Trial Outcomes & Findings for Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS) (NCT NCT03154333)
NCT ID: NCT03154333
Last Updated: 2019-11-05
Results Overview
Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
54 participants
Primary outcome timeframe
Baseline to Week 8
Results posted on
2019-11-05
Participant Flow
Participant milestones
| Measure |
Diacerein 1% Ointment
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
|---|---|---|
|
Overall Study
STARTED
|
28
|
26
|
|
Overall Study
COMPLETED
|
21
|
21
|
|
Overall Study
NOT COMPLETED
|
7
|
5
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
Baseline characteristics by cohort
| Measure |
Diacerein 1% Ointment
n=28 Participants
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
n=26 Participants
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
Total
n=54 Participants
Total of all reporting groups
|
|---|---|---|---|
|
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
|
24 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
47 Participants
n=5 Participants
|
|
Age, Customized
Age <8 years old
|
13 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
|
Age, Customized
Age greater than or equal to 8 years old
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
24 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
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 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)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Baseline BSA(Body Surface Area) of EBS(Epidermolysis Bullosa Simplex) Lesions within Assessment Area
|
5.76 Percent of BSA
STANDARD_DEVIATION 4.674 • n=5 Participants
|
7.13 Percent of BSA
STANDARD_DEVIATION 7.606 • n=7 Participants
|
6.42 Percent of BSA
STANDARD_DEVIATION 6.237 • n=5 Participants
|
|
Baseline IGA Score of 3 or 4
IGA Score: 3
|
26 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
|
Baseline IGA Score of 3 or 4
IGA Score: 4
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 8Population: All efficacy analyses were conducted using the Intent to Treat (ITT) Population that consisted of all randomized subjects who were dispensed study drug. Subjects were included in the treatment group to which they were assigned.
Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8
Outcome measures
| Measure |
Diacerein 1% Ointment
n=28 Participants
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
n=26 Participants
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
|---|---|---|
|
Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area
|
16 Participants
|
14 Participants
|
SECONDARY outcome
Timeframe: Baseline to Week 8Population: All efficacy analyses were conducted using the Intent to Treat (ITT) Population that consisted of all randomized subjects who were dispensed study drug. Subjects were included in the treatment group to which they were assigned.
The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4. Success on the IGA was defined as ≥2-point reduction from Baseline to Visit 6 (Week 8). IGA Scoring: 0 = Clear; 1 = Near Clear; 2 = Mild; 3 = Moderate; 4 = Severe Minimum score = 0 Maximum score = 4; higher score = worse outcome
Outcome measures
| Measure |
Diacerein 1% Ointment
n=28 Participants
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
n=26 Participants
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
|---|---|---|
|
The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)
|
12 Participants
|
7 Participants
|
Adverse Events
Diacerein 1% Ointment
Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths
Vehicle Ointment
Serious events: 2 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Diacerein 1% Ointment
n=28 participants at risk
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
n=26 participants at risk
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
|---|---|---|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.00%
0/28 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
Ear and labyrinth disorders
Meniere's Disease
|
0.00%
0/28 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
Other adverse events
| Measure |
Diacerein 1% Ointment
n=28 participants at risk
diacerein 1% ointment will be used for 8 weeks
diacerein 1% ointment: diacerein 1% ointment administered topically
|
Vehicle Ointment
n=26 participants at risk
vehicle ointment will be used for 8 weeks
vehicle ointment administered topically
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
14.3%
4/28 • Number of events 4 • Baseline to Week 16
|
7.7%
2/26 • Number of events 2 • Baseline to Week 16
|
|
Infections and infestations
Skin Infection
|
10.7%
3/28 • Number of events 4 • Baseline to Week 16
|
11.5%
3/26 • Number of events 4 • Baseline to Week 16
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
4/28 • Number of events 5 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
Gastrointestinal disorders
Diarrhoea
|
10.7%
3/28 • Number of events 4 • Baseline to Week 16
|
0.00%
0/26 • Baseline to Week 16
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
10.7%
3/28 • Number of events 3 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
Nervous system disorders
Headache
|
3.6%
1/28 • Number of events 1 • Baseline to Week 16
|
11.5%
3/26 • Number of events 3 • Baseline to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
4/28 • Number of events 4 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
7.7%
2/26 • Number of events 2 • Baseline to Week 16
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
3.6%
1/28 • Number of events 1 • Baseline to Week 16
|
7.7%
2/26 • Number of events 3 • Baseline to Week 16
|
|
Gastrointestinal disorders
Nausea
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
0.00%
0/26 • Baseline to Week 16
|
|
Skin and subcutaneous tissue disorders
Blister
|
3.6%
1/28 • Number of events 1 • Baseline to Week 16
|
7.7%
2/26 • Number of events 2 • Baseline to Week 16
|
|
Injury, poisoning and procedural complications
Skin Abrasion
|
0.00%
0/28 • Baseline to Week 16
|
7.7%
2/26 • Number of events 2 • Baseline to Week 16
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/28 • Baseline to Week 16
|
11.5%
3/26 • Number of events 4 • Baseline to Week 16
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
General disorders
Application Site Pruritus
|
0.00%
0/28 • Baseline to Week 16
|
7.7%
2/26 • Number of events 2 • Baseline to Week 16
|
|
General disorders
Fatigue
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
0.00%
0/26 • Baseline to Week 16
|
|
Ear and labyrinth disorders
Ear Pain
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
3.8%
1/26 • Number of events 1 • Baseline to Week 16
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
7.1%
2/28 • Number of events 2 • Baseline to Week 16
|
0.00%
0/26 • Baseline to Week 16
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place