Rancho Santa Margarita

Researchers are evaluating KB707, a genetically modified herpes simplex type 1 virus designed to stimulate the immune system against tumors, in adults with advanced or metastatic solid tumors, including melanoma. This Phase 12, open-label study aims to assess the safety, tolerability, preliminary efficacy, and immune effects of KB707 alone and in combination with immune checkpoint inhibitors in subjects who have progressed on or cannot tolerate standard therapies. Participants in initial dose escalation and expansion cohorts receive intratumoral KB707 injections approximately every three weeks. Later cohorts evaluate KB707 given biweekly combined with either Opdualag, a dual immune checkpoint inhibitor, or Keytruda, an immune checkpoint inhibitor dosed every 4 or 6 weeks respectively. Treatment continues until disease progression, unacceptable side effects, or other study or participant decisions. Throughout the study, subjects undergo assessments for adverse events, serious adverse events, and overall response rate up to 36 months. Researchers monitor safety, tolerability, and tumor response while participants receive regular intratumoral injections and immune therapies. The study includes ongoing follow-up to evaluate treatment effects and participant health over an extended period.

Researchers are evaluating KB803, an ophthalmic suspension containing a modified herpes simplex virus expressing human collagen VII protein, in patients with dystrophic epidermolysis bullosa DEB who experience recurrent corneal abrasions. This Phase 3, double-blind, randomized, placebo-controlled crossover study aims to assess the safety and effectiveness of KB803 compared to a matched placebo in both children and adults with DEB. The study is sponsored by Krystal Biotech, Inc. Participants who have been in the Sponsors natural history study for at least 12 weeks and meet other criteria will be randomly assigned to receive either KB803 or placebo eye drops three times a week for the first 12 weeks. Then, they will switch to the alternate treatment for another 12 weeks. The eye treatments are given at home by a trained person, with monitoring by the study team. During the 24-week study, researchers will monitor safety and tolerability, track the frequency of corneal abrasion symptoms, and assess eye pain. Participants or their guardians will keep weekly symptom diaries and complete monthly questionnaires about symptoms and disease severity. Study staff will regularly check for any side effects or changes in treatment. The total participation lasts 24 weeks with ongoing safety and symptom evaluations.

Researchers are evaluating the pharmacokinetics and safety of Dupilumab in children and adolescents aged 6 months to less than 18 years with prurigo nodularis, a chronic skin condition characterized by itchy nodules. This Phase 3, multicenter, open-label study aims to better understand how Dupilumab behaves in the body and its safety profile in this young population. Participants will receive Dupilumab administered by subcutaneous injection, with dosing based on their weight and age. The study includes three periods a screening period lasting 2 to 4 weeks, a treatment period of 24 weeks during which Dupilumab is given, and a post-intervention follow-up period of 16 weeks. Each participant will have a total of 6 planned study visits over approximately 42 to 44 weeks. Throughout the study, participants will complete daily symptom diaries and undergo evaluations to measure Dupilumab concentration in the blood from Day 1 to Week 40. The study will also monitor any treatment-emergent or serious adverse events and check for the development of antibodies against Dupilumab. Safety and effectiveness assessments will continue during the follow-up period to ensure comprehensive monitoring of participant health and treatment response.

Researchers are studying Epidermolysis Bullosa EB in children aged 1 month to 12 years with Simplex, Recessive Dystrophic, or Junctional non-Herlitz JEB-nH types. This Phase 3, multi-center trial compares the safety and effectiveness of SD-101-6.0 cream, a topical treatment, versus placebo applied to wounds. The study aims to evaluate wound healing and itching over two months. Participants will apply either SD-101-6.0 cream or placebo once daily to the entire body for 60 days. Each patient has one target wound between 10 and 50 cm2, present for at least 21 days. The wound is photographed and measured with a specialized device at baseline and follow-up visits. If the wound heals earlier, no further assessments are done on it. Patients will attend four visits over two months. During the study, researchers will assess wound closure, changes in wound burden, itching, and body surface area affected by wounds. Assessments include photographic documentation, wound measurements, and itch evaluations at each visit. After completing the study, participants may continue to receive SD-101-6.0 cream at home in an open-label extension. The entire study period involves minimal visits and limited assessments to reduce burden on patients.

This research aims to evaluate the long-term safety of gene therapy products developed by Krystal Biotech, Inc. These products share a common herpes simplex virus type 1 HSV-1 backbone and have been given to participants in previous studies. The study focuses on participants with conditions such as dystrophic epidermolysis bullosa, including its recessive and dominant forms, who have received at least one dose of the investigational gene therapy. The study is observational and involves annual follow-up visits for up to five years after the participants last visit in their previous treatment study. These follow-up visits are designed to monitor and identify any delayed adverse events and to better understand how long the gene therapy products persist in the body. No new treatments are given during this follow-up period. Participants will be asked to provide consent and will undergo yearly safety assessments to track serious adverse events over five years. The study collects data on the participants health and any side effects that may appear long after the initial gene therapy treatment. This long-term monitoring helps ensure ongoing safety and provides valuable information about the gene therapy products.