac disorders
Volume overload
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Abdominal cramping
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Abdominal pain
|
75.0%
6/8 • Number of events 7
|
|
Renal and urinary disorders
Acute kidney injury
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Acute pancreatitis
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Anorexia
|
25.0%
2/8 • Number of events 2
|
|
Psychiatric disorders
Anxiety
|
87.5%
7/8 • Number of events 7
|
|
Cardiac disorders
Arrhythmia
|
12.5%
1/8 • Number of events 1
|
|
Cardiac disorders
Atrial fibrillation
|
25.0%
2/8 • Number of events 3
|
|
General disorders
Back pain
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Balance problems
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Blister at PICC site
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Bloating
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Blood in stool
|
25.0%
2/8 • Number of events 2
|
|
Eye disorders
Blurred vision
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Body aches
|
12.5%
1/8 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Bowel obstruction
|
12.5%
1/8 • Number of events 1
|
|
Cardiac disorders
Bradycardia
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Buttocks wound
|
12.5%
1/8 • Number of events 1
|
|
Eye disorders
Changes in vision
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Chest discomfort
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Chest pain
|
25.0%
2/8 • Number of events 2
|
|
Hepatobiliary disorders
Cholecystitis
|
12.5%
1/8 • Number of events 1
|
|
Immune system disorders
CMV reactivation
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Constipation
|
37.5%
3/8 • Number of events 4
|
|
Cardiac disorders
Coronary artery disease
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Decreased appetite
|
12.5%
1/8 • Number of events 2
|
|
Renal and urinary disorders
Decreased urine output
|
25.0%
2/8 • Number of events 3
|
|
General disorders
Dehydration
|
12.5%
1/8 • Number of events 1
|
|
Psychiatric disorders
Depression
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Diarrhea
|
75.0%
6/8 • Number of events 10
|
|
Psychiatric disorders
Disturbing dreams
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Dizziness
|
25.0%
2/8 • Number of events 2
|
|
Eye disorders
Dry eyes
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Dry mouth
|
25.0%
2/8 • Number of events 4
|
|
General disorders
Dry nose/sinuses
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Dysphagia
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
75.0%
6/8 • Number of events 8
|
|
Renal and urinary disorders
Dysuria
|
12.5%
1/8 • Number of events 1
|
|
Ear and labyrinth disorders
Ear fullness
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Edema
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Electrolyte imbalance
|
25.0%
2/8 • Number of events 2
|
|
Hepatobiliary disorders
Increased LFTs
|
25.0%
2/8 • Number of events 3
|
|
Gastrointestinal disorders
Erythematous mucosa in duodenal bulb
|
12.5%
1/8 • Number of events 1
|
|
Eye disorders
Eye movement abnormality
|
12.5%
1/8 • Number of events 1
|
|
Eye disorders
Eye tearing
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Face/upper neck swelling
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Fatigue
|
75.0%
6/8 • Number of events 6
|
|
General disorders
Feels "fuzzy"
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Feet/ankle/leg swelling
|
25.0%
2/8 • Number of events 3
|
|
General disorders
Fever
|
87.5%
7/8 • Number of events 20
|
|
General disorders
Flank pain
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Fluid overload
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Flu-like symptoms
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Headache
|
62.5%
5/8 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
Hematoma
|
12.5%
1/8 • Number of events 1
|
|
Renal and urinary disorders
Hematuria
|
12.5%
1/8 • Number of events 1
|
|
Hepatobiliary disorders
Hepatitis
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
HHV-6 viremia
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Hiccups
|
25.0%
2/8 • Number of events 3
|
|
Blood and lymphatic system disorders
Hyperbilirubinemia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hyperglycemia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypernatremia
|
37.5%
3/8 • Number of events 4
|
|
Blood and lymphatic system disorders
Hyperphosphatemia
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Hyperpigmentation
|
25.0%
2/8 • Number of events 3
|
|
Blood and lymphatic system disorders
Hypertension
|
62.5%
5/8 • Number of events 5
|
|
Blood and lymphatic system disorders
Hypocalcemia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypokalemia
|
37.5%
3/8 • Number of events 3
|
|
Blood and lymphatic system disorders
Hypomagnesemia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hyponatremia
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Hypotension
|
50.0%
4/8 • Number of events 4
|
|
General disorders
Hypothermia
|
37.5%
3/8 • Number of events 4
|
|
General disorders
Hypoxia
|
37.5%
3/8 • Number of events 3
|
|
Gastrointestinal disorders
Ileus
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Increased creatinine
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Insomnia
|
37.5%
3/8 • Number of events 4
|
|
General disorders
Itchiness in throat
|
12.5%
1/8 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Leg pain
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Lethargy
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Lightheadedness
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Malaise
|
12.5%
1/8 • Number of events 1
|
|
Metabolism and nutrition disorders
Malnutrition
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Mental status change
|
62.5%
5/8 • Number of events 7
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
50.0%
4/8 • Number of events 4
|
|
Gastrointestinal disorders
Mucositis
|
62.5%
5/8 • Number of events 5
|
|
Musculoskeletal and connective tissue disorders
Muscle pain
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Nausea
|
75.0%
6/8 • Number of events 8
|
|
Nervous system disorders
Neuropathy
|
12.5%
1/8 • Number of events 1
|
|
Renal and urinary disorders
Nocturia
|
25.0%
2/8 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Non-productive cough
|
12.5%
1/8 • Number of events 1
|
|
Gastrointestinal disorders
Occult blood
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Oral discomfort
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Orthostatic hypotension
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Pain at PEG tube site
|
12.5%
1/8 • Number of events 1
|
|
Blood and lymphatic system disorders
Pericatheter thrombus
|
12.5%
1/8 • Number of events 1
|
|
Eye disorders
Photophobia
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Post nasal drip
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pressure ulcer
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
37.5%
3/8 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Psoriasis
|
12.5%
1/8 • Number of events 1
|
|
Eye disorders
Ptosis
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Purulent drainage
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash
|
75.0%
6/8 • Number of events 12
|
|
Renal and urinary disorders
Renal failure
|
25.0%
2/8 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory crackles
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhea
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Rigors/chills
|
50.0%
4/8 • Number of events 4
|
|
Reproductive system and breast disorders
Scrotal swelling
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Shoulder pain
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Skin tear
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Sore throat
|
25.0%
2/8 • Number of events 2
|
|
Gastrointestinal disorders
Stomach fluttering
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Sweats
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Syncope
|
12.5%
1/8 • Number of events 1
|
|
Cardiac disorders
Tachycardia
|
62.5%
5/8 • Number of events 6
|
|
General disorders
Tenderness at PICC site
|
12.5%
1/8 • Number of events 1
|
|
Infections and infestations
Thrush
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Tingling in lips
|
12.5%
1/8 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Tissue wound
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Tremors
|
37.5%
3/8 • Number of events 3
|
|
General disorders
Trouble sleeping
|
25.0%
2/8 • Number of events 2
|
|
Renal and urinary disorders
Urinary incontinence
|
12.5%
1/8 • Number of events 1
|
|
Renal and urinary disorders
Urinary retention
|
25.0%
2/8 • Number of events 2
|
|
Renal and urinary disorders
Urinary urgency
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Vision - flashing lights
|
12.5%
1/8 • Number of events 1
|
|
Psychiatric disorders
Vivid dreams/hallucinations
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Vomiting
|
25.0%
2/8 • Number of events 2
|
|
General disorders
Weight loss
|
12.5%
1/8 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
12.5%
1/8 • Number of events 1
|
|
General disorders
Wrist tenderness
|
12.5%
1/8 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place