A PHASE 1B/2 PAN-TUMOR OPEN-LABEL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF IFINATAMAB DERUXTECAN (I-DXD) IN SUBJECTS WITH RECURRENT OR METASTATIC SOLID TUMORS (IDeate-Pantumor02)
This is a global, multicenter, pan-tumor, open-label, single-treatment arm, parallel-cohort, Phase 1b/2 study ofI-DXd in subjects with recurrent or metastatic solid tumors previously treated with 1 or more systemic therapiesfor the selected tumor indication.This study will consist of 13 cohorts targeting the following tumor types: endometrial cancer (EC); head and necksquamous cell carcinoma (HNSCC); pancreatic ductal adenocarcinoma (PDAC); colorectal cancer (CRC);hepatocellular carcinoma (HCC); adenocarcinoma of esophagus, gastroesophageal junction, and stomach(Ad-Eso/GEJ/gastric); urothelial carcinoma (UC); ovarian cancer (OVC); cervical cancer (CC); biliary tractcancer (BTC); human epidermal growth factor 2 (HER2)-low breast cancer (BC); HER2 immunohistochemistry(IHC) 0 BC; and cutaneous melanoma.Each cohort starts with Stage 1 and may continue to Stage 2 if sufficient safety and efficacy data are observed.Throughout both stages, subjects will be closely monitored for safety.
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 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 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 Multicenter Dose Ranging Study Evaluating the Efficacy and Safety of GS-1427 in Adult Participants With Moderately to Severely Active Ulcerative Colitis (UC)
The study team is evaluating an oral investigational drug to see if it reduces the signs and symptoms of moderately to severely active ulcerative colitis (UC) by blocking certain processes involved in the inflammation pathway. The study is also evaluating if there are any adverse events or other safety issues associated with the investigational drug.
A Phase 2 randomized open-label active-controlled study of JNJ-90301900 in combination with chemoradiation followed by durvalumab in locally advanced and unresectable Stage III non-small cell lung cancer
This is a Phase 2 study testing whether adding a new medicine, JNJ-90301900 (radioenhancer) to the standard treatment of chemotherapy and radiation, followed by a medication called durvalumab (a type of immunotherapy) can be a better treatment option for patients with a type of lung cancer called non-small cell lung cancer (NSCLC). This study will be done in two parts. In Part 1, patients will be enrolled sequentially into two groups to receive a JNJ-90301900 injection at two different dose levels along with standard treatment of chemotherapy and radiation, followed by a medication called durvalumab. In part 2, the patients will be randomly divided into three groups. Patients in Groups A and B will receive the study medicine at two different dose levels along with standard treatment of chemotherapy and radiation, followed by a medication called durvalumab. Patients in Group C will receive the standard chemotherapy and radiation treatment, followed by a medication called durvalumab. The study will compare the effects of this new combination treatment with those of the standard treatment alone. All patients will have blood samples taken to see how their bodies handle the study medicines and how well the cancer responds to this treatment over time. Patients will have special scans taken to monitor how the cancer is changing in response to the study medication. The study team will monitor all patients for side effects and safety concerns throughout the study. All patients will be followed up after their last study dose for up to 24 months.