Trial Outcomes & Findings for Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS (NCT NCT02626715)
NCT ID: NCT02626715
Last Updated: 2024-07-16
Results Overview
Number of non-relapsed deaths that occur
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
Day 100
Results posted on
2024-07-16
Participant Flow
Participant milestones
| Measure |
Reduced-Intensity Conditioning
Conditioning Arm nonrandomly assigned based on eligibility (inclusion/exclusion) criteria.
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
Conditioning Arm nonrandomly assigned based on eligibility (inclusion/exclusion) criteria.
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
15
|
|
Overall Study
COMPLETED
|
6
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS
Baseline characteristics by cohort
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) , Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
17.3 years
n=5 Participants
|
8 years
n=7 Participants
|
12.3 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
14 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Region of Enrollment
Puerto Rico
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Diagnosis
Acute Myeloid Leukemia (AML)
|
5 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Diagnosis
Myelodysplastic syndrome (MDS)
|
1 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Stem Cell Source
Bone Marrow
|
4 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Stem Cell Source
Peripheral Blood
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Stem Cell Source
Umbilical Cord Blood
|
2 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Donor Source
Sibling donor
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Donor Source
Unrelated donor
|
2 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 100Number of non-relapsed deaths that occur
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Number of Non-relapsed Deaths by 100 Days Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
|
1 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: 6 monthsEvent-free survival at 6 months, where events are defined as relapse or death
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Preliminary Efficacy (Event-free Survival at 6 Months) in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
|
5 Participants
|
14 Participants
|
PRIMARY outcome
Timeframe: Day 180Population: Analysis population includes only subjects without relapse (i.e. subjects who relapse are therefore excluded from this analysis).
Number of non-relapsed deaths that occur
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=14 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Number of Non-relapsed Deaths by 6 Months Post-transplant in Pediatric Patients Receiving a Myeloablative or Reduced-intensity Preparative Regimen Prior to HSCT for High-risk AML and MDS.
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day of transplant to end of study (Day 365)Neutrophil Engraftment (the first of three consecutive days in which the absolute neutrophil count (ANC) exceeded 500/mcL)
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
The Pace of Neutrophil Recovery
|
13 Days
Interval 9.0 to 18.0
|
13 Days
Interval 9.0 to 24.0
|
SECONDARY outcome
Timeframe: Day of transplant to end of study (Day 365)Platelet Engraftment (the first of three consecutive days in which the platelet count exceeded 20,000/mm3 without platelet transfusions for the preceding 7 days)
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
The Pace of Platelet Recovery
|
38 days
Interval 25.0 to 41.0
|
32 days
Interval 18.0 to 50.0
|
SECONDARY outcome
Timeframe: Day of transplant to end of study (Day 365)Population: Subjects must survive at least until 100 days post-transplant for evaluation for acute graft-versus-host disease, i.e. subjects who die before 100 days post-transplant are not evaluable and therefore excluded from analysis.
Grade 0: no stage 1-4 of any organ. Grade I: stage 1-2 skin rash (stage 1: \<25%, stage 2: 25-50% body surface area affected by maculopapular rash), no gut or liver involvement. Grade II: stage 3 skin rash (\>50% body surface area affected), or stage 1 GI involvement (10-19.9 mL/kg/day volume of diarrhea), or stage 1 liver involvement (2-3 mg/dL total bilirubin level). Grade III: stage 0-3 skin rash with stage 2-4 GI involvement (stage 2: 20-30 mL/kg/day, stage 3: \>30 mL/kg/day volume of diarrhea, stage 4: severe abdominal pain with or without ileus and/or grossly blood stool) or stage 2-3 liver involvement (stage 2: 3-6 mg/dL, stage 3: 6-15 mg/dL total bilirubin level). Grade IV: stage 4 skin rash (generalized erythroderma with bullous formation) or stage 4 liver involvement (\>15 mg/dL total bilirubin level).
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=5 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
No aGVHD (Grade 0)
|
3 Participants
|
7 Participants
|
|
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
aGVHD Grade I
|
2 Participants
|
3 Participants
|
|
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
aGVHD Grade II
|
0 Participants
|
5 Participants
|
|
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
aGVHD Grade III
|
0 Participants
|
0 Participants
|
|
Number of Participants Developing Acute Graft Versus Host Disease (aGVHD) by Grade
aGVHD Grade IV
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day of transplant to end of study (Day 365)Population: Subjects must survive at least until 365 days post-transplant for evaluation for chronic graft-versus-host disease, i.e. subjects who die before 365 days post-transplant are not evaluable and therefore excluded from analysis.
Limited cGVHD: localized skin involvement and/or hepatic dysfunction due to cGVHD. Extensive cGVHD: one or more of the following: generalized skin involvement; liver histology showing chronic aggressive hepatitis, bridging necrosis, or cirrhosis; involvement of eye, minor salivary glands or oral mucosa based on biopsy, or any other target organ. Mild cGVHD: 1 or 2 organs involved with no more than score 1 plus lung score 0. Moderate cGVHD: 3 or more organs involved with no more than score 1, or at least one organ (not lung) with score 2, or lung score 1. Severe cGVHD: at least one organ with score 3, or lung score 2 or 3.
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=5 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=13 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
No cGVHD
|
3 Participants
|
8 Participants
|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Limited cGVHD
|
1 Participants
|
4 Participants
|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Extensive cGVHD
|
1 Participants
|
1 Participants
|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Mild cGVHD
|
2 Participants
|
4 Participants
|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Moderate cGVHD
|
0 Participants
|
0 Participants
|
|
Number of Participants Developing Chronic Graft Versus Host Disease (cGVHD) by Grade
Severe cGVHD
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day 100 and 180 post-transplantThe number of subjects who are alive without relapse/leukemia
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Disease-free Survival (DFS)
Day 100
|
5 Participants
|
14 Participants
|
|
Disease-free Survival (DFS)
Day 180
|
5 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: Day 100 and 180 post-transplantPopulation: Subjects who die due to transplant-related causes by 100 and 180 days post-transplant, thus subjects who relapse before that time are excluded from analysis.
The number of subjects deceased due to transplant-related (i.e. non-relapse) causes
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=14 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Treatment-related Mortality (TRM)
Day 100
|
1 Participants
|
0 Participants
|
|
Treatment-related Mortality (TRM)
Day 180
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 100 and 180 post-transplantThe number of subjects who are alive
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
The Number of Subjects With Overall Survival (OS)
Day 100
|
5 Participants
|
15 Participants
|
|
The Number of Subjects With Overall Survival (OS)
Day 180
|
5 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: Day 0Population: Only 2 subjects in Reduced-Intensity Conditioning arm and no subjects in Myeloablative Conditioning arm had a Day 0 Campath (Alemtuzumab) level drawn.
Campath (Alemtuzumab) level measured on the day of transplant
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=2 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Day 0 Campath (Alemtuzumab) Level
|
0.24 mcg/mL
Interval 0.16 to 0.32
|
—
|
SECONDARY outcome
Timeframe: Day 180Population: One subject in each arm did not survive to Day 180
Immune recovery as measured by lymphocyte subsets
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=5 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=14 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Pace of Immune Reconstitution
CD3 Count (Total T-cells)
|
405 cells/mm3
Interval 212.0 to 900.0
|
405 cells/mm3
Interval 122.0 to 2569.0
|
|
Pace of Immune Reconstitution
CD4 Count (Helper T-cell)
|
171 cells/mm3
Interval 128.0 to 270.0
|
199.5 cells/mm3
Interval 56.0 to 733.0
|
|
Pace of Immune Reconstitution
CD8 Count (Suppressor T-cells)
|
222 cells/mm3
Interval 65.0 to 577.0
|
113 cells/mm3
Interval 39.0 to 2067.0
|
|
Pace of Immune Reconstitution
CD19 Count (Total B-cells)
|
347 cells/mm3
Interval 101.0 to 1615.0
|
366.5 cells/mm3
Interval 0.0 to 2022.0
|
|
Pace of Immune Reconstitution
CD16/56 Count (Total NK cells)
|
359 cells/mm3
Interval 147.0 to 726.0
|
218 cells/mm3
Interval 25.0 to 1288.0
|
SECONDARY outcome
Timeframe: Post-transplant to 42 days post-transplantThe failure to achieve an ANC ≥500/μL after 42 days, determined by three consecutive measurements on different days, and not caused by recurrent leukemia.
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Incidence of Primary Graft Failure.
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 180Adverse events as assessed by CTCAE
Outcome measures
| Measure |
Reduced-Intensity Conditioning
n=6 Participants
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 Participants
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Incidence of Grades 4 and 5 Adverse Events
White blood dell decreased
|
6 Participants
|
15 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Platelet count decreased
|
6 Participants
|
15 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Lymphocyte count decreased
|
6 Participants
|
15 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Neutrophil count decreased
|
6 Participants
|
12 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Sepsis
|
1 Participants
|
6 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Anemia
|
1 Participants
|
1 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Hyperglycemia
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Alkaline phosphatase elevated
|
0 Participants
|
1 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
GGT increased
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Febrile neutropenia
|
0 Participants
|
1 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Pneumonitis
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Pneumothorax
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Bronchopulmonary hemorrhage
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Multi-organ failure
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Cardiac arrest
|
1 Participants
|
0 Participants
|
|
Incidence of Grades 4 and 5 Adverse Events
Encephalitis infection
|
1 Participants
|
0 Participants
|
Adverse Events
Reduced-Intensity Conditioning
Serious events: 6 serious events
Other events: 0 other events
Deaths: 1 deaths
Myeloablative Conditioning
Serious events: 15 serious events
Other events: 0 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Reduced-Intensity Conditioning
n=6 participants at risk
Campath (alemtuzumab), Droxia (hydroxyurea), Fludara (fludarabine), Alkeran (melphalan), Thiotepa (triethylenethiophosphoramide)
Trade Name (generic name)
Reduced-Intensity Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Droxia (hydroxyurea) - drug class: antimetabolite Fludara (fludarabine) - drug class: antimetabolite Alkeran (melphalan) - drug class: alkylating agent Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent
|
Myeloablative Conditioning
n=15 participants at risk
Campath (alemtuzumab), Thiotepa (triethylenethiophosphoramide) ,Fludara (fludarabine), Busulfex (busulfan)
Trade Name (generic name)
Myeloablative Conditioning Regimen: Campath (alemtuzumab) - drug class: monoclonal antibody Thiotepa (triethylenethiophosphoramide) - drug class: cytotoxic agent Fludara (fludarabine) - drug class: antimetabolite Busulfex (busulfan) - drug class: alkylating agent
|
|---|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 2 • Day 180
|
|
Cardiac disorders
Cardiac arrest
|
16.7%
1/6 • Number of events 2 • Day 180
|
0.00%
0/15 • Day 180
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Gastrointestinal disorders
Mucositis oral
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
General disorders
Multi-organ failure
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Hepatobiliary disorders
Sinusoidal obstruction syndromw
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Infections and infestations
Sepsis
|
50.0%
3/6 • Number of events 4 • Day 180
|
66.7%
10/15 • Number of events 11 • Day 180
|
|
Infections and infestations
Viremia
|
16.7%
1/6 • Number of events 2 • Day 180
|
13.3%
2/15 • Number of events 3 • Day 180
|
|
Infections and infestations
Enterocolitis infectious
|
0.00%
0/6 • Day 180
|
13.3%
2/15 • Number of events 3 • Day 180
|
|
Infections and infestations
Lung infection
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Infections and infestations
Upper respiratory infection
|
16.7%
1/6 • Number of events 1 • Day 180
|
20.0%
3/15 • Number of events 5 • Day 180
|
|
Injury, poisoning and procedural complications
Vascular access complication
|
0.00%
0/6 • Day 180
|
13.3%
2/15 • Number of events 2 • Day 180
|
|
Nervous system disorders
Syncope
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Psychiatric disorders
Delirium
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/6 • Day 180
|
13.3%
2/15 • Number of events 2 • Day 180
|
|
Vascular disorders
Thromboembolic event
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Investigations
White blood cell decreased
|
100.0%
6/6 • Number of events 6 • Day 180
|
100.0%
15/15 • Number of events 15 • Day 180
|
|
Investigations
Platelet count decreased
|
100.0%
6/6 • Number of events 6 • Day 180
|
100.0%
15/15 • Number of events 15 • Day 180
|
|
Investigations
Lymphocyte count decreased
|
100.0%
6/6 • Number of events 6 • Day 180
|
100.0%
15/15 • Number of events 15 • Day 180
|
|
Investigations
Neutrophil count decreased
|
100.0%
6/6 • Number of events 6 • Day 180
|
80.0%
12/15 • Number of events 12 • Day 180
|
|
Blood and lymphatic system disorders
Anemia
|
16.7%
1/6 • Number of events 1 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Investigations
Alkaline phosphatase increased
|
0.00%
0/6 • Day 180
|
6.7%
1/15 • Number of events 1 • Day 180
|
|
Investigations
GGT increased
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
|
Infections and infestations
Encephalitis infection
|
16.7%
1/6 • Number of events 1 • Day 180
|
0.00%
0/15 • Day 180
|
Other adverse events
Adverse event data not reported
Additional Information
Dr. Randy Windreich
UPMC Children's Hospital of Pittsburgh
Phone: 412-692-5055
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place