Waterford

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation (CIC). This study is a 26-week, multi-center, randomized, double-blind, placebo-controlled trial followed by a 4-week treatment-free safety follow-up period. It aims to compare three different doses of tenapanor with a placebo taken twice daily to assess their impact on constipation symptoms. The study includes a 2-week screening period to confirm eligibility, followed by a 26-week randomized treatment period where patients receive either 5 mg, 25 mg, or 50 mg of tenapanor twice daily, or a matching placebo. Patients record their constipation symptoms daily in an electronic diary. After the treatment period, there is a 4-week safety follow-up without treatment to monitor any adverse effects. Participants will have regular visits every 2 to 6 weeks for safety checks including medical assessments, vital signs, ECG, and lab tests. Their symptom diaries will be reviewed throughout the study. The main outcome measured is the durable complete spontaneous bowel movements response at 12 weeks. Secondary outcomes include changes in bowel movement frequency, stool consistency, and straining. The total study duration is approximately 32 weeks including all phases.

Researchers are evaluating efruxifermin (EFX) in a randomized, double-blind, placebo-controlled Phase 3 study involving adults aged 18 to 80 with compensated cirrhosis caused by Nonalcoholic Steatohepatitis (NASH) or Metabolic Dysfunction-Associated Steatohepatitis (MASH). The study aims to assess the safety and efficacy of EFX in preventing disease progression and liver complications over a long-term period. Participants receive either efruxifermin or a placebo by subcutaneous injection. The study includes multiple groups, with one cohort requiring biopsy-proven compensated cirrhosis and specific metabolic features, and another allowing biopsy or non-invasive diagnosis. The treatment and follow-up last up to five years, with detailed monitoring at 96 weeks and beyond. During the trial, participants will undergo regular assessments of liver fibrosis, markers of liver injury, lipoproteins, insulin sensitivity, glycemic control, and body weight. Researchers will also monitor adverse events, laboratory tests, ECGs, ultrasounds, and vital signs throughout the study. The primary outcome measures include time to significant clinical events and improvements in fibrosis and steatohepatitis, with extensive safety and efficacy evaluations conducted over five years.

Researchers are evaluating the safety and effectiveness of efruxifermin (EFX) 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 will involve about 1,650 participants divided into two groups based on fibrosis stage and will use a randomized, double-blind, placebo-controlled design. Participants will be assigned to receive either 28 mg or 50 mg of efruxifermin or a placebo, all given by weekly subcutaneous injection. The study includes a screening period of up to 12 weeks, a 52-week primary treatment phase, and long-term follow-up that may last up to approximately 240 weeks. Liver biopsies and other assessments will be done at specific times, including weeks 52, 96, and 240, to evaluate liver changes and treatment effects. Throughout the study, participants will undergo various assessments such as liver biopsies, blood tests, FibroScan scans, and questionnaires to monitor liver health, fibrosis progression, and overall safety. Researchers will track outcomes like resolution of NASH/MASH, fibrosis improvement, liver-related events, and survival over the long term. After the last dose, participants will have a follow-up visit about 30 days later to check their condition.

Researchers are evaluating the efficacy and safety of duvakitug, a drug given by subcutaneous injection, in people with moderately to severely active Ulcerative Colitis (UC). This Phase 3 randomized, double-blind, placebo-controlled study includes participants aged 16 to 80 years and aims to assess clinical remission and other health improvements over a series of treatment periods. The study lasts up to 35 weeks and includes a screening period followed by three possible sub-studies: a 12-week open-label induction, a 12-week pivotal induction, and a 12-week extended induction for those who do not respond initially. Participants receive injections of either duvakitug at one of two doses or placebo according to the study protocol, with up to 15 visits scheduled for those in extended induction. During the study, participants will undergo assessments including clinical remission rates at week 12, endoscopic and histological improvements, symptom tracking, quality of life questionnaires, and safety monitoring for adverse events. Follow-up visits occur up to 45 days after the last dose for those not continuing in the maintenance study. Researchers will also measure drug levels and immune responses over the study period to better understand treatment effects.

Researchers are evaluating the outcomes of two treatments for lumbar spinal stenosis with neurogenic claudication (LSS with NC) in Medicare beneficiaries. This observational study compares the rates of surgical and minimally invasive interventions, as well as any harms, occurring within 24 months after receiving either the MILD procedure or Interspinous Process Decompression (IPD). The study uses Medicare claims data starting from patients treated on or after January 1, 2017, and continues enrollment until the sponsor stops it. The study groups include Medicare patients who underwent the MILD procedure, which involves a partial decompression performed under fluoroscopic image guidance through the removal of tissue and bone at the symptomatic spinal level. The control group consists of Medicare patients treated with Interspinous Process Decompression during the same enrollment period. Both groups are monitored for reoperation and harms for 24 months following their initial treatment. Participants are included based on Medicare claims with the study's NCT number, which automatically enrolls them without requiring prior consent. Researchers will analyze Medicare claims data to track surgical or minimally invasive interventions and any complications related to the initial procedure over two years. The study does not involve direct patient visits or interventions and is exempt from Institutional Review Board oversight. The total follow-up duration for outcome measurement is 24 months after the index procedure.

Researchers are evaluating the effectiveness of budesonide orodispersible tablets in adult patients with active eosinophilic esophagitis (EoE). The study aims to demonstrate that a 6-week treatment with 1 mg tablets taken twice daily is not less effective than 2 mg tablets taken once daily in inducing clinico-pathological remission. This phase III trial is randomized and double-blind to compare these two dosing regimens. Participants receive either 2 mg budesonide tablets once daily or 1 mg tablets twice daily for six weeks. Following this, there is an open-label extension phase where participants may take 0.5 mg or 1 mg budesonide tablets twice daily. The tablets are taken 30 minutes after a meal. The study monitors how well these treatments induce remission and their tolerability. During the study, participants are regularly assessed for histological remission after six weeks, which is the primary outcome. Clinical remission after six weeks is also measured as a secondary outcome. The study involves close monitoring to ensure adherence to treatment and to evaluate safety. Overall participation lasts through the initial treatment and the open-label extension phase, with multiple visits for evaluation.