Trial Outcomes & Findings for Safety and Efficacy of Ingenol Mebutate Gel 0.015% for Treatment of AK on the Face in Solid Organ Transplant Recipients (NCT NCT02866695)
NCT ID: NCT02866695
Last Updated: 2022-03-09
Results Overview
To evaluate the safety of ingenol mebutate gel 0.015% on the face in solid organ transplant recipients
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
20 participants
Primary outcome timeframe
From screening to Day 57 or study early termination if applicable
Results posted on
2022-03-09
Participant Flow
Participant milestones
| Measure |
Open Label Treatment
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Overall Study
STARTED
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20
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Overall Study
COMPLETED
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18
|
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Overall Study
NOT COMPLETED
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2
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Reasons for withdrawal
| Measure |
Open Label Treatment
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Overall Study
Adverse Event
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1
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Overall Study
Lost to Follow-up
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1
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Baseline Characteristics
Safety and Efficacy of Ingenol Mebutate Gel 0.015% for Treatment of AK on the Face in Solid Organ Transplant Recipients
Baseline characteristics by cohort
| Measure |
Open Label Treatment
n=20 Participants
All patients will be treated with ingenol mebutate gel 0.015%. There is no placebo or comparator for this study. The investigator will identify the patient's treatment area at Baseline, and provide a detailed application instruction sheet. The first dose will be applied in clinic under the supervision of the investigator.
ingenol mebutate gel 0.015%: ingenol mebutate gel 0.015% to be applied on treatment area
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|---|---|
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Age, Continuous
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65.3 years
n=5 Participants
|
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Sex: Female, Male
Female
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6 Participants
n=5 Participants
|
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Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=5 Participants
|
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Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
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Race (NIH/OMB)
Asian
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
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Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
20 Participants
n=5 Participants
|
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Race (NIH/OMB)
More than one race
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0 Participants
n=5 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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Region of Enrollment
United States
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20 participants
n=5 Participants
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Organ Transplant Type
Lung
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9 Participants
n=5 Participants
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Organ Transplant Type
Kidney
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7 Participants
n=5 Participants
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Organ Transplant Type
Kidney/Pancreas
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2 Participants
n=5 Participants
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Organ Transplant Type
Kidney/Liver
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1 Participants
n=5 Participants
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Organ Transplant Type
Kidney/Heart
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1 Participants
n=5 Participants
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Years Since Transplant
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10.9 years
n=5 Participants
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PRIMARY outcome
Timeframe: From screening to Day 57 or study early termination if applicableTo evaluate the safety of ingenol mebutate gel 0.015% on the face in solid organ transplant recipients
Outcome measures
| Measure |
Open Label Treatment
n=20 Participants
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Number of Participants With Adverse Events
Facial edema
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12 Participants
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Number of Participants With Adverse Events
Pain in Treatment Area
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11 Participants
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Number of Participants With Adverse Events
Pruritus
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6 Participants
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Number of Participants With Adverse Events
Headache
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3 Participants
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Number of Participants With Adverse Events
Squamous Cell Carcinoma Outside Treatment Area
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3 Participants
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Number of Participants With Adverse Events
Diarrhea
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2 Participants
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Number of Participants With Adverse Events
Xerosis
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2 Participants
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Number of Participants With Adverse Events
Urosepsis
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1 Participants
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Number of Participants With Adverse Events
Worsening of CKD
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3 Participants
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Number of Participants With Adverse Events
Hyperglycemia
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2 Participants
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Number of Participants With Adverse Events
Acute Kidney Injury
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1 Participants
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Number of Participants With Adverse Events
Hyperkalemia
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1 Participants
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Number of Participants With Adverse Events
Anemia
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1 Participants
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Number of Participants With Adverse Events
Hypertriglyceridemia
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1 Participants
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SECONDARY outcome
Timeframe: Day 57 (assessed at screening, day 1, day 29, day 57, and study early termination if applicable, clearance at day 57 reported)To evaluate the efficacy of ingenol mebutate for reduction of actinic keratoses on the face in organ transplant recipients.
Outcome measures
| Measure |
Open Label Treatment
n=20 Participants
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Number of Participants With a Reduction of Actinic Keratosis
75% Clearance of Actinic Keratoses at Day 57
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12 Participants
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Number of Participants With a Reduction of Actinic Keratosis
100% Clearance of Actinic Keratoses at Day 57
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8 Participants
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SECONDARY outcome
Timeframe: Day 1, Day 4 (assessed at screening, day 1, day 4, day 29, day 57, and study early termination if applicable, results at day 1 and day 4 reported)To investigate the local skin response to ingenol mebutate in organ transplant recipients. Local skin reaction (LSR) will be graded by visual assessment on a 4 point scale where 0=none, 1=mild, 2=moderate, 3=severe. Each of the following four reactions will be graded: erythema, vesiculation/postulation, crusting/scabbing/erosion, and edema/swelling. A composite score will be calculated (range 0-16). A high LSR indicated a worse outcome. LSR assessment will be performed at days 1, 8, 29, and 84.
Outcome measures
| Measure |
Open Label Treatment
n=20 Participants
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Participant's Local Skin Response Grading Scale
Baseline Local Skin Reaction Composite Score
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1.2 score on a scale
Standard Deviation 0.8
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Participant's Local Skin Response Grading Scale
Day 4 Local Skin Reaction Composite Score
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11.7 score on a scale
Standard Deviation 4.2
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Adverse Events
Open Label Treatment
Serious events: 5 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Open Label Treatment
n=20 participants at risk
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Renal and urinary disorders
Urosepsis
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Gastrointestinal disorders
Diarrhea
|
5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Renal and urinary disorders
Chronic Kidney Disease
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Blood and lymphatic system disorders
Anemia
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Metabolism and nutrition disorders
Hypertriglyceridemia
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Other adverse events
| Measure |
Open Label Treatment
n=20 participants at risk
A total of 20 solid organ transplant recipients with 4 to 20 actinic keratoses were enrolled and treated for 3 consecutive days with 4 single-dose tubes of ingenol mebutate gel 0.015% applied to a 100 cm2 area of the face according to study protocol. There was no placebo or comparator for this study. All but 2 participants completed the treatment. One kidney transplant participant forgot to apply the treatment on day 3, but continued in the study. One lung transplant participant who reported pain in the treatment area withdrew from the study after day 2 dosing due to travel considerations and was lost to follow-up.
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|---|---|
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Metabolism and nutrition disorders
Hyperglycemia
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10.0%
2/20 • Number of events 2 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
|
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Renal and urinary disorders
Acute Kidney Injury
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20.0%
4/20 • Number of events 4 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Renal and urinary disorders
Hyperkalemia
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Renal and urinary disorders
Hypocalcemia
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Renal and urinary disorders
Hyponatremia
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10.0%
2/20 • Number of events 2 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Renal and urinary disorders
Hypoalbuminemia
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10.0%
2/20 • Number of events 2 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Blood and lymphatic system disorders
Neutropenia
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5.0%
1/20 • Number of events 1 • Each participant was assessed for 57 days from the start of treatment.
CTC Adverse Events Grading Criteria version 4.0 was used to define adverse events
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Additional Information
Dr. Sarah Arron, MD, PhD
University of California San Francisco Dept. of Dermatology
Phone: 415-353-9684
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place