Auchenflower

Researchers are evaluating the safety, tolerability, and how the body processes and responds to VX-670 in adults with Myotonic Dystrophy Type 1 DM1. This study is a Phase 12 trial that compares different doses of VX-670 to placebo in people diagnosed with DM1 who have a confirmed genetic test showing a specific repeat in their DNA. Participants will be randomly assigned to receive either single or multiple doses of VX-670 or matching placebo. The study has two parts Part A focuses on single ascending doses, while Part B includes both single and multiple ascending doses. The dosing levels in Part B will be based on results from Part A. Both VX-670 and placebo are given as intravenous solutions. During the study, participants will be monitored for adverse events up to 42 days in Part A and 168 days in Part B to assess safety and tolerability. Researchers will measure drug concentrations in blood plasma and muscle at various time points, including baseline, days 15, and 120. Muscle biopsies will be used to analyze changes in gene splicing. The total participation duration varies by study part, with detailed monitoring of drug effects and safety throughout.

The trial investigates the safety and effectiveness of a combination of two investigational kinase inhibitor drugs, avutometinib and defactinib, compared to standard treatments chosen by doctors for women with recurrent low-grade serous ovarian cancer LGSOC who have not responded to prior platinum-based therapy. The goal is to improve progression-free survival and evaluate overall survival, safety, quality of life, and disease symptoms in this patient group. Participants will receive either the combination of avutometinib taken orally twice weekly and defactinib taken orally twice daily on a 21 days on, 7 days off schedule per 28-day cycle, or one of four standard treatments selected by their doctor. These standard treatments include intravenous pegylated liposomal doxorubicin, intravenous paclitaxel, or oral anastrozole or letrozole, each given according to specified doses and schedules. Patients initially receiving standard treatments who experience disease progression may switch to the investigational combination. During the study, participants will have regular follow-up visits to monitor their health, side effects, and disease progression over up to 24 months for progression-free survival and up to 5 years for overall survival. Researchers will assess tumor response, adverse events, drug levels in blood, and quality of life using validated questionnaires. The study involves randomized assignment to treatment groups and includes ongoing assessments to understand the impact of therapies on cancer control and patient well-being.

Researchers are evaluating the safety, tolerability, how the body processes and responds to BG-75098 alone and combined with BGB-43395 and fulvestrant in adults with advanced solid tumors. This Phase 1a1b study aims to understand these effects and preliminary antitumor activity in participants with advanced, metastatic, or unresectable solid tumors, including those who have progressed after other treatments. The study has two phases Phase 1a involves dose escalation where increasing doses of BG-75098 are tested alone and in combination with BGB-43395 and fulvestrant. Phase 1b involves dose expansion where participants receive BG-75098 at the recommended dose from Phase 1a either alone or combined with the other drugs. BG-75098 and BGB-43395 are given orally, while fulvestrant is given by injection. Participants will be monitored for adverse events, drug levels in the blood, and tumor response up to approximately two years. Assessments include pharmacokinetics, pharmacodynamics, and measuring tumor shrinkage or control. Safety and tolerability will be followed from the first dose through 30 days after the last dose, with ongoing evaluations during treatment cycles and follow-up periods up to two years.

Researchers are evaluating elacestrant compared to standard endocrine therapies in adults with node-positive, Estrogen Receptor-positive ER, HER2-negative early breast cancer who are at high risk of cancer returning. The study focuses on those who have had prior endocrine therapy and aims to measure how well elacestrant may prevent invasive breast cancer recurrence over five years. Participants are randomly assigned to receive either 345 mg of elacestrant daily for five years or continue their prior standard endocrine therapy, which may include an aromatase inhibitor anastrozole, letrozole, or exemestane or tamoxifen. The trial is open-label, meaning both participants and researchers know which treatment is given. During the study, participants will have regular assessments to monitor cancer recurrence, survival, side effects, and quality of life. Evaluations include questionnaires on health status and physical functioning at baseline, six months, and annually for up to five years. Safety is tracked through adverse event reporting up to five years plus 28 days. The total participation duration can last up to five years with ongoing monitoring and data collection.

Researchers are evaluating if adding LY3537982 olomorasib to standard anti-cancer drugs improves treatment for participants with untreated advanced non-small cell lung cancer NSCLC that has a specific KRAS G12C gene change. This Phase 3 treatment study includes participants with locally advanced or metastatic NSCLC and aims to compare this combination against standard care. The study is sponsored by Eli Lilly and Company and could last up to 3 years depending on individual response and disease progression. Participants receive LY3537982 orally combined with pembrolizumab given intravenously in 21-day cycles. Some groups also receive chemotherapy drugs pemetrexed and platinum cisplatin or carboplatin intravenously. There are different dose levels and combinations being tested, including placebo groups for comparison. Treatment continues until specific discontinuation criteria are met. Parts of the study are randomized and double-blinded, with some parts non-randomized for safety lead-in. During the study, participants have regular assessments including imaging scans to measure tumor response, blood tests, and questionnaires about symptoms and quality of life. Researchers monitor side effects and survival outcomes. The main measures include progression-free survival and treatment-emergent adverse events over about one year, with overall survival followed for up to three years. Participants are closely followed throughout treatment and after to evaluate the effects and safety of the study medications.

Researchers are studying MGC026, a drug designed to target advanced solid tumors that are relapsed, refractory, unresectable, locally advanced, or metastatic. This Phase 11b study aims to evaluate the safety, tolerability, how the body processes the drug, immune response, and preliminary antitumor effects. The study includes a dose escalation phase followed by a cohort expansion phase to better understand MGC026s effects in different groups of patients. Participants will receive MGC026 through intravenous infusion every three weeks. The dose will be assigned at enrollment. They may continue treatment for up to 35 cycles as long as there are no severe side effects and the cancer does not worsen. The drug is a topoisomerase 1 inhibitor-based antibody-drug conjugate targeting B7-H3. The study monitors participants for side effects, cancer progression, and collects blood samples for routine lab testing and research. During the study, participants will have regular monitoring for adverse events and cancer status over a period of up to 135 weeks. Blood samples will be taken at scheduled times for pharmacokinetic and immunogenicity analyses. Researchers will assess the number and severity of side effects, response rates, duration of response, and development of antibodies against MGC026. Participants health and safety will be closely observed throughout the trial period.

Researchers are evaluating ROC-101 in adults with Pulmonary Arterial Hypertension PAH or Pulmonary Hypertension associated with Interstitial Lung Disease ILD-PH. This Phase 2a, open-label, single-arm, multicenter study aims to assess the safety, tolerability, and effectiveness of oral ROC-101 combined with standard care in these patients. The study focuses on adults aged 18 to 80 and uses measures such as pulmonary vascular resistance and heart catheterization data to evaluate outcomes. Participants will receive standard therapy plus ROC-101 starting at 10 mg orally once daily, increasing up to 40 mg daily over a 24-week treatment period. After this main period, eligible participants may continue in a long-term extension phase until the end of the program or until marketing approval. The study includes detailed heart and lung assessments and monitors for adverse events throughout treatment. During the study, participants will undergo evaluations including right heart catheterization to measure hemodynamic parameters, pulmonary function tests, walk distance tests, and safety assessments. Researchers will track changes from baseline at 24 weeks and monitor adverse events. The studys total duration includes the initial 24-week treatment followed by an extended period, with continuous monitoring of participant health and response to the study drug.

Ovarian cancer is a serious disease with a high number of new cases and deaths worldwide. This study evaluates the safety and disease activity changes of mirvetuximab soravtansine combined with carboplatin, bevacizumab, or bevacizumab alone in female participants with ovarian cancer confirmed to express folate receptor alpha FR. The trial is a Phase 2 study involving approximately 400 participants worldwide, assessing different treatment combinations given by intravenous infusion. Participants will be assigned to one of three substudies and receive treatment in groups called arms. Substudy 1 includes arms where participants receive one of two doses of mirvetuximab soravtansine with bevacizumab or bevacizumab alone. Substudy 2 involves mirvetuximab soravtansine with carboplatin followed by mirvetuximab soravtansine alone. Substudy 3 combines mirvetuximab soravtansine, bevacizumab, and carboplatin followed by mirvetuximab soravtansine with bevacizumab. Treatments are given by intravenous infusion, and the study duration ranges up to approximately 40 months depending on the substudy. Participants will attend regular visits at hospitals or clinics where they will receive infusions and undergo medical assessments including blood tests and scans. Researchers will monitor treatment-emergent adverse events, ocular events, overall response, progression-free survival, and other outcomes up to about 40 months. The study includes careful safety monitoring and evaluation of disease activity during and after treatment to assess the effects and safety of these drug combinations.

Researchers are evaluating an experimental drug called odronextamab combined with chemotherapy in adults with Diffuse Large B-cell Lymphoma DLBCL, including those who have not been treated before as well as those with relapsed or refractory disease. The study aims to assess the safety, tolerability, and dosing schedule of odronextamab with chemotherapy, and to compare its effectiveness against the current standard treatment of rituximab combined with chemotherapy. Additional goals include understanding side effects, drug levels in the blood, immune responses to the drug, and impact on quality of life and daily activities. The study consists of three parts Part 1A involves dose escalation to find a safe dose, Part 1B explores two dosing regimens of odronextamab combined with chemotherapy, and Part 2 randomly assigns participants to receive either odronextamab plus chemotherapy Odro-CHOP or rituximab plus chemotherapy R-CHOP. Odronextamab and rituximab are given by intravenous infusion, with chemotherapy drugs including cyclophosphamide, doxorubicin, vincristine, and prednisone or prednisolone administered as part of the treatment regimen. Participants will be closely monitored throughout the study for side effects, disease progression, and response to treatment. Assessments include measuring dose limiting toxicities, treatment-emergent adverse events, progression free survival, quality of life questionnaires, and blood tests for drug levels and antibodies. The study follows participants for up to 5 years to track long-term outcomes and safety, with regular visits and evaluations scheduled during and after treatment.

Researchers are evaluating how well mocertatug rezetecan Mo-Rez works compared to standard chemotherapy treatments in women with endometrial cancer who have previously received platinum-based chemotherapy and immunotherapy. This Phase 3 study also examines the safety and tolerability of Mo-Rez and aims to better understand the main side effects of the drugs. The trial is sponsored by GlaxoSmithKline and involves adult female participants. Participants are randomly assigned to one of two groups one group receives mocertatug rezetecan, and the other receives standard of care chemotherapy with either paclitaxel or doxorubicin, depending on the investigators choice. The study follows a parallel design with single masking. Treatments are given according to the study protocol, and the trial includes up to approximately 97 weeks of follow-up for primary outcomes and up to 156 weeks for secondary outcomes. During the study, participants will have regular assessments including scans reviewed by blinded independent central reviewers, quality of life questionnaires, and monitoring for adverse events through laboratory tests, vital signs, and ECGs. Researchers will measure objective response rates, progression-free survival, overall survival, and other treatment outcomes. The study tracks safety events, antibody responses to Mo-Rez, and changes in participants quality of life over time. Participation may last several years, with ongoing evaluations to monitor health and treatment effects.