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, typically on the head and face but possibly anywhere on the body. This research aims to evaluate how safe, effective, and tolerable the drug upadacitinib is for adolescents and adults with severe AA. The study is a Phase 3 randomized, placebo-controlled trial involving about 1500 participants worldwide with severe AA, sponsored by AbbVie. Participants will be randomly assigned to one of three groups receiving either one of two doses of upadacitinib or a placebo. In the initial period, some participants receive placebo for 24 weeks, then based on their hair loss severity measured by the Severity of Alopecia Tool (SALT) score, they may be re-randomized to continue placebo or start one of the upadacitinib doses. Those on upadacitinib continue their assigned dose. Participants who complete the first studies can join an extension study with upadacitinib treatment for up to 108 weeks. Tablets are taken orally once daily for up to 160 weeks. Throughout the study, participants will attend regular hospital or clinic visits for medical assessments, blood tests, side effect monitoring, and questionnaires. Researchers will measure changes in hair loss using the SALT score and track any adverse events during treatment and up to 30 days after the last dose. The study also evaluates other patient-reported outcomes related to hair growth and quality of life over time.

Researchers are evaluating the safety and effectiveness of tapinarof cream, 1%, in young children aged 3 months to less than 24 months who have atopic dermatitis, a skin condition. This global Phase 3 clinical study aims to better understand how well the cream works and how safe it is for this very young age group. Participants will be randomly assigned to apply either tapinarof cream or a vehicle cream (a placebo) once daily to affected skin areas for up to 8 weeks during a double-blind period. After this phase, all participants may use tapinarof cream once daily as needed during an open-label period lasting up to 56 weeks. The study compares the effects of tapinarof cream against the vehicle cream and monitors participants over time. Throughout the study, caregivers and researchers will assess skin improvement using the Investigator Global Assessment for Atopic Dermatitis (vIGA-AD) and the Eczema Area and Severity Index (EASI). Safety will be closely monitored by recording any treatment-related side effects and local skin reactions. The trial includes regular visits and evaluations over the initial 8-week double-blind phase and continued monitoring during the longer open-label phase, which can last up to 56 weeks in total.

Researchers are studying the effects of ORKA-002 in adults with moderate-to-severe plaque psoriasis. This multicenter trial is a randomized, double-blinded, placebo-controlled phase 2 study aiming to find the best induction dose of ORKA-002 to treat this skin condition. The study involves about 160 participants to evaluate both the effectiveness and safety of the medication compared to a placebo. Participants will be randomly assigned to receive one of three different dosing regimens of ORKA-002 or a placebo, with all treatments given by subcutaneous injection. The study includes a screening period to determine eligibility, an induction period where participants receive the assigned treatment, and a post-treatment follow-up period to monitor outcomes and safety. During the trial, participants will undergo assessments including skin evaluations using PASI and IGA scores to measure psoriasis severity and improvement. Safety will be closely monitored through reports of treatment-emergent adverse events up to 48 weeks. The total involvement includes screening, treatment, and follow-up to assess both short- and longer-term effects of ORKA-002.

Researchers are evaluating the safety, tolerability, and effects of a topical cream called RLS-1496 for adults with actinic keratosis (AK) on their arms. The study aims to find out if applying RLS-1496 once daily for 28 days is safe and whether it reduces or clears AK lesions compared to no treatment. The trial involves comparing treated skin on the left forearm with untreated skin on the right forearm. Participants will apply RLS-1496 cream to a designated area on the left forearm daily for 28 days, starting under supervision and then at home. The right forearm remains untreated as a control. After treatment, participants will be followed for 28 more days. The study includes skin biopsies and assessments of disease markers in the skin and blood. During the study, participants will have clinic visits to monitor safety, count AK lesions on both arms, and undergo physical exams, laboratory tests, and skin evaluations. Local skin reactions and treatment effects will be tracked. The total participation time may last up to 85 days, including screening, treatment, and follow-up. About 24 patients are expected to join the study.

Researchers are evaluating the long-term safety of Deucravacitinib compared to Ustekinumab in adults with moderate-to-severe plaque psoriasis who have certain cardiovascular risk factors. This Phase 3b/4 randomized, open-label study aims to assess cardiovascular and other safety outcomes over several years to better understand risks associated with these treatments in this patient group. The study is sponsored by Bristol-Myers Squibb. Participants will be randomly assigned to receive either Deucravacitinib or Ustekinumab at specified doses on designated days. The study is designed to compare these two treatments over a long-term period, including up to five years of follow-up for major cardiovascular events and other health outcomes related to psoriasis treatment. During the study, participants will undergo regular monitoring for cardiovascular events such as heart attacks, strokes, and revascularization procedures, as well as other serious health events including infections and malignancies. Researchers will also track changes in liver function and lipid levels. The total duration of participant involvement can last up to five years, with safety assessments extending up to 60 days after the last dose of study medication.

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 type—either the active drug Levulan or a vehicle without active ingredient—and 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.