New Brighton

Researchers are evaluating the safety and pharmacokinetics of QTORIN rapamycin 3.9% anhydrous gel, called PTX-022, in adults with Pachyonychia Congenita PC who have specific keratin gene mutations and who previously participated in the PALV-05 VAPAUS trial. This open-label treatment study is conducted at multiple centers to better understand long-term exposure effects of the gel in this patient group. Participants receive the study drug PTX-022 applied as a gel, with the research focusing on monitoring safety and how the drug moves through and is processed by the body over time. The study does not have a placebo group or masking, and treatment details are based on prior enrollment and dosing established in earlier trials. Participants are monitored for treatment-emergent adverse events over six months, with pharmacokinetic measurements taken before dosing and at 12 and 24 hours after application. The trial assesses drug safety and how the body absorbs and metabolizes the gel during this period, helping researchers understand long-term use implications.

Alopecia areata AA is a condition where the immune system attacks hair follicles, causing hair loss mainly on the head and face but possibly on other body parts. This research evaluates the safety, effectiveness, and tolerance of upadacitinib, an approved drug, in adolescents and adults with severe AA. The study is a Phase 3 randomized, placebo-controlled, double-blind trial enrolling about 1500 participants worldwide. Participants are randomly assigned to one of three groups receiving different treatments two doses of upadacitinib or placebo. In initial periods, some may switch from placebo to upadacitinib based on their Severity of Alopecia Tool SALT score. Those completing early parts may enter an extension phase receiving upadacitinib for up to 108 weeks. Treatment involves taking oral tablets once daily for up to 160 weeks, with possible re-randomization at Weeks 24 and 52. Throughout the study, participants attend regular clinic visits for medical assessments, blood tests, side effect monitoring, and questionnaires to track treatment effects. Researchers measure hair loss improvement using SALT scores and record adverse events over approximately 164 weeks. Participants are followed for up to 30 days after their last dose for safety monitoring.

Researchers are evaluating the efficacy and safety of ORKA-002 in adults with moderate-to-severe plaque psoriasis. This multicenter, randomized, double-blinded, placebo-controlled study aims to find the best induction dosing regimen of ORKA-002. Approximately 160 adult participants with this skin condition will take part in the Phase 2 trial. Participants will be randomly assigned to receive one of three different induction dosing regimens of ORKA-002 or a placebo. Both ORKA-002 and placebo are given by subcutaneous injection. The study includes three periods Screening, Induction, and Post-treatment Follow-up. During the study, participants will undergo assessments including evaluations of skin severity using PASI and IGA scores. Researchers will monitor treatment-emergent adverse events through Week 48. The primary outcome is the proportion of participants achieving complete clearance of psoriasis at Week 16. The total participation time includes screening, treatment, and follow-up periods to assess safety and treatment response.

Researchers are evaluating the long-term safety of Deucravacitinib compared to Ustekinumab in adults with moderate-to-severe plaque psoriasis. This Phase 3b4 study focuses on cardiovascular events and other health outcomes over an extended period, aiming to understand how these treatments impact patients with psoriasis who have cardiovascular risk factors. The study is led by Bristol-Myers Squibb and involves random assignment of participants to either treatment. Participants receive either Deucravacitinib or Ustekinumab at specified doses on scheduled days. The study is open-label, meaning both participants and researchers know which treatment is being given. This trial runs for up to 5 years, during which patients are monitored for cardiovascular safety and other health events related to their psoriasis treatment. Throughout the study, participants undergo regular assessments to track major cardiovascular events like heart attacks, strokes, and hospitalizations, as well as monitoring for infections, cancer, and treatment side effects. Researchers collect data on liver function and lipid levels up to 60 days after the last dose. The long-term follow-up helps evaluate safety and health outcomes over several years, with study activities continuing until early 2031.

Researchers are establishing a Research Sample Repository to collect blood samples related to histocompatibility and hematopoietic cell transplantation HCT or other cellular therapies. This repository supports research aimed at understanding molecular factors affecting transplant outcomes, studying tissue type distributions, and evaluating the success of transplantation and supportive care in marrow toxic injuries. The research includes anonymous studies and aims to facilitate broader scientific investigations in these areas. The repository accepts samples from four main groups donors of hematopoietic cells or other cellular therapies, cord blood units used in treatment, recipients of hematopoietic cell transplants or cellular therapies, and patients treated for marrow toxic injuries. Samples come from donors who have donated or plan to donate cells, cord blood units infused at treatment centers, transplant or therapy recipients, and marrow toxic injury patients receiving various treatments including supportive care or stem cell transplants. Participants contribute samples that are made available to researchers for various analyses including genomic and biomolecular studies. The primary goal is to provide blood samples for research related to histocompatibility and transplantation outcomes. Participation includes adults and children with or without decision-making capacity. The repository facilitates access to these samples for approved research, supporting studies from multiple centers and investigators over an extended period.

Researchers are evaluating the safety and effectiveness of VDPHL01, an investigational oral drug, in female subjects with Androgenetic Alopecia AGA, a genetic condition causing hair loss due to an excessive response to hormones called androgens. This Phase 3, multi-center, double-blind study aims to better understand treatment outcomes for women aged 18 to 65 with mild to moderate AGA. The study is sponsored by Veradermics, Inc. and includes about 13 months of participation with 11 scheduled visits. Participants will be randomly assigned to one of several groups receiving different regimens of VDPHL01 or placebo. Some groups take VDPHL01 twice daily BID for the entire 12 months, while others begin with placebo or a combination of VDPHL01 once daily QD and placebo before switching treatments after 6 months. The study maintains a double-blind design, meaning neither participants nor researchers know who receives the active drug or placebo during the first 6 months. Throughout the study, participants will attend visits for screening, baseline assessment, and follow-ups at weeks 2, months 1, 2, 4, 6, 8, 10, 12, and 13. Researchers will evaluate hair counts in target areas at 6 months and participants own assessment of treatment benefit. Participants agree to maintain consistent hair length, style, and color, and will have a small tattoo placed on their scalp for photographic monitoring. Safety and treatment effects will be carefully monitored during the study period.

Researchers are evaluating the efficacy and safety of reformulated Levulan Kerastick aminolevulinic acid HCl 20% combined with photodynamic therapy PDT for treating actinic keratosis AK lesions on the face or bald scalp. This Phase 3, randomized, multi-center, evaluator-blind, vehicle-controlled study involves about 160 adult patients having 4 to 8 mild to moderate AK lesions. The study aims to compare different treatment approaches and incubation times to understand their effects on AK clearance and patient satisfaction. Participants will be assigned to one of four groups based on treatment typeeither the active drug Levulan or a vehicle without active ingredientand one of two incubation times before blue light therapy is applied. Up to two PDT sessions may be given depending on how the lesions respond. The study includes both drug and device interventions, specifically topical solutions and a blue light PDT device. During the study, participants will undergo multiple assessments including evaluations of lesion clearance, changes in lesion count and size, cosmetic response, and patient satisfaction at various time points up to 24 weeks. Safety will be monitored through adverse event reporting, skin reaction assessments, vital signs, and laboratory tests. The main outcome measure is the complete clearance rate of AK lesions at 12 weeks. Participants will be followed closely to assess treatment effects and safety over the study period.

Researchers are evaluating the efficacy and safety of a reformulated Levulan Kerastick solution containing aminolevulinic acid hydrochloride ALA HCl 20%, combined with photodynamic therapy PDT, for treating actinic keratosis AK lesions on the upper extremities. This Phase 3, randomized, multi-center, evaluator-blind, vehicle-controlled study focuses on adults with 4-8 mild to moderate AK lesions on one arm. The study aims to assess complete clearance of AK lesions and other related outcomes including partial clearance, lesion count changes, recurrence, cosmetic response, and patient satisfaction. Participants will be randomly assigned to receive either the active treatment with reformulated Levulan Kerastick plus blue light PDT or a vehicle solution plus blue light PDT. Treatment involves applying the solution with occlusion and incubation followed by exposure to blue light using the BLU-U4170E device. Up to two PDT sessions may be administered depending on lesion clearance. The study includes a control group receiving the vehicle solution to compare outcomes. During the study, participants will undergo evaluations at multiple time points including weeks 4, 8, 12, 20, and 24 to assess lesion clearance, cosmetic response, and patient satisfaction. Safety monitoring includes checks for adverse events, local skin reactions, vital signs, and laboratory tests. The primary outcome, complete clearance rate, will be measured at week 12. The total study duration involves follow-up through week 24 to assess sustained effects and recurrence rates.

Researchers are evaluating the efficacy and safety of ICP-332 in adults aged 18 to 75 years with Prurigo Nodularis PN, a chronic skin condition characterized by itchy nodules. This phase 2, randomized, double-blind, placebo-controlled study aims to investigate different doses of ICP-332 for treating PN, addressing the need for effective therapies in patients with this condition. The trial is sponsored by Beijing InnoCare Pharma Tech Co., Ltd. and uses a parallel study design with multiple groups. Participants are randomly assigned to receive either ICP-332 at doses of 80 mg or 120 mg or a placebo. The study compares these groups over a period of up to 40 weeks, with primary assessment focusing on peak itch intensity using a numeric rating scale from baseline to week 16. Secondary measures include longer-term itch improvement, skin condition scores, quality of life assessments, drug concentration levels, and safety monitoring of adverse events from baseline through week 40. During the trial, participants will undergo regular evaluations including itch severity scales, skin assessments, quality of life questionnaires, and laboratory tests to monitor drug levels and safety. The study tracks outcomes up to 40 weeks to assess both efficacy and tolerability. Participants are expected to adhere to study visits and procedures as scheduled, with safety and treatment response closely monitored throughout the trial duration.