Rancho Santa Margarita

Researchers are studying KB707, a genetically modified herpes simplex type 1 virus designed to trigger an immune response against tumors. This Phase 1/2 open-label trial is focused on adults with locally advanced or metastatic solid tumors, including advanced melanoma, especially those who have progressed on or cannot tolerate standard treatments. The study also explores the safety, tolerability, preliminary effectiveness, and immune effects of KB707 when combined with immunotherapy drugs for melanoma. Participants receive KB707 through injections directly into tumors approximately every three weeks during the dose escalation and initial expansion phases, which are now closed to new participants. In later phases, those with advanced melanoma receive biweekly KB707 injections combined either with Opdualag every four weeks or Keytruda every six weeks. Treatment continues until disease progression, unacceptable side effects, worsening symptoms, maximum response, patient choice, investigator decision, or study termination. During the study, participants undergo evaluations including assessments of adverse and serious adverse events for up to 36 months. Researchers monitor safety, tolerability, immune response, and preliminary efficacy through clinical exams, imaging, and laboratory tests. The goal is to understand how KB707 alone and with immunotherapy acts in treating advanced solid tumors and melanoma over the treatment and follow-up periods.

Researchers are studying KB803, an ophthalmic suspension containing a modified herpes simplex virus that expresses human collagen VII protein, in children and adults who have recurrent corneal abrasions caused by dystrophic epidermolysis bullosa (DEB). This Phase 3, double-blind, randomized, placebo-controlled crossover trial aims to evaluate the safety and effectiveness of KB803 compared to a matched placebo in reducing corneal abrasion symptoms in this population. Participants who have been in the sponsor's natural history study for at least 12 weeks and meet eligibility requirements are randomly assigned to receive either KB803 or placebo eye drops three times a week for 12 weeks. After this period, they switch to the other treatment for another 12 weeks. The study drug is administered at home by a trained caregiver or designee. Investigators regularly contact participants or their guardians to track any side effects or changes in treatments. Throughout the 24-week study, participants or their guardians complete weekly symptom diaries and monthly questionnaires to record corneal abrasion symptoms and their frequency. Researchers assess safety and tolerability and monitor changes in disease severity and symptoms. The study includes careful follow-up to measure the impact of KB803 on corneal abrasion frequency and overall safety in participants with DEB.

Researchers are evaluating the pharmacokinetics and safety of dupilumab in children aged 6 months to under 18 years who have prurigo nodularis, a skin condition characterized by itchy nodules. This Phase 3, multicenter, open-label study aims to understand how the drug behaves in the body and monitor its safety in this young population. The study involves three main periods: a 2 to 4 week screening phase, a 24-week treatment phase where participants receive dupilumab as a subcutaneous injection, and a 16-week post-treatment follow-up phase. Each participant will attend a total of six study visits throughout the approximately 42 to 44 week duration. Dupilumab is given as an injection solution under the skin to assess its effects and safety in managing prurigo nodularis. Participants will undergo regular assessments including blood tests to measure dupilumab levels in the serum from Day 1 to Week 40. They and their caregivers will complete daily electronic symptom diaries to track itch severity and other symptoms. Safety and treatment response will be closely monitored throughout the study and during the follow-up period to gather comprehensive information on the drug's behavior and tolerability in children with prurigo nodularis.

Researchers are conducting a long-term follow-up study to evaluate the safety of gene therapy products developed by Krystal Biotech, Inc. These gene therapies share a common herpes simplex virus type 1 (HSV-1) backbone, and the study focuses on participants who have received at least one dose of these investigational products. The aim is to identify and manage any long-term risks and delayed adverse effects associated with these treatments in people with dystrophic epidermolysis bullosa, including recessive and dominant forms. The study involves annual monitoring of participants for five years following their last study visit from the parent treatment protocol. This observational study will collect data without adding new treatments, focusing on understanding how long the gene therapy products persist in the body and tracking any serious adverse events over time. Participants have already completed or discontinued their initial treatment studies sponsored by Krystal Biotech, Inc. Participants will be asked to provide consent or assent and will be followed with yearly assessments to gather information about their health status and any adverse events. The main outcome measured is the occurrence of serious adverse events during the five-year follow-up. This long-term monitoring helps ensure the continued safety and understanding of these gene therapy products over an extended period.