Trial Outcomes & Findings for Sirolimus & Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor HCT (NCT NCT01220297)
NCT ID: NCT01220297
Last Updated: 2017-06-05
Results Overview
Assessed as the incidence of grade 2 to 4 acute graft-vs-host disease (GvHD) at Day 100 post-transplant. Stage of Acute GvHD was assessed as follows. * Stage 1: Skin: rash \< 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea \> 500 mL/day or persistent nausea with positive biopsy for GvHD * Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea \>1000 mL/day. * Stage 3: Skin: rash \> 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea \> 1500 mL/day. * Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin \> 15 mg/dL. Gut: severe abdominal pain with or without ileus Grade of Acute GvHD was determined as follows. * Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage * Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut * Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut * Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
3 participants
Primary outcome timeframe
100 days post-transplant
Results posted on
2017-06-05
Participant Flow
Participant milestones
| Measure |
Graft-vs-Host Disease (GvHD) Prophlyaxis
Sirolimus \& Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Graft-vs-Host Disease (GvHD) Prophlyaxis
Sirolimus \& Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative
|
|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Sirolimus & Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor HCT
Baseline characteristics by cohort
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
—
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
—
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
—
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Disease Characteristics
Acute Lymphoblastic Leukemia (ALL)
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Disease Characteristics
Acute Myeloid Leukemia (AML)
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Disease Characteristics
Biphenotypic Acute Leukaemia (BAL)
|
—
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 days post-transplantAssessed as the incidence of grade 2 to 4 acute graft-vs-host disease (GvHD) at Day 100 post-transplant. Stage of Acute GvHD was assessed as follows. * Stage 1: Skin: rash \< 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea \> 500 mL/day or persistent nausea with positive biopsy for GvHD * Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea \>1000 mL/day. * Stage 3: Skin: rash \> 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea \> 1500 mL/day. * Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin \> 15 mg/dL. Gut: severe abdominal pain with or without ileus Grade of Acute GvHD was determined as follows. * Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage * Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut * Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut * Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage
Outcome measures
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 100 days post-transplantAssessed as the incidence of grade 3 to 4 acute GvHD at Day 100 post-transplant. Stage of Acute GvHD was assessed as follows. * Stage 1: Skin: rash \< 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea \> 500 mL/day or persistent nausea with positive biopsy for GvHD * Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea \>1000 mL/day. * Stage 3: Skin: rash \> 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea \> 1500 mL/day. * Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin \> 15 mg/dL. Gut: severe abdominal pain with or without ileus Grade of Acute GvHD was determined as follows. * Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage * Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut * Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut * Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage
Outcome measures
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Acute GvHD (Grade 3 to 4)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 yearsAssessed as survival without recurrence of disease
Outcome measures
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Disease-free Survival (DFS)
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 yearsOverall survival is defined as time from enrollment to time of death or last follow-up, within 2 years.
Outcome measures
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Overall Survival
|
—
|
405 Days
Interval 72.0 to 423.0
|
SECONDARY outcome
Timeframe: 100 days post-transplantAssessed as the incidence of veno-occlusive disease (VoD) at 100 days post-transplant.
Outcome measures
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 Participants
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Veno-occlusive Disease (VoD)
|
0 Participants
|
1 Participants
|
Adverse Events
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
Serious events: 3 serious events
Other events: 3 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 participants at risk
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Death
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Renal and urinary disorders
Veno-occlusive disease (VOD)
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Renal and urinary disorders
Hematuria
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 3 • 3 years
|
|
General disorders
Fever
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Neutropenic fever
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 3 • 3 years
|
|
Blood and lymphatic system disorders
AML relapsed
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Cardiac disorders
Centrilobular nodularity
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Macular erythema
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 2 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Acute graft vs host disease (GvHD)
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Infections and infestations
Pneumonia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Sepsis
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Renal and urinary disorders
Urgency to urinate
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Vesicles
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
Other adverse events
| Measure |
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after chemotherapeutic regimen of carmustine (BCNU) + etoposide (VP-16) + cyclophosphamide (Cyclo)
|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo
n=3 participants at risk
Graft-vs-host disease (GvHD) prophylaxis of sirolimus \& mycophenolate mofetil (MMF) after therapeutic regimen of cyclophosphamide (Cyclo) chemotherapy and fractionated total body irradiation (FTBI)
|
|---|---|---|
|
Gastrointestinal disorders
Dyspepsia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Hyperglycemia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Abdominal cramping
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
BLE Numbness
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 2 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Congestion, Nasal
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Constipation
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
Dehydration
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Gastrointestinal disorders
Diarrhea
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
Dizziness
|
—
0/0 • 3 years
|
100.0%
3/3 • Number of events 4 • 3 years
|
|
Renal and urinary disorders
Dysuria
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Edema
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Blood and lymphatic system disorders
Epistaxis
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
Fatigue
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
General disorders
Hallucinations
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Hand Pain
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
Headaches
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Renal and urinary disorders
Hemorrhoidal Pain
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Hypokalemia
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Hypoxia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Klebsiella pneumoniae
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Psychiatric disorders
Insomnia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Mucositis
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Nausea
|
—
0/0 • 3 years
|
100.0%
3/3 • Number of events 8 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Neuropathy
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Nervous system disorders
Paresthesia
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Pressure ulcer
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Respiratory, thoracic and mediastinal disorders
Sore Throat
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Blood and lymphatic system disorders
Syncope
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Cardiac disorders
Tachycardia
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Infections and infestations
Thrush
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Toe pain
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Upper Respiratory Infection
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Gastrointestinal disorders
Vomiting
|
—
0/0 • 3 years
|
100.0%
3/3 • Number of events 4 • 3 years
|
|
General disorders
Weakness
|
—
0/0 • 3 years
|
66.7%
2/3 • Number of events 2 • 3 years
|
|
General disorders
Chills
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Musculoskeletal and connective tissue disorders
Rigoring
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 1 • 3 years
|
|
Infections and infestations
Multifocal Pneumonia
|
—
0/0 • 3 years
|
0.00%
0/3 • 3 years
|
|
General disorders
Madibular pain
|
—
0/0 • 3 years
|
33.3%
1/3 • Number of events 2 • 3 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place