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diacerein 1%

Phase 2

Epidermolysis Bullosa | Small molecule | Rare Disease |Castle Biosciences, Inc.|Last Updated: Sep 16, 2020

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment51

FDA Designations

No designations recorded

Clinical trial landscape

diacerein 1% · 6 trials · 6 indications

Phase 2 1Phase 1 5
NCT03389308Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa SimplexEpidermolysis Bullosa
COMPLETED51 Analytics
PHASE2COMPLETED
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Epidermolysis BullosaUnlock trial analytics

Study Endpoints

Primary Endpoints

Treatment-emergent Adverse Events
up to 52 weeks

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

Detectable Plasma Concentrations of Diacerein and Rhein
Days 1-10, at select time points per protocol

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.

Observed Sensitization
56 days

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.

Skin Irritation Score
Daily for 21 days

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.

Observed Photoallergy (Photosensitization): Number of Skin Sites by Maximum Total Irritation Score
Skin sites evaluated 24, 48, and 72 hours after patch removal and irradiation challenge

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

Observed Phototoxicity
Days 3 and 4 (24 and 48 hours post site irradiation procedure)

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.

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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Treatment periodEXPERIMENTALSubjects 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)EXPERIMENTALAdolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.
Cohort 2 (Children)EXPERIMENTALChildren with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.
Single cohort (Healthy Volunteers)EXPERIMENTALdiacerein 1% ointment
study in healthy volunteersOTHERdiacerein 1% ointment

Interventions

NameTypeDescription
diacerein 1% ointmentDRUGTopically apply study medication to lesions identified by the Investigator that require. treatment
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Eligibility Criteria

Age Range6 Months to 99 Years
SexALL
Healthy VolunteersNo
Study Sites3

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 ...

Countries:United States
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Frequently asked questions about diacerein 1%

What is Diacerein 1% used for?

Diacerein 1% is an investigational topical ointment being studied for the treatment of Epidermolysis Bullosa (EB), a rare genetic skin disorder. It is also used in clinical trials involving healthy subjects to evaluate its safety profile, including skin sensitization, phototoxicity, and photoallergic potential.

Who makes Diacerein 1%?

Diacerein 1% is being developed by Castle Biosciences, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol CSTL. The company is conducting clinical trials to evaluate the drug's safety and pharmacokinetics in patients with Epidermolysis Bullosa.

What phase is Diacerein 1% in?

Diacerein 1% is in Phase 1 clinical development. All four clinical trials associated with the drug are Phase 1 studies, and all have been completed. The drug is investigational and has not been approved by regulatory authorities for any indication.

What clinical trials is Diacerein 1% in?

Diacerein 1% has been studied in four completed Phase 1 clinical trials: NCT03472287 (pharmacokinetics in EB patients), NCT03472547 (skin sensitization in healthy subjects), NCT03473184 (phototoxicity in healthy volunteers), and NCT03473197 (photoallergic potential in healthy volunteers).

What does Diacerein 1% target?

Diacerein 1% is a small molecule that works by inhibiting the activity of interleukin-1 beta (IL-1β), a pro-inflammatory cytokine involved in the blistering and inflammation seen in Epidermolysis Bullosa. By reducing IL-1β activity, the drug aims to decrease skin inflammation and blister formation.

Is Diacerein 1% the same as diacerein?

Diacerein 1% is a topical formulation of diacerein, a compound that has been used orally for osteoarthritis in some countries. In this context, diacerein is formulated as a 1% ointment for topical application to treat Epidermolysis Bullosa, a different indication and route of administration.