CTIS2023-508818-42-00Active, not recruitingPhase 1
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study with Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS) - AC-203-EBS-007
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- Twi Biotechnology Inc.
- Enrollment
- 87
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 0 to 64 (—)
- Sex
- All
Inclusion Criteria
- •Patient is at least 6 months old at Visit 2 (Day 1/Baseline A)., Female patient of childbearing potential is willing to practice highly effective contraception (i.e., pregnancy prevention method with a failure rate of < 1% per year) from Screening throughout the end of the study., Patients has been clinically diagnosed with severe EBS or intermediate EBS, confirmed by documented genetic diagnosis to have autosomal dominant mutations in KRT5 or KRT14 gene, Patients with = 5% BSA of EBS lesions excluding hands and feet at Visit 2 (Day 1/Baseline A)., Patient’s EBS lesions within the Treatment Area have an IGA score of =3 at Visit 2 (Day 1/Baseline A)., Patient/caregiver agrees to follow study medication application instructions., Patient (and caregiver/legal guardian) agrees to report use of all prescription and over-the-counter medications, including topical therapies applied to the body, e.g., medical cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc. for the duration of the study., Patient (and caregiver/legal guardian) is willing and able to comply with all study visits and all the protocol requirements, including completing questionnaires., Patient (and caregiver/legal guardian) is able to provide written informed consent; assent based on age., Female patient of childbearing potential must have a negative pregnancy test prior to randomization.
Exclusion Criteria
- •Patient has a clinically significant skin disease other than EBS (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or a vascular disorder associated with cutaneous erosions/ulcerations, that may confound assessments of efficacy or safety., Patient has been treated with any investigational drug or device within 30 days or 5 half-lives, whichever is longer, prior to Visit 2 (Day 1/Baseline A)., Patient has a history of allergy or hypersensitivity to any component of study medications, including diacerein or rhein., Patient is pregnant or breastfeeding/lactating., Patient has a planned or anticipated major surgical procedure or other activity that would interfere with their ability to comply with protocol requirements., Patient has a clinically significant underlying medical condition, psychiatric condition (such as major depressive or psychotic disorder, severe intellectual disability, or alcohol or drug use disorder), or requires concomitant medication that based on the investigator’s judgement may impair evaluation of the Treatment Area or exposes the patient to an unacceptable risk by study participation., Patient has used any diacerein-containing product within 6 months prior to Visit 2 (Day 1/Baseline A)., Patient has had a cutaneous infection in the Treatment Area or use systemic antibiotics within 7 days prior to Visit 2 (Day 1/Baseline A) and prior to Visit 7 (Week 16/Baseline B)., Patient has uncontrolled diabetes mellitus (HbA1c = 6.5%), hepatic enzyme abnormalities (alanine aminotransferase or aspartate aminotransferase >2.5 the upper limit of normal (ULN), or total bilirubin >2.0x ULN), or renal abnormalities (estimated glomerular filtration rate [eGFR]< 30 ml/min/1.73 m2) during the Screening period., Patient has a current malignancy, or a history of treatment for a malignancy within 5 years (with the exception of treated non-melanoma cutaneous malignancy e.g., surgically resected with clear margins) prior to Visit 2 (Day 1/Baseline A)., Patient is treated with protocol-excluded topical therapies within 2 weeks prior to Visit 2 (Day 1/Baseline A) that might influence the assessment of the Treatment Area throughout the study period., Patient has been treated with topical or systemic corticosteroids within 4 weeks and with other immunosuppressive/immunomodulatory therapies or chemotherapy within 8 weeks prior to Visit 2 (Day 1/Baseline A) and prior to Visit 7 (Week 16/Baseline B)., Patient has been treated with an approved or investigational biologic anti- inflammatory therapy (such as monoclonal antibodies that target to modulate the immune responses) within 8 weeks prior to Visit 2 (Day 1/Baseline A).
Investigators
Similar Trials
Active, not recruiting
Not Applicable
An International, Multicenter, Randomized, Double-blind, Parallel-group, Placebo-controlled, Active-controlled Study of the Efficacy and Safety of Sustained-release Quetiapine Fumarate (Seroquel SR™ ) in the Treatment of Generalized Anxiety Disorder (SILVER Study) - Silver StudyGeneralised Anxiety Disorder (GAD)EUCTR2005-005054-46-DKAstraZeneca AB805
Active, not recruiting
Phase 1
An International, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Once Daily Administration of Two Strengths of Ciprofloxacin for Inhalation Compared with Placebo for Inhalation in the Management of Pseudomonas aeruginosa in Patients with Non Cystic Fibrosis Bronchiectasis - N/Aon-cystic fibrosis bronchiectasisMedDRA version: 12.0Level: LLTClassification code 10006446Term: Bronchiectasis NOSEUCTR2009-014412-35-DEAradigm Corporation96
Active, not recruiting
Phase 3
DARE-19 (Dapagliflozin in Respiratory failure in patients with COVID-19)CTRI/2020/09/027829Saint Lukes Mid America Heart Institute
Active, not recruiting
Not Applicable
An International, Multicenter, Randomized, Double-Blind Study of Vorinostat (MK-0683) or Placebo in Combination with Bortezomib in Patients with Multiple MyelomaMedDRA version: 9.1Level: LLTClassification code 10028228Term: Multiple myelomamultiple myelomaEUCTR2008-003752-30-GRMerck & Co., Inc742
Active, not recruiting
Phase 1
An International Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)Epidermolysis Bullosa Simplex (EBS)MedDRA version: 20.0Level: PTClassification code 10014989Term: Epidermolysis bullosaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2016-004427-24-GBCastle Creek Pharmaceuticals, LLC54