A Phase 1b/2 Study to Demonstrate the Safety and Efficacy of EXE-346 Live Biotherapeutic to Reduce High Bowel Movement Frequency in Subjects With an Ileal Pouch-Anal Anastomosis
The aim of this study is to assess the safety and preliminary efficacy of treatment with EXE-346, a live biotherapeutic product (LBP)containing a fixed proportion mixture of 8 individual bacterial strains, which may reduce bowel movement frequency in patients with an IPAA and lead to a higher quality of life (QoL). The Phase 1b part of the study is an open label (OL), single-arm study to assess the safety of EXE-346 administered orally at a dose of 1500 × 109 CFU BID for up to 4 weeks. The Phase 2 part of the study will initially be a randomized, double-blinded study to assess the safety and efficacy of the same dose of EXE-346 administered orally BID for up to 8 weeks, compared with placebo. Subjects who complete this Phase 2 double-blinded part of the study will be eligible to participate in an optional OL extension phase. Subjects who participate in the optional OL extension phase will receive EXE-346 orally at a dose of 1500 × 109 CFU BID for up to 8 weeks.
A Phase 1b/2a Study of Gemcitabine and Nab-paclitaxel in Combination with VS-6766 and Defactinib in Patients with Previously Untreated Metastatic Adenocarcinoma of the Pancreas
This study will assess the safety and efficacy of avutometinib (VS-6766) and defactinib in combination with gemcitabine and nab-paclitaxel in patients with Pancreatic Ductal Adenocarcinoma (PDAC) who have been previously untreated.
A Phase 2 double-blind placebo-controlled parallel-group study to assess the safety tolerability pharmacokinetics pharmacodynamics and potential efficacy of multiple doses of ONO-2808 in patients with Multiple System Atrophy (MSA)
The purpose of this clinical research study is to look at the safety and tolerability of the investigational study drug, ONO-2808, and whether it works when given to people with Multiple System Atrophy (MSA). MSA is a rare nervous system disease that can affect movement, balance, speech, blood pressure, bladder, and digestive function. Currently there is no FDA approved treatment for MSA, ONO-2808 may have the potential to reduce the a-synuclein (mis-folded a-synuclein is the protein which is believed to be the cause of MSA) in the central nervous system (CNS). ONO-2808 may promote nerve healing which, can lead to slowing of disease progression and provided symptomatic improvement. The study will look at 3 different doses of the study drug, ONO-2808, compared to a placebo. ONO-2808is an investigational drug which means it is not FDA approved and in this study is experimental. The purpose of this clinical research study is to: Look at how tolerable the study drug, ONO-2808, is Whether it works when given to people with MSA What doses of the study drug are well tolerated How the study drug behaves inside the human body There are currently no approved drugs to slow disease progression or provide a cure for MSA. This is a Phase 2 study. Phase 2 studies test whether or not a potential new drug works. The study drugwill have been already tested in healthy volunteers. This is the first study of ONO-2808 in MSA patients.
A Phase 2 Double-Blind Placebo-Controlled Single-Dose Study of Pharmacodynamics Pharmacokinetics Safety and Tolerability of REGN7544 an NPR1 Antagonist Monoclonal Antibody in Patients with Postural Orthostatic Tachycardia Syndrome
The purpose of this research study is to research an experimental drug called REGN7544. The study is focused on participants with postural orthostatic tachycardia syndrome (POTS). The aim of the study is to see how safe, tolerable, and effective the study drug is.
A PHASE 2 MULTICENTER MULTINATIONAL RANDOMIZED DOUBLE BLIND PLACEBO CONTROLLED STUDY TO ASSESS THE SAFETY EFFICACY AND PHARMACOKINETICS OF MULTIPLE DOSE LEVELS OF ESK 001 IN ADULT PATIENTS WITH SYSTEMIC LUPUS ERYTHEMATOSUS
Overall Design: This is a Phase 2, multicenter, multinational, randomized, double blind, placebo controlled study to evaluate the safety, efficacy, PK, and PD of 3 dose levels of ESK 001 (20 mg once daily [QD], 40 mg [taken as 20 mg twice daily (BID)], and 80 mg [taken as 40 mg twice daily (BID)] in patients with moderately to severely active, autoantibody positive SLE while receiving standard of care (SOC) treatment in adult patients aged 18 to 70 years of age.The purpose of this study is to assess the clinical efficacy, safety, PK, and PD of multiple dose levels of ESK 001 compared with placebo in adult patients with SLE.Study details include the following:• Randomization will be stratified using the SLEDAI 2K score at screening (
A Phase 2 Multicenter Randomized Double-blind Placebo-controlled Study to Evaluate the Safety Tolerability Pharmacokinetics and Efficacy of TTI-101 in Participants with Idiopathic Pulmonary Fibrosis
This multicenter, randomized, double-blind, placebo-controlled, parallel-group clinicalstudy is designed to evaluate the safety and tolerability of 3 dose levels of TTI-101 vs placebo in participants with idiopathic pulmonary fibrosis (IPF).Following a screening period of up to 28 days, 100 participants will be randomly assigned (1:1:1:1) to receive 1 of 3 dose levels of TTI-101 or matching placebo.
A Phase 2 Open-label Randomized Study of V940 in Combination With BCG Versus BCG Monotherapy in Participants With High-risk Non-muscle Invasive Bladder Cancer (INTerpath-011)
This study is designed to compare the antitumor activity of V940 in combination with BCG to BCG monotherapy, and evaluate safety of both treatments, in participants who have high risk NMIBC and are BCG-naïve (Cohort A), and, in an exploratory fashion, eva
A Phase 2 Open-label Study Evaluating Dosimetry Randomized Dose Optimization Dose Escalation and Efficacy of Ac-225 Rosopatamab Tetraxetan in Participants with PSMA PET-Positive Castration-Resistant Prostate Cancer
This is a three-part study which includes biodistribution (Part 1), dose optimization (Part 2) to determine the optimal dose of Ac-225 rosopatamab tetraxetan to be evaluated in future studies, and dose escalation (Part 3) in post-Lu-177-PSMA-RL participants using the Bayesian optimal interval (BOIN) design. The study will evaluate participants with CRPC previously treated with at least one ARSI with and without prior exposure to Lu-177-PSMA-RL.
A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment With or Without Dialysis PHYOX7
The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.Primary hyperoxaluria is typically diagnosed by measuring oxalate levels in urine. However, as kidney function decreases, the renal excretion of oxalate also decreases and may no longer reflect daily oxalate loads (Perinpam et al., 2017). Decreasing renal excretion of oxalate results in increasing Pox levels (Hoppe et al., 1998, Hoppe et al., 2009). Because a decrease in Pox is reasonably likely to predict clinical benefit due to its causal role in systemic oxalosis in CKD Stages 3b to 5, Pox may be a more relevant endpoint in patients with PH who have severe renal impairment (Milliner et al., 2020).
A Phase 2 Randomized Double-Blind Placebo-Controlled Dose-Ranging Study to Investigate the Safety and Efficacy of Oral Brepocitinib in Adults with Cutaneous Sarcoidosis
The purpose of this clinical research study is to learn more about the use of an investigational medicine, called brepocitinib, for the treatment of adults with cutaneous sarcoidosis. The study will also look at how safe and effective brepocitinib is when taken for a period up to 16 weeks.