Redwood City

Researchers are evaluating the safety and dosing of two types of autologous Chimeric Antigen Receptor T (CART) cell therapies, DSG3-CAART and CABA-201, in people with active pemphigus vulgaris (PV), an autoimmune disorder causing painful blisters on skin and mucous membranes. This phase 1/2, open-label study focuses on patients whose disease is not well controlled by standard treatments. The study aims to find safe doses and schedules for these investigational cell therapies and assess their potential to induce lasting remission. The study has two parts. The first, now closed to enrollment, tested different doses and infusion schedules of DSG3-CAART, given alone or with immunosuppressive drugs, to find the best tolerated treatment for mucosal PV. The second part is an ongoing sub-study evaluating CABA-201, given as a single intravenous infusion at escalating doses with or without preconditioning, in patients with mucosal-dominant or mucocutaneous PV. Both therapies are being studied for their safety and ability to produce clinical responses. Participants undergo screening to confirm active PV and antibody positivity. During the study, they receive the assigned CART cell therapy and are closely monitored for adverse events up to 3 months or 28 days after infusion for the sub-study. Researchers collect clinical assessments, antibody tests, and safety data to evaluate treatment effects and tolerability. The study includes careful follow-up to track outcomes and potential side effects over time.

Researchers are evaluating the safety and preliminary effectiveness of CTX112, a CD19-directed CAR T cell immunotherapy, in adults with difficult-to-treat autoimmune diseases such as systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM). This Phase 1, open-label, multi-center study focuses on adults aged 18 to under 70 years who have refractory forms of these autoimmune conditions. CTX112 is made from healthy donor T cells genetically modified using CRISPR-Cas9 technology to target CD19. Participants receive CTX112, an allogeneic T cell therapy prepared outside the body and designed to modify immune responses. The study involves ascending doses to assess safety and gather initial data on treatment effects. The treatment is administered as an infusion. Up to 80 subjects may take part in this study to help understand how well patients tolerate the therapy and its preliminary impact on disease. During the study, participants will undergo various assessments including lab tests and disease activity evaluations. Safety is closely monitored from the time of CTX112 infusion through 28 days afterward. Researchers will track adverse effects and disease symptoms to evaluate both safety and early effectiveness. Participants must comply with scheduled visits, laboratory tests, and other study procedures over the study period to support these evaluations.

Researchers are evaluating efruxifermin (EFX) in adults aged 18 to 80 who have compensated cirrhosis caused by nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of EFX in improving liver health and delaying disease progression in this population. The study focuses on subjects with advanced liver fibrosis (stage 4) but without liver decompensation. Participants are randomly assigned to receive either efruxifermin or a placebo, both administered by subcutaneous injection. The study includes two cohorts: Cohort 1 requires biopsy confirmation of liver fibrosis and specific metabolic features, while Cohort 2 allows biopsy or non-invasive diagnosis. Treatment and observation continue over an extended period to evaluate changes in liver fibrosis and clinical events. During the study, researchers will monitor the time until significant clinical events such as disease progression or liver decompensation occur, with a follow-up of up to five years. For Cohort 1, the proportion of participants showing improvement in fibrosis without worsening steatohepatitis will be assessed at 96 weeks. Participants will undergo regular evaluations including clinical assessments and laboratory tests to track liver function and safety throughout the study period.

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and anti-tumor activity of DR-01 in adults with large granular lymphocytic leukemia or various types of cytotoxic lymphomas. This is a first-in-human, multicenter Phase 1/2 study aiming to find appropriate dosing and to assess how well DR-01 works in these populations. DR-01 is a non-fucosylated human immunoglobulin G1 monoclonal antibody given to participants. The study includes multiple parts: Part A focuses on safety and finding the best dose by monitoring adverse events, dose-limiting toxicities, and drug effects for up to six months. Part B evaluates overall response rates, measuring how many participants achieve complete or partial response over up to 24 months. Participants will undergo various assessments including monitoring for adverse events using CTCAE criteria, pharmacokinetic and pharmacodynamic tests, and disease-specific response evaluations. Tissue samples or biopsies may be collected if needed. Safety follow-up can last up to 25 months, with ongoing evaluation of treatment effects and side effects. The study requires participants to meet specific disease and health criteria and to comply with study procedures throughout the treatment and observation periods.

Idiopathic Hypersomnia (IH) is a condition where adults feel very sleepy during the day, especially in the morning, even after getting plenty of sleep at night. They may find it hard to wake up and stay alert, affecting their ability to focus, think clearly, and carry out daily activities. This study is designed to evaluate the safety and tolerability of TAK-360, a drug that mimics orexin, a brain chemical that helps keep people awake, in adults with IH. Researchers also want to find out if TAK-360 helps improve wakefulness and determine the appropriate dose needed. Participants will be randomly assigned to receive either TAK-360 tablets or matching placebo tablets, which look the same but contain no active medicine. This study is a Phase 2 trial with a double-blind design, meaning neither the participants nor the researchers know who receives TAK-360 or placebo, to fairly assess the treatment's effects. The study focuses on finding the right dose of TAK-360 and monitoring its safety and tolerability. During the study, researchers will track treatment-emergent adverse events up to week 8 to evaluate safety. Participants will be monitored closely for how well they tolerate TAK-360 and any side effects. The study includes adults aged 18 to 70 years with a diagnosis of IH. Researchers will collect various health information to understand the drug's effects and ensure participant safety throughout the trial.

Narcolepsy Type 2 (NT2), also known as narcolepsy without cataplexy, is a lifelong condition causing excessive daytime sleepiness despite normal nighttime sleep. People with NT2 may suddenly fall asleep, struggle to stay awake, or have poor nighttime sleep, leading to problems with attention, memory, and daily activities like driving or working. This research evaluates the safety, tolerability, and effectiveness of TAK-360, a drug that mimics orexin, a brain chemical that helps maintain wakefulness, in adults with NT2. Participants in this phase 2 trial are randomly assigned to receive either TAK-360 tablets or matching placebo tablets. The study uses a double-blind design, meaning neither participants nor researchers know who receives the drug or placebo during the treatment period. The goal is to find the proper dose of TAK-360 and assess how well adults with NT2 tolerate it compared to placebo. During the study, researchers will monitor the number of participants who experience any treatment-related side effects over up to 15 weeks. They will also evaluate safety and tolerability throughout the study. Participants' responses to treatment will provide insights into TAK-360's potential to help people with NT2 stay awake and improve their quality of life.

Researchers are evaluating the safety and tolerability of TAK-861 in people with narcolepsy type 1 (NT1) who have already been exposed to TAK-861 in earlier studies. The study also aims to observe improvements in symptoms such as excessive daytime sleepiness and the frequency of cataplexy episodes. This long-term extension trial continues from previous phase 2 and phase 3 trials and includes participants who completed those earlier studies. All participants in this trial will receive TAK-861 tablets. Those who were previously given a placebo in parent trials will be randomly assigned to a dose of TAK-861. The study plans to enroll up to 500 participants worldwide and will last approximately 5 years, or until the study is stopped or the drug is approved and launched. Participants will visit clinics multiple times, with some visits possibly done at home, and will have a follow-up check 4 weeks after their last dose. During the study, participants will be monitored for treatment-emergent adverse events from the time they consent until 4 weeks after their final dose, covering up to about 5 years. Researchers will assess safety and tolerability regularly through these visits and follow-ups. The focus is on identifying any side effects and understanding the long-term effects of TAK-861 in people with NT1.

Researchers are evaluating the effectiveness and safety of upadacitinib at different doses in adults with moderate to severe atopic dermatitis (AD) who have not responded adequately to dupilumab treatment. AD is a skin condition causing rash and itching due to inflammation, and some people require systemic treatments beyond topical therapies. This phase 3b/4 study aims to provide data on upadacitinib's impact on AD symptoms in this specific population. The study is conducted in two open-label periods. In Period 1, participants are randomly assigned to receive either upadacitinib 15mg orally once daily or dupilumab 300mg by subcutaneous injection every two weeks. After two weeks, those on upadacitinib 15mg may have their dose increased to 30mg based on their response. Period 2 lasts 24 weeks, during which participants either continue their assigned dose or switch doses depending on their eczema severity scores. The entire treatment duration is 32 weeks with follow-up for 30 days after treatment ends. Participants will undergo regular visits at hospitals or clinics for medical assessments, blood tests, side effect monitoring, and questionnaires to evaluate treatment effects. The main outcome measured is the number of participants achieving at least a 90% improvement in their eczema severity index by week 8. The study includes a 35-day screening period before treatment begins and monitors safety and efficacy throughout the study duration.

Hidradenitis suppurativa (HS) is a chronic and often painful skin disease that causes lumps, abscesses, and scars in areas like under the breasts, armpits, inner thighs, groin, and buttocks. Researchers are evaluating the investigational drug lutikizumab compared to placebo in adults and adolescents with moderate to severe HS. This study aims to assess the disease activity and safety of lutikizumab in a Phase 3 clinical trial involving about 1280 participants worldwide.