Auchenflower

This research aims to study the safety, tolerability, pharmacokinetics, and pharmacodynamics of the drug VX-670 in adults who have Myotonic Dystrophy Type 1 (DM1). The trial involves participants aged 18 to 64 years with a confirmed diagnosis of DM1, including a genetic test showing a specific CTG repeat count. The study is a Phase 1/2 trial designed to assess how the drug behaves and how well it is tolerated in this population. Participants will receive VX-670 or a placebo, both administered intravenously, in single and multiple dose escalations. The study is randomized, double-blind, and placebo-controlled to compare the effects of the drug against a non-active treatment. The treatment periods include initial dosing and extended follow-up to evaluate responses over time. During the study, researchers will monitor participants closely for any adverse events from the start up to 42 days in the initial phase and up to 168 days in the extended phase. Safety and tolerability will be the main focus, alongside collecting data on how the drug is processed by the body and its biological effects. Participants will undergo assessments to track these outcomes throughout their involvement in the trial.

This research aims to evaluate the safety and effectiveness of the combination of avutometinib and defactinib compared to standard treatments chosen by investigators in women with recurrent low-grade serous ovarian cancer (LGSOC) who have experienced disease progression after prior platinum-based therapy. Both avutometinib and defactinib are investigational kinase inhibitors designed to block cancer cell growth. The study will also assess overall survival, other measures of treatment effectiveness, safety, and quality of life impacts. Participants will be randomly assigned to receive either the combination of oral avutometinib and defactinib or one of four standard treatments recommended for recurrent LGSOC: pegylated liposomal doxorubicin and paclitaxel (both given intravenously), or the oral drugs letrozole or anastrozole. Patients treated with standard therapies who experience disease progression may be eligible to switch to the investigational combination. The study is open-label and conducted internationally by specialists in gynecological cancer. Throughout the study, participants will have regular follow-up visits including scans and tests to measure disease progression, with a primary focus on progression-free survival up to 24 months. Researchers will monitor safety, side effects, and overall survival while collecting information on quality of life and symptoms. The study involves ongoing assessments of treatment effects and participant health until the study concludes or disease progression occurs.

Researchers are evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and early antitumor effects of the drug BG-75098 alone and combined with BGB-43395 and fulvestrant in adults with advanced solid tumors. The study focuses on participants with measurable disease and good performance status, including those with tumors resistant to previous treatments or who have limited options. This is a phase 1 trial conducted in two parts to explore appropriate dosing and effectiveness. The study is divided into two phases: Phase 1a Dose Escalation and Phase 1b Dose Expansion. BG-75098 and BGB-43395 are given orally, while fulvestrant is administered by intramuscular injection. In Phase 1a, doses of BG-75098 are increased gradually to find the maximum tolerated or administered dose, both alone and in combination with the other drugs. Phase 1b expands on this to assess the recommended doses and evaluate the treatment's impact on tumor response over up to two years. Participants will undergo regular monitoring throughout the study, including assessments of adverse events from the first dose up to 30 days after the last dose, and evaluations of tumor response by investigators. The study tracks safety and dosage levels for up to two years. Participants will receive various tests such as imaging, laboratory studies, and performance evaluations to measure treatment effects and safety. The total duration of involvement depends on the treatment phase and follow-up periods.

Researchers are evaluating how well elacestrant works compared to standard endocrine therapy in adults with node-positive, Estrogen Receptor-positive (ER+), Human Epidermal Growth Factor-2 negative (HER2-) early breast cancer who are at high risk of the cancer returning. This is a Phase 3 global, multicenter, randomized, open-label study focusing on participants who have had early invasive breast cancer removed and meet specific receptor and risk criteria. The study aims to understand which treatment better prevents invasive breast cancer over up to five years. Participants will receive either elacestrant or one of several standard endocrine therapies, including anastrozole, letrozole, exemestane, or tamoxifen, all given as oral tablets. Treatments will be administered according to the study plan, with careful monitoring throughout the trial. The study includes adults who have already received between 24 and 60 months of prior endocrine therapy, with or without certain inhibitors, and who have completed or stopped these treatments as required. During the study, participants will be monitored for invasive breast cancer-free survival for up to five years. Researchers will perform regular assessments to track treatment effects, side effects, and cancer recurrence. The study also includes safety monitoring and may involve additional tests or evaluations as needed to ensure participant well-being throughout the trial.

Researchers are evaluating the effectiveness of adding LY3537982 (olomorasib) to standard anti-cancer drugs compared to standard treatment alone in participants with untreated advanced non-small cell lung cancer (NSCLC) that has a specific KRAS G12C gene mutation. This pivotal Phase 3 trial includes participants with locally advanced or metastatic NSCLC and considers their programmed death-ligand 1 (PD-L1) expression levels. The study includes multiple parts: Dose Optimization, Part A, and Part B are randomized, while Safety Lead-In for Part B and Part C are non-randomized. Treatments being assessed include LY3537982 taken orally, pembrolizumab administered intravenously, and standard chemotherapy drugs such as cisplatin, carboplatin, and pemetrexed given intravenously. Participants receive these treatments according to their assigned groups based on their PD-L1 expression and tumor histology. Participants will be monitored with regular assessments including measuring disease progression, safety evaluations, and treatment emergent adverse events for up to approximately one year, with overall study participation potentially lasting up to three years depending on individual response and health status. Outcome measures focus on progression-free survival and safety, capturing any adverse events from the start of treatment until disease progression or death.

Researchers are studying the safety, tolerability, pharmacokinetics, immunogenicity, and early antitumor effects of MGC026 in adults with relapsed or refractory, unresectable, locally advanced, or metastatic solid tumors. This phase 1/1b study includes a dose escalation phase and a cohort expansion phase to better understand how MGC026 works and its safety profile in various advanced cancers such as lung, breast, ovarian, melanoma, and others. Participants will receive MGC026 through intravenous (IV) infusions. The initial dose is assigned when participants join the study, and they may receive up to 35 treatments as long as severe side effects do not occur and the cancer does not worsen. The study includes escalating doses in the dose escalation phase and a recommended dose in the expansion phase to find the best balance of safety and activity. Throughout the study, lasting up to 135 weeks, participants will be closely monitored for side effects and cancer progression. Blood samples will be collected regularly for routine lab tests and research purposes. Researchers will track adverse events, including serious ones and those causing dose changes or treatment stops, to evaluate the overall safety and tolerability of MGC026.

Researchers are evaluating the effects of TX000045 in patients with pulmonary hypertension caused by heart failure with preserved ejection fraction (PH-HFpEF). This Phase 2, double-blind, randomized, placebo-controlled proof-of-concept study aims to assess two dosing regimens of TX000045 over a 24-week treatment period to understand its impact on pulmonary vascular resistance and safety profile. Participants will be randomly assigned to one of three groups: a placebo group receiving subcutaneous injections every two weeks, a group receiving Dose A of TX000045 subcutaneously every two weeks, and a group receiving Dose B of TX000045 subcutaneously every four weeks alternating with placebo every two weeks. The treatment period lasts for 24 weeks. Throughout the study, participants will undergo assessments including pulmonary vascular resistance measurements, physical examinations, laboratory tests, and monitoring for adverse events from baseline up to 30 weeks after the first dose. Safety evaluations focus on treatment-related side effects and changes in lab values. The study plans to enroll about 180 participants between 18 and 83 years old with specific heart and lung function criteria.

Ovarian cancer is a serious disease with many new cases and deaths worldwide. This research aims to study the safety and effects of different combinations of an investigational drug called mirvetuximab soravtansine with carboplatin, bevacizumab, or bevacizumab alone in adults with ovarian cancer who have tested positive for folate receptor alpha. The study is a Phase 2 trial designed to understand adverse events and changes in disease activity. Participants will be assigned to one of two substudies. In substudy 1, participants receive one of two doses of mirvetuximab soravtansine with bevacizumab or bevacizumab alone. In substudy 2, participants receive one of two doses of mirvetuximab soravtansine combined with carboplatin, followed by mirvetuximab soravtansine alone. All treatments are given through intravenous infusion. Around 320 participants will take part across about 100 sites worldwide. Participants will have regular visits at hospitals or clinics for medical assessments, blood tests, and scans. The study lasting up to approximately 40 months will track treatment-emergent adverse events, ocular side effects, treatment discontinuations due to side effects, overall response to treatment, and progression-free survival. Researchers will assess disease status using imaging criteria and monitor participant safety throughout the study.

Researchers are studying the long-term safety, tolerability, and effects of a medicine called PF-07868489 for people with pulmonary arterial hypertension (PAH), a condition where high blood pressure in the arteries makes it harder for the heart to pump blood to the lungs, potentially harming the right side of the heart. This study is an open-label extension, meaning participants and healthcare providers know the treatment being given, and it allows patients from a previous clinical study with PF-07868489 to continue to assess long-term benefits and safety. Participants will receive subcutaneous doses of PF-07868489 every 4 weeks. This ongoing treatment period follows completion of the prior study, focusing on evaluating the medicine’s effects over a longer time. The study does not include placebo or other treatment groups since it is an extension of earlier research. During the study, participants will have regular visits where researchers will monitor safety by tracking adverse events, changes in laboratory test results, and vital signs from baseline up to week 111. Participants must follow scheduled visits, treatment plans, and study procedures, including lifestyle considerations and laboratory tests. The study aims to gather long-term data on how the medicine affects participants with PAH over an extended period.

Researchers are evaluating an experimental drug called linvoseltamab (REGN5458) for adults with relapsed or refractory multiple myeloma who have had one to four previous treatments and have standard treatment options available. This phase 3 study compares linvoseltamab to a combination of three cancer drugs: elotuzumab, pomalidomide, and dexamethasone (EPd). The study aims to assess the safety and effectiveness of linvoseltamab compared to EPd, including how long participants benefit, tumor response, side effects, survival, and pain improvement. Linvoseltamab is given by intravenous infusion, while the comparison group receives elotuzumab by infusion and pomalidomide capsules and dexamethasone tablets or capsules by mouth or IV. Participants are randomly assigned to receive either linvoseltamab or the EPd combination. The study includes participants who have previously received lenalidomide, a proteasome inhibitor, and in some cases, a CD38 antibody. Treatment continues as per protocol with ongoing monitoring. Participants will undergo regular assessments to evaluate their disease response and side effects. Researchers will monitor progression-free survival for up to approximately five years. Assessments include measuring tumor response, survival, pain levels, and safety. Participants must have measurable disease and adequate organ function, and they will be followed closely to assess how well the treatments work and their safety over time.