Trial Outcomes & Findings for Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (NCT NCT01005576)
NCT ID: NCT01005576
Last Updated: 2017-12-13
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
1 year
Results posted on
2017-12-13
Participant Flow
Participants were recruited based on physician referral at 11 academic medical centers. The first participant was enrolled in May 2010 and the last participant was enrolled in April 2012.
Participant milestones
| Measure |
Conditioning Regimen
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Baseline characteristics by cohort
| Measure |
Conditioning Regimen
n=21 Participants
Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan: Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Age, Continuous
|
10 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Asian
|
5 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Middle Eastern
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · African American
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Hispanic
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Pakistani
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Caucasian
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · Vietnamese
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race/Ethnicity · More than 1 race reported
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
21 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Conditioning Regimen
n=21 Participants
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Primary Objective: Event-free Survival at 1 Year.
|
17 Participants
|
SECONDARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Conditioning Regimen
n=21 Participants
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Development of Graft Versus Host Disease (GVHD)
Participants who developed any GVHD
|
12 Participants
|
|
Development of Graft Versus Host Disease (GVHD)
Participants who did not develop any GVHD
|
9 Participants
|
SECONDARY outcome
Timeframe: 100 daysOutcome measures
| Measure |
Conditioning Regimen
n=21 Participants
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Median Time to ANC Engraftment
|
14 days
Interval 10.0 to 46.0
|
SECONDARY outcome
Timeframe: 100 daysPopulation: 3 participants did not engraft platelets
Outcome measures
| Measure |
Conditioning Regimen
n=18 Participants
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Median Time to Platelet Engraftment
|
27.5 days
Interval 18.0 to 234.0
|
SECONDARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Conditioning Regimen
n=21 Participants
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Incidence of Disease Recurrence
|
1 Participants
|
Adverse Events
Conditioning Regimen
Serious events: 10 serious events
Other events: 21 other events
Deaths: 4 deaths
Serious adverse events
| Measure |
Conditioning Regimen
n=21 participants at risk
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary toxicity
|
19.0%
4/21
|
|
General disorders
Pain
|
4.8%
1/21
|
|
General disorders
Graft rejection
|
4.8%
1/21
|
|
General disorders
Edema
|
4.8%
1/21
|
|
Nervous system disorders
Posterior Reversible Encephalopathy Syndrome
|
4.8%
1/21
|
|
Infections and infestations
Sepsis
|
4.8%
1/21
|
|
Nervous system disorders
Headache
|
4.8%
1/21
|
Other adverse events
| Measure |
Conditioning Regimen
n=21 participants at risk
Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan:
Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion
|
|---|---|
|
Infections and infestations
CMV reactivation
|
66.7%
14/21
|
|
Hepatobiliary disorders
Veno Occlusive Disease
|
4.8%
1/21
|
|
Investigations
Hyperbilirubinemia
|
4.8%
1/21
|
|
Investigations
Hypercalcemia
|
4.8%
1/21
|
|
Renal and urinary disorders
Acute Kidney Injury
|
4.8%
1/21
|
|
Infections and infestations
Infection (other than CMV)
|
100.0%
21/21
|
Additional Information
Dr. Shalini Shenoy
Washington University School of Medicine
Phone: 314-454-6018
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place