Markham

Researchers are evaluating the effectiveness of delgocitinib cream 20 mg/g applied twice daily compared to a cream vehicle in treating adults with mild to severe palmoplantar pustulosis (PPP). This phase 2a, double-blind, two-arm trial focuses on adults diagnosed with PPP, a condition characterized by persistent pustules on the palms and/or soles, lasting more than three months. The study aims to assess skin improvement using the PPP Area and Severity Index (PPPASI) and other clinical evaluations over 16 weeks. Participants will be randomly assigned to apply either delgocitinib cream or the cream vehicle twice daily during a 16-week treatment period. The study involves approximately 9 visits over about 18 weeks in total. The trial is designed to compare the two treatments' effects on PPP severity, with a thorough monitoring process throughout the study period. During the trial, participants will undergo clinical assessments including photographic evaluations to confirm PPP diagnosis, PPPASI scoring to measure skin improvement, and physician global assessments. Safety and treatment adherence will be closely monitored. The main outcome measure is the number of participants achieving at least a 75% improvement in PPPASI score from baseline at week 16. The study also includes safety checks and follow-up to ensure participant well-being throughout the trial.

Researchers are investigating the long-term safety and effectiveness of APG777, a treatment for moderate-to-severe atopic dermatitis (AD), in patients who have already completed an initial APG777 study. This phase 2, multicenter, double-blind study focuses on those who may benefit from extended treatment with APG777 to better understand its ongoing effects and safety over time. The study includes three main periods: a screening visit that occurs at the end of the previous study's maintenance period, an extended treatment period where participants receive APG777 subcutaneous injections every 12 or 24 weeks, and a post-treatment follow-up period. Participants will continue using their chosen non-medicated moisturizer from the previous study throughout this extension. During the study, participants' health and response to treatment will be closely monitored, including tracking any treatment-emergent adverse events for up to three years. Researchers will assess the long-term safety and efficacy of APG777 while ensuring participants remain compliant with study protocols. This ongoing observation aims to provide detailed information on how APG777 affects patients over extended use.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are conducting a global study to understand the impact of moderate to severe alopecia areata (AA), non-segmental vitiligo (NSV), and hidradenitis suppurativa (HS) on adolescents and adults. This study aims to assess the burden these conditions place on patients' quality of life and daily functioning in a large real-world population. The study involves participants diagnosed by a physician with one of the three conditions: AA, NSV, or HS. There are no interventional treatments or medications being tested in this study, as it is observational in nature. Data collection focuses on patient-reported outcomes and measures that evaluate disease severity and its effects. Participants will complete various questionnaires and assessments related to their condition, such as the Alopecia Areata Symptom Impact Scale (AASIS) for AA, the Severity of Alopecia Tool (SALT) for scalp hair loss in AA, the Facial Vitiligo Area Scoring Index (F-VASI) and Vitiligo Quality of Life Score (VitiQoL) for vitiligo, and the Dermatology Life Quality Index (DLQI) and International Hidradenitis Suppurativa Severity Scoring System (IHS4) for HS. These tools help researchers understand how symptoms affect quality of life and disease severity. The study collects information up to the day of the study visit.

Researchers are evaluating the safety and effectiveness of baricitinib, a medication taken by mouth, for treating severe or very severe alopecia areata, a type of hair loss, in children aged 6 to less than 18 years. This Phase 3 study involves children and adolescents who have had alopecia areata for at least one year and are experiencing a current episode lasting at least six months with significant hair loss. The study aims to see how well baricitinib works compared to a placebo. The study is divided into four distinct periods: a 5-week screening period to determine eligibility, a 36-week double-blind treatment period where participants receive either baricitinib or placebo, an approximately 2-year long-term extension period for continued treatment, and a 4-week post-treatment follow-up period to monitor participants after stopping the medication. Both baricitinib and placebo are administered orally. Participants will undergo various assessments throughout the study, including measuring the severity of hair loss using the Severity of Alopecia Tool (SALT) score. The main outcome is the percentage of participants achieving a SALT score of 20 or less after 36 weeks of treatment. Safety and pharmacokinetics of baricitinib will also be monitored during the study. The total participation may last over two years, including treatment and follow-up phases.

Researchers are evaluating the safety and effectiveness of KT-621, an oral drug, in adults and adolescents aged 12 to 75 years with moderate-to-severe atopic dermatitis (AD), a common form of eczema. This phase 2b study aims to assess how well KT-621 reduces the severity and extent of AD, its safety and tolerability, how the drug behaves in the body, and the body's response to the drug. The study includes a 16-week double-blind, placebo-controlled period followed by a 52-week open-label phase. Participants will receive either KT-621 or a matching oral placebo during the initial 16-week double-blind period. Following this, all participants have the option to enter a 52-week open-label period where they receive KT-621. During these phases, participants will continue their regular use of moisturizers twice daily and adhere to the treatment plan. The study is conducted across multiple centers and includes dose-ranging to evaluate different doses of KT-621. Throughout the study, participants will have scheduled visits for assessments including clinical evaluations of eczema severity using the Eczema Area and Severity Index (EASI) score, which measures changes from baseline through week 16. Other evaluations include questionnaires, laboratory tests, and adherence monitoring via an electronic diary. Safety and tolerability are closely monitored, and participants are expected to comply with all study procedures over the entire duration of the trial, including both the double-blind and open-label periods.

Researchers are evaluating pitolisant in a global, Phase 3 study involving patients with Prader-Willi syndrome who are 6 years of age or older. The study aims to assess whether pitolisant can reduce excessive daytime sleepiness (EDS) and also evaluate its effects on irritability, disruptive behaviors, hyperphagia, and other behavioral problems such as social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech. The study includes up to a 45-day screening and baseline period, followed by a double-blind treatment period where patients are randomly assigned to receive either pitolisant or a placebo once daily. In-person visits during this period occur on Day 29, Day 57, and Day 77. After this, patients may choose to enter an optional open-label extension period with visits on Day 113, Day 260, and Day 441. Follow-up visits occur 15 and 30 days after the final dose in both the double-blind and extension periods. Participants will have assessments to measure the severity of EDS using patient-reported outcomes at the start and end of the double-blind period. Caregivers will complete behavioral evaluations, and researchers will monitor safety and treatment effects throughout. Total participation may last over a year if the optional extension is chosen, with continued follow-up after treatment ends.

This trial investigates treatments for children aged 2 to less than 12 years with moderate to severe atopic dermatitis, a skin condition causing rash and itching due to inflammation. It compares oral upadacitinib, a drug approved for patients 12 years and older, with subcutaneous dupilumab, focusing on safety, adverse events, and changes in disease activity. The study is phase 3, open-label, and efficacy-assessor-blinded, enrolling about 675 participants worldwide who require systemic anti-inflammatory treatment beyond topical therapies. Participants will be randomly assigned to receive upadacitinib daily as oral tablets or oral solution for 160 weeks, or dupilumab by injection according to its approved dosing every 2 or 4 weeks for 52 weeks. Participants are stratified by disease severity, age, and previous treatment response. After completing treatment, follow-up visits occur for 30 days after upadacitinib and at least 12 weeks after dupilumab. The trial may involve more treatment visits than standard care. Throughout the study, participants attend regular hospital or clinic visits for clinical assessments, blood tests, and questionnaires to monitor disease severity and side effects. Researchers measure the percentage of participants achieving significant improvement in eczema severity by week 16 and track adverse events up to about week 172. This careful monitoring helps evaluate the safety and efficacy of the treatments over the long term.

Researchers are evaluating the safety and effectiveness of upadacitinib in treating adults and adolescents with moderate to severe hidradenitis suppurativa (HS) who have not responded to or cannot tolerate anti-tumor necrosis factor (TNF) therapy. HS is an inflammatory skin disease causing painful lesions in areas such as the underarms, groin, and anal/genital regions. This phase 3, double-blind study involves approximately 1328 participants worldwide and aims to monitor disease activity and adverse events over time. Participants will receive oral tablets of either upadacitinib or placebo once daily during Period 1 and Period 2, lasting a total of 36 weeks. In Period 1, participants are randomly assigned to one of two treatment groups, with a 50% chance of receiving placebo. Based on results and placement in earlier periods, participants enter Period 2 with six potential treatment groups. Eligible participants from these periods may continue into Period 3, a long-term extension lasting 68 weeks, continuing the same daily oral treatment. Following the treatment periods, participants will be followed for approximately 30 days. During the study, participants will attend regular outpatient visits for medical assessments, monitoring for side effects, and completing questionnaires. Researchers will measure the percentage of participants achieving a clinical response called HiSCR 50 from baseline to week 16 and track adverse events up to approximately week 108. The study may require a higher treatment commitment compared to usual care, but provides close monitoring of disease activity and safety throughout all study phases.

Researchers are evaluating the drug levels, effectiveness, and safety of Deucravacitinib in children aged 4 to under 18 years who have moderate to severe plaque psoriasis. The study includes two age groups: Cohort 1 for ages 12 to under 18, and Cohort 2 for ages 4 to under 12. Each cohort has two parts, with Part A focusing on determining the appropriate dose levels of Deucravacitinib, and Part B assessing how well two selected dose levels work and their safety. A long-term extension phase lasting up to five years will monitor the ongoing safety and tolerability of the medication in young participants who completed the initial study parts. Participants receive Deucravacitinib or a matching placebo at specified doses on set days. Part A evaluates drug levels to select doses for Part B, which then measures efficacy and safety of the chosen doses in pediatric patients with moderate to severe plaque psoriasis. Following Parts A or B, participants may enter the long-term extension period to continue safety and tolerability assessments over several years. Throughout the study, researchers will measure drug concentrations in the blood at Week 2 and assess clinical improvements at Week 16 using the Psoriasis Area and Severity Index and Physician's Global Assessment scores. Safety monitoring includes tracking adverse events, serious adverse events, and growth measures such as weight, height, and sexual maturation stages for up to 316 weeks. This comprehensive evaluation helps understand how the drug works and its impact on children's health over both short and long periods.