Trial Outcomes & Findings for Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (NCT NCT03389308)

Last Updated: 2020-09-16

Results Overview

Number and percentage of participants with any treatment-emergent adverse event.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

51 participants

Primary outcome timeframe

up to 52 weeks

Results posted on

2020-09-16

Participant milestones
Measure	Diacerein 1% Ointment Subjects with active lesions as determined Investigator's clinical assessment, will apply diacerein 1% ointment once-daily, to their EBS lesions for 8 weeks. Following an off treatment period of 8 weeks or more, subjects may enter into another Treatment Period of 8 weeks. The subject's participation may range from 32 weeks to 52 weeks.
Overall Study STARTED	51
Overall Study COMPLETED	39
Overall Study NOT COMPLETED	12

Reasons for withdrawal
Measure	Diacerein 1% Ointment Subjects with active lesions as determined Investigator's clinical assessment, will apply diacerein 1% ointment once-daily, to their EBS lesions for 8 weeks. Following an off treatment period of 8 weeks or more, subjects may enter into another Treatment Period of 8 weeks. The subject's participation may range from 32 weeks to 52 weeks.
Overall Study Withdrawal by Subject	5
Overall Study Lost to Follow-up	3
Overall Study Adverse Event	2
Overall Study Sponsor Request	2

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort
Measure	Diacerein 1% Ointment n=51 Participants Subjects with active lesions as determined Investigator's clinical assessment, will apply diacerein 1% ointment once-daily, to their EBS lesions for 8 weeks. Following an off treatment period of 8 weeks or more, subjects may enter into another Treatment Period of 8 weeks. The subject's participation may range from 32 weeks to 52 weeks.
Age, Categorical <=18 years	40 Participants n=51 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=51 Participants
Age, Categorical >=65 years	0 Participants n=51 Participants
Age, Continuous	12.6 years STANDARD_DEVIATION 10.70 • n=51 Participants
Sex: Female, Male Female	26 Participants n=51 Participants
Sex: Female, Male Male	25 Participants n=51 Participants
Region of Enrollment Austria	1 Participants n=51 Participants
Region of Enrollment Israel	5 Participants n=51 Participants
Region of Enrollment Australia	2 Participants n=51 Participants
Region of Enrollment United Kingdom	7 Participants n=51 Participants
Region of Enrollment Netherlands	3 Participants n=51 Participants
Region of Enrollment France	9 Participants n=51 Participants
Region of Enrollment Germany	2 Participants n=51 Participants
Region of Enrollment United States	22 Participants n=51 Participants

Timeframe: up to 52 weeks

Population: Safety population: All subjects who received at least one application of diacerein 1% ointment.

Number and percentage of participants with any treatment-emergent adverse event.

Outcome measures
Measure	Diacerein 1% Ointment n=51 Participants Subjects with active lesions as determined Investigator's clinical assessment, will apply diacerein 1% ointment once-daily, to their EBS lesions for 8 weeks. Following an off treatment period of 8 weeks or more, subjects may enter into another Treatment Period of 8 weeks. The subject's participation may range from 32 weeks to 52 weeks.
Treatment-emergent Adverse Events	40 Participants

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

Adverse event data not reported

Other adverse events
Measure	Diacerein 1% Ointment n=51 participants at risk Subjects with active lesions as determined Investigator's clinical assessment, will apply diacerein 1% ointment once-daily, to their EBS lesions for 8 weeks. Following an off treatment period of 8 weeks or more, subjects may enter into another Treatment Period of 8 weeks. The subject's participation may range from 32 weeks to 52 weeks.
Infections and infestations Nasopharyngitis	13.7% 7/51 • Number of events 10 • Up to 52 weeks
Infections and infestations Skin infection	9.8% 5/51 • Number of events 9 • Up to 52 weeks
Infections and infestations Ear infection	5.9% 3/51 • Number of events 3 • Up to 52 weeks
Infections and infestations Gastroenteritis viral	5.9% 3/51 • Number of events 3 • Up to 52 weeks
General disorders Pyrexia	7.8% 4/51 • Number of events 4 • Up to 52 weeks
Respiratory, thoracic and mediastinal disorders Cough	7.8% 4/51 • Number of events 5 • Up to 52 weeks
Skin and subcutaneous tissue disorders Pruritus	7.8% 4/51 • Number of events 6 • Up to 52 weeks

Castle Creek Pharmaceuticals

Phone: 312-847-1291

Restriction type: LTE60