Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as diacerein 1% ointment, Diacerein 1% ointment, Diacerein 1% Ointment
diacerein 1% · 6 trials · 6 indications
Number and percentage of participants with any treatment-emergent adverse event.
Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods. For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose. Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only. Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations.
The determination of dermal sensitization potential was based on the recurrence of a cutaneous response at re-challenge that was equivalent or more severe than the cumulative irritation score observed during the challenge period. Cumulative irritation score is a visual score rating the degree of erythema, edema, and other signs of cutaneous irritation: Minimum = 0; Maximum = 6; higher score is worse outcome.
Evaluation of the sum of all subjects' cumulative irritation scores for 21 days at each patch site. Cumulative Irritation Score is a visual score rating the degree of erythema, edema, and other signs of cutaneous irritation: Minimum = 0; Maximum = 6; higher score is worse outcome.
The determination of photosensitization reactions was summarized by frequency counts of the total dermal irritation score during the Challenge Phase. Total Irritation Score is a visual score summing the degree of erythema and edema. Minimum score=0, maximum score=5; higher score=worse outcome
The mean of the total irritation scores 24 and 48 hours post the site irradiation procedure. ( i.e. Day 3 and 4) Total Irritation Score is a visual score summing the degree of erythema and edema. Minimum score= 0; Maximum score = 5; higher score = worse outcome.
| Arm | Type | Description |
|---|---|---|
| Treatment period | EXPERIMENTAL | Subjects with active lesions as determined Investigator's clinical assessment, will initiate a cycle of applying diacerein 1% ointment once-daily, study medication, at home to their EBS lesions for 8 weeks. Following the Treatment Period, subjects will go Off Treatment for 8 weeks using only investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens. As determined Investigator's clinical assessment, subjects may enter into another Treatment Period of 8 weeks. The duration of a subject's participation in the extension study may be as short as 32 weeks or as long as 52 weeks depending on the cycle initiation schedule for each individual subject. |
| Cohort 1 (Adolescents, Adults) | EXPERIMENTAL | Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days. |
| Cohort 2 (Children) | EXPERIMENTAL | Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days. |
| Single cohort (Healthy Volunteers) | EXPERIMENTAL | diacerein 1% ointment |
| study in healthy volunteers | OTHER | diacerein 1% ointment |
| Name | Type | Description |
|---|---|---|
| diacerein 1% ointment | DRUG | Topically apply study medication to lesions identified by the Investigator that require. treatment |
Key Inclusion Criteria: * The subject is capable of understanding and complying with protocol requirements. * The subject or the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any ...
Diacerein 1% is an investigational topical ointment being studied for the treatment of Epidermolysis Bullosa (EB), a group of rare genetic skin disorders. It has been evaluated in clinical trials for Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, and Junctional Epidermolysis Bullosa. It is also studied in healthy subjects for safety assessments.
Diacerein 1% is being developed by Castle Biosciences, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol CSTL. The company has sponsored clinical trials evaluating the safety and pharmacokinetics of this topical formulation in patients with Epidermolysis Bullosa.
Diacerein 1% has completed Phase 1 and Phase 2 clinical trials. The development program includes a completed Phase 2 long-term open-label safety study and three completed Phase 1 studies. It remains an investigational drug and is not approved by the FDA.
Diacerein 1% has completed four clinical trials. NCT03389308 was a Phase 2 long-term open-label safety study in Epidermolysis Bullosa Simplex. NCT03472287 evaluated pharmacokinetics in EB patients. NCT03472547 and NCT03473197 assessed sensitizing and photoallergic potential in healthy subjects.
Diacerein 1% is a specific topical formulation of the small molecule diacerein, developed as a 1% ointment. It is being studied for topical application in Epidermolysis Bullosa. The drug is also known by its active metabolite rhein, which was measured in pharmacokinetic studies.