Novi

Researchers are conducting a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of HBS-301 in adults aged 18 years and older with narcolepsy. The study focuses on treating excessive daytime sleepiness EDS, cataplexy, sleepinesswakefulness, and fatigue in participants diagnosed with narcolepsy. Approximately 258 participants will be included in this trial. Participants will be randomly assigned to receive either HBS-301 tablets or matching placebo tablets once daily in the morning upon waking. The study includes a ScreeningBaseline period lasting up to 28 days, followed by an 8-week Double-blind Treatment period. After this, participants may choose to enter a 1-year Open-label Extension period where they will receive HBS-301. Finally, a 30-day safety follow-up will monitor participants after treatment ends. Throughout the study, participants will undergo various assessments including evaluations of daytime sleepiness using the Epworth Sleepiness Scale, cataplexy frequency, wakefulness through the Maintenance of Wakefulness Test, fatigue levels, cognitive complaints, quality of life, and work productivity. Safety will be monitored by tracking adverse events and pharmacokinetic measurements. The total study participation may last up to approximately 16 months including all phases.

Researchers are studying the use of a personalized mobile application called SHIFT to improve sleep in night shift workers with Shift Work Sleep Disorder. The study aims to build evidence for fair access and effective use of this precision sleep medicine tool, which uses body-clock data collected from an Apple Watch to provide tailored light exposure schedules. The trial is randomized and double-blind, comparing the SHIFT app to a waitlist control group over a period of up to eight months. Participants in the SHIFT app group receive access codes to download the app and attend an orientation session. They then follow a four-week period of guided app use where they start their day with the app and plan their activities based on its recommendations, using the app about four times per week. This is followed by self-directed use, with a booster session at four months that includes two additional weeks of guided use before continuing self-directed use until the study ends. The waitlist control group completes questionnaires during the first eight months and may choose to use the app afterward if still working night shifts. During the study, participants complete surveys and app usage is monitored to assess engagement, experience, and accuracy of the app on different platforms. The main outcomes include evaluating the effect of SHIFT on participant-centered results and comparing app versions from enrollment through eight months. Additional assessments explore factors that help or hinder app use. The study involves regular evaluations over the first eight months, with follow-up and safety monitoring continuing until the study concludes in mid-2027.

This research aims to evaluate the clinical benefit, performance, and safety of a totally implantable cochlear implant TICI system in adults with sensorineural hearing loss, which is hearing loss caused by damage to the inner ear or auditory nerve. The study is pivotal and pre-market, focusing on how this system, which includes a microphone placed under the skin to detect speech and sounds without any visible external parts, can affect hearing ability and daily life. Participants will be implanted with the TI1132 device, a totally implantable cochlear implant system. The study involves a single group design where all participants receive this investigational implant. The research team will monitor and assess the implants performance and safety over time from the implantation through post-activation periods. During the study, participants will undergo hearing tests and complete questionnaires before implantation and up to 12 months after activation of the device. The tests will measure speech recognition performance in quiet and noise, and various quality of life and hearing questionnaires will evaluate the impact on daily living. Safety will be monitored by tracking adverse events and device issues. The total participation period includes assessments up to 12 months post-activation, with the study completion expected in August 2028.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating efruxifermin EFX in adults with non-cirrhotic nonalcoholic steatohepatitis NASH or metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage 2 or 3. This Phase 3, multi-center, randomized, double-blind, placebo-controlled study aims to assess the safety and efficacy of EFX compared with placebo. The trial includes about 1,650 participants divided into two cohorts based on liver biopsy characteristics and fibrosis stage. Participants will be randomly assigned to one of three groups EFX 28 mg, EFX 50 mg, or placebo, each given as a weekly subcutaneous injection. Cohort 1 will be evaluated over 52 weeks for histologic efficacy endpoints, while Cohort 2 will have assessments over 96 weeks. After these periods, participants may continue long-term treatment and clinical follow-up for up to approximately 240 weeks total. A follow-up visit will occur about 30 days after the last dose. During the study, participants will undergo liver biopsies, blood tests, and non-invasive assessments such as FibroScan and Enhanced Liver Fibrosis ELF score to monitor liver health and fibrosis. Researchers will track liver-related clinical outcomes, including liver events and survival, as well as safety and tolerability of the treatment. Participants who stop the study drug may still continue with scheduled assessments to support long-term safety and efficacy evaluations.

Researchers are evaluating the safety and tolerability of TAK-861 in people with narcolepsy type 1 NT1. This study focuses on participants who have already been exposed to TAK-861 doses in previous clinical trials. The goal is to monitor how TAK-861 affects symptoms such as excessive daytime sleepiness and cataplexy episodes over a long period. All participants in this trial will receive TAK-861 tablets. Those who previously received a placebo will be randomly assigned to one of the TAK-861 dose groups. The study is a long-term extension conducted worldwide and is expected to last approximately five years or until the product is approved or the study is stopped. Participants may switch doses as needed and will attend multiple clinic visits, some of which can be done at home. Throughout the trial, participants will be regularly assessed for safety by tracking any treatment-emergent adverse events. Researchers will also measure changes in sleep latency, sleepiness scores, and cataplexy rates compared to baseline data from earlier trials. Follow-up assessments will take place four weeks after the final dose to monitor ongoing effects and ensure participant safety.

This trial studies newly diagnosed multiple myeloma in participants who are not candidates for stem cell transplant. It compares the effects of two drug combinations teclistamab with daratumumab and lenalidomide Tec-DR, and talquetamab with daratumumab and lenalidomide Tal-DR, against the standard treatment of daratumumab, lenalidomide, and dexamethasone DRd. The goal is to assess how these combinations affect disease progression and treatment response. Participants are randomly assigned to one of three groups receiving either Tec-DR, Tal-DR, or DRd. Teclistamab and talquetamab are given as subcutaneous injections, daratumumab is given subcutaneously, lenalidomide is taken orally, and dexamethasone can be given orally or intravenously. Treatments are administered according to the study protocol over an extended period, with follow-up lasting up to nine years to monitor outcomes. During the study, participants undergo regular evaluations including disease progression monitoring, minimal residual disease status at 12 months, and assessments of response levels. Researchers also track survival, adverse events, laboratory and vital sign changes, quality of life, and drug concentrations. The study involves multiple visits for treatment and assessment to carefully evaluate the long-term impact of these drug combinations on patient health and disease control.

Researchers are evaluating whether adding immunotherapy drugs brentuximab vedotin and nivolumab to the standard treatment of chemotherapy with or without radiation improves survival for patients aged 5 to 60 with early stage classical Hodgkin lymphoma. This phase III trial compares progression-free survival and overall survival between the standard therapy and the immunotherapy-enhanced approach, as well as patient-reported outcomes and long-term side effects. Participants initially receive two cycles of ABVD chemotherapy every 28 days and then undergo imaging to classify their early response. Based on risk level and response, patients are assigned to one of eight treatment arms that include either continuing standard chemotherapy, receiving immunotherapy drugs, or combinations with involved-site radiation therapy. Treatments are delivered intravenously or orally in cycles lasting 28 days. Imaging and blood samples are collected throughout the trial. Participants are monitored regularly with PET scans, CT or MRI imaging, and blood tests. Follow-up visits occur every 3 months in the first year, then every 6 months for years two and three, and annually up to 12 years from registration. Researchers assess survival outcomes, adverse events, patient-reported symptoms and quality of life, and metabolic tumor burden. Long-term effects such as cardiovascular and pulmonary health are also evaluated using questionnaires and clinical assessments.

Researchers are evaluating the optimal doses of the drug E2086 compared to placebo in adults with narcolepsy, a condition characterized by excessive daytime sleepiness EDS. The study focuses on reducing EDS as measured by the Mean Sleep Latency MSL using the first four maintenance of wakefulness tests MWTs. This Phase 2 trial includes participants diagnosed with either narcolepsy type 1 NT1 or type 2 NT2 within the last 10 years. Participants will be randomly assigned to receive either E2086 or a matching placebo tablet taken orally once daily for four weeks at low, middle, and high doses. Each dosing period is separated by a washout period of at least seven days, with a total treatment duration of approximately 14 weeks. This design allows comparison of the effects of different doses of E2086 on narcolepsy symptoms. During the study, participants will undergo assessments to measure changes in sleep latency, cataplexy episodes, and sleepiness scales. Additional safety evaluations include monitoring for adverse events, laboratory tests, vital signs, ECG parameters, and suicidality assessments. Blood samples will be collected to analyze drug concentration levels. The study involves regular monitoring up to Day 113 and aims to assess both efficacy and safety over the course of treatment and follow-up.

Researchers are evaluating the safety, tolerability, and effectiveness of apraglutide compared with a placebo in adults with short bowel syndrome associated with intestinal failure SBS-IF. This Phase 3, double-blind, randomized, placebo-controlled study aims to include about 124 adult participants who rely on parenteral support PS at least three days per week. Participants are stratified by anatomy and baseline PS volume to ensure balanced study groups. Participants will be randomly assigned to receive either weekly subcutaneous injections of apraglutide or placebo for 24 weeks. The study consists of four periods Screening including optimization and stabilization, Treatment, Safety Follow-up after the last dose, and an Anti-Drug Antibody Follow-up period. Those completing treatment may join a long-term extension study with apraglutide or complete a safety follow-up visit before entering the antibody monitoring phase. During the study, participants will have their weekly parenteral support volume measured to assess changes. Additional assessments include reductions in PS days per week, achievement of enteral autonomy, and patient-reported global impression of change. Safety will be monitored through adverse event tracking and laboratory tests. Participants are expected to adhere to fluid intake monitoring and may undergo colonoscopy or imaging to remove any polyps. The entire participation timeline extends beyond treatment to ensure thorough safety and immune response evaluation.