Study Designed to Evaluate the Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1...
KPPP1Pachyonychia1 moreIn this phase 1 open label study, up to 10 eligible patients with type I punctate palmoplantar keratoderma or pachyonychia congenital will be recruited to be treated twice daily, for 12 weeks, with 1% topical KM-001. Treatment safety and efficacy will be assessed in the clinic on Days 7, 28, 42, 63and 84 after initiation of treatment. In between visits, safety will also be assessed by phone on Days 14, 21, 35, 49, 56, 70 and 77 of treatment. At the in-clinic visits, treatment efficacy (lesion clearance - IGA) will also be assessed. PK blood samples will be collected on Days 0, 7, 84 and One week after the end of treatment (EoT) visit, patients will return to the clinic for final safety, efficacy and PK evaluations.
Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC
Punctate Palmoplantar Keratoderma Type 1Pachyonychia CongenitaThis Phase 1b, open-label, single-center, prospective trial will be assessing the safety, tolerability, and efficacy of topical KM-001 1% in patients with PPPK1 or PC diseases. Patients will be provided jars of 30 g KM-001 1% and instructed to apply the cream twice daily on the plantar surfaces, for 84 consecutive days. Safety (AEs, blood work [at specific visits], vital signs), tolerability, and efficacy parameters (overall lesion improvement) will be assessed during in-clinic visits (Days 1 [enrolment], 7, 28, 42, 63, 84 [EoT], 112 [follow-up]). Safety and treatment compliance assessment will be done by phone calls on Days 14, 49, 70, and 98 (follow-up). PK samples will be collected to assess plasma levels of KM-001 on Screening, Day 112 & ET: any time during the visit. Day 7 and Day 84 (EoT), at 1 h, 2 h, 3 h, 6 h (+15 min) post-dose. Days 28 and 42 visit: 1 sample after the first dose, before the second dose, as late as possible in the visit. The patient will complete a patient-reported diary, consisting of treatment compliance and self-assessments for efficacy. Follow up- 2 weeks after EoT by phone call, and 4 weeks after EoT in clinic visit.
FARD (RaDiCo Cohort) (RaDiCo-FARD)
Inherited Epidermolysis BullosaIchthyosis7 moreThe goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical) for patients and/or families. Two types of indicators will be used to reach this objective : an individual burden score calculated based on a burden questionnaire created specifically, approved and designed to understand the tendency to changes in care and lifestyles. The burden questionnaire should be used by patients and/or their family themselves in self-assessment. a descriptive analysis of all resources (medical and non-medical) used by the family unit to manage the disease.
The Evaluation of Oral Acitretin in the Treatment of Psoriasis, Cutaneous Disorders of Keratinization,...
Basal Cell CarcinomaKeratosis Palmaris et Plantaris1 moreThis is a continuing study which evaluates the long-term safety and efficacy of oral acitretin in an open manner in the treatment of psoriasis, cutaneous disorders of keratinization, multiple basal cell carcinomas and other retinoid responsive diseases.
Telangiectatic Palmoplantar Keratoderma in Systemic and Subacute Lupus Erythematosus
Systemic Lupus ErythematosusLichen PlanusPalmoplantar keratoderma (PPK) associated to livid telangiectatic erythema during systemic lupus erythematosus (SLE) and subacute cutaneous lupus erythematosus (SCLE) is a rare phenomenon seldom reported in literature. The investigators hypothesize that clinic-immunologic assessment and detailed investigation of cutaneous biopsy specimen of PPK and erythema of patients suffering from SLE and SCLE could lead to determine more precisely nosological settings of this injury. Report the different therapeutics with efficacy assessment could be helpful to highlight useful treatment for these patients.