This is a randomized, double-blind, parallel group, vehicle-controlled phase to evaluate the efficacy and safety of diacerein 1% ointment applied topically once daily for 8 weeks for the treatment of adult and pediatric (age ≥ 6 months) patients with generalized EBS. The duration of study participation is anticipated to be approximately ~16 to 20 weeks per patient consisting of a Screening Period of up to 4 weeks, a Treatment Period of 8 weeks and a No Treatment Follow-up Period of 8 weeks. Patients that complete this portion of the study will be eligible to participate in an open-label, 24-week extension phase to evaluate the long-term safety of diacerein 1% ointment for the treatment of generalized EBS.

This study aims to evaluate the effectiveness of ruxolitinib cream compared to a placebo (vehicle) cream in treating vitiligo in children aged 2 to 12 years. The vehicle cream looks identical to the ruxolitinib cream but contains no active medication. By comparing these two treatments, researchers hope to determine whether ruxolitinib is more effective than the placebo in improving facial and body vitiligo symptoms. This study could provide important insights into new treatment options for young children affected by this condition.

The primary purpose of this study is to assess the efficacy and safety of ruxolitinib 1.5% cream applied twice daily in participants with mild to moderate HS (Hurley Stage I or II) without draining tunnels.

This multicenter, randomized, placebo-controlled, double-blind study aims to evaluate the efficacy of anifrolumab subcutaneous (SC) injection compared to a placebo in reducing overall disease activity in participants with moderate to severe idiopathic inflammatory myopathies (IIM), specifically polymyositis (PM) and dermatomyositis (DM), while they receive standard care treatment.

M1095-HS-303 is a Phase 3, multicenter, open-label extension study to evaluate the long-term safety, tolerability, and efficacy of sonelokimab in participants with moderate to severe HS who were previously enrolled in a parental study.
Rollover to this OLE study will occur at the time of the End-of-Treatment (EOT) visit of the parental study. Upon enrollment into this study, each participant can continue to receive sonelokimab 120 mg every 4 weeks (Q4W) for up to 2 years, with a safety follow-up (SFU). Visit 8 weeks after the last dose of study treatment. Following successful training and supervision, sonelokimab will be self-administered by the participant (or caregiver if required) at home and at the study center.

The purpose of this clinical research study is to learn more about the use of an investigational medicine, called brepocitinib, for the treatment of Lichen Planopilaris (LPP). The study will also look at how safe and effective brepocitinib is and will monitor the long-term safety of brepocitinib when taken for a period up to 52 weeks.

The purpose of this study is to measure the efficacy and safety of KP-001 compared to placebo in patients with common venous malformations (VM), common lymphatic malformations (LM), or KTS/CLOVES syndrome. This phase 3, double-blind, randomized, placebo-controlled, parallel-group study will take place at multiple sites across North America. Vascular malformations like VM, LM, and KTS/CLOVES syndrome are serious, rare diseases with significant unmet medical needs. The study includes a 24-week double-blind treatment period with either KP-001 or placebo, followed by an open-label phase where all patients receive KP-001 up to Week 52. Patients weighing 40 kg or more will receive 100 mg of KP-001 once daily after breakfast, while those weighing less than 40 kg will receive a reduced dose based on their body weight.

This study will compare the efficacy and safety of SD-101-6.0 cream with SD-101-0.0 (placebo) in approximately 80 patients with Epidermolysis Bullosa (EB), including Simplex, Recessive Dystrophic, and JEB-nH subtypes. Patients will apply SD-101-6.0 or placebo topically once daily to the entire body for 60 days. Each patient will have one target wound that is 21 days or older and 10–50 cm² in size selected at baseline. The primary goal is to determine how many patients in each treatment group achieve complete closure of the target wound (skin re-epithelialization without drainage) by Month 1.

Patients will return for follow-up visits around Days 14, 30, and 60 to assess wound healing using the ARANZ SilhouetteStar™ device, with the target wound measured at each visit until it is documented as closed. Secondary assessments include changes in total wound burden based on body surface area (BSA), itching, and incidence of skin infections across the entire body. Itching will be evaluated at multiple time points but will not be recorded as an adverse event. Patients who complete this trial may be eligible to enroll in an open-label extension study (SD-008) to receive active treatment. Safety will be monitored through adverse event reporting, tolerability assessments, and physical examinations, with urine pregnancy testing performed at the investigator's discretion.

Epidermolysis Bullosa is a genetic disorder manifesting typically at birth comprising several genetic subtypes that manifest as blistering or erosion of the skin in response to little or no apparent trauma. This clinical study is being conducted for research purposes. The cream being studied has allantoin as the active ingredient and the aim of this study is to assess the long-term safety of this cream when applied to the skin of participants with Epidermolysis Bullosa. This study is an open label extension to study SD-007. The study will be conducted by the study doctor, in other words, the physician responsible for the clinical study at your child's study site. Your child's participation in the clinical study is expected to last for approximately 12 months and approximately eighty (80) children will participate in this study. There will be a total of five (5) study visits over the course of one year. Study visits 1, 3 and 5 will be in person and visits 2 and 4 will be telephone visits.