Clinical trials

This study is the first time the new medicine CPV-104 is being tested in people. CPV-104 is designed to regulate the complement system, which can be overactive in diseases such as C3 glomerulopathy (C3G), an ultra-rare kidney disorder. The study includes healthy adults and adult patients with C3G to assess safety, tolerability, how the body processes the medicine, and whether the immune system reacts to it. The study is divided in two part; in Part 1 (SAD), healthy volunteers receive one IV dose of CPV-104 or a placebo while in Part 2 (MAD) patients with C3G receive four weekly IV doses of CPV-104 (no placebo). Participants will have close monitoring, including side-effect checks, blood and urine tests, ECGs, vital signs, and blood samples to measure drug levels and antibodies. For those with C3G, researchers will also observe kidney function, although the main goal is safety, not testing effectiveness. A Safety Review Committee will regularly review results to ensure it is safe to continue to the next dose or study group.

This is an open-label, solid tumor, continuation, rollover trial which enrolls participants from ongoing BMS parent studies that evaluated adagrasib (MRTX849, BMS-986503) either as monotherapy or in combination with other cancer therapies in patients with non-small cell lung cancer (NSCLC), colorectal cancer (CRC) and other advanced solid tumors.

This is a Ph 2, randomized, double-blind, placebo-controlled global multicenter study to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of PIPE-791 in participants with a diagnosis of Idiopathic Pulmonary Fibrosis (IPF) with or without background treatment.

The main goals of this clinical trial are to find out what the best dose of the study drug, OTP-01, is for patients with solid tumors through understanding how it is tolerated and any side effects that it may cause. The trial will also see if OTP-01 causes tumors to shrink and how the body processes OTP-01 by measuring drug levels in the blood. The main questions this study aims to answer are: * What is the recommended dose of OTP-01 for adults with solid tumors? * Is OTP-01 safe and tolerable? * Does OTP-01 reduce tumor growth? Participants will: * Receive OTP-01 through an infusion into a vein. Doses will be spaced out and never more than once a week. * Have blood tests to evaluate safety and drug levels of OTP-01. These will be done often at first and then less frequently as treatment continues. * Have radiographic scans of their tumor at baseline and during the study at regular intervals. * Have the choice to have an optional tumor biopsy before and after treatment to help researchers understand how OTP-01 affects cancer and the immune system. These biopsies are voluntary and will not affect participation in the study.

This is a Phase 3, open-label, randomized, clinical trial evaluating the efficacy and safety of gedatolisib and palbociclib plus endocrine therapy for the treatment of patients with locally advanced or metastatic HR+/HER2- advanced breast cancer.

The purpose of this open-label, multicenter, phase IIIb, single-arm study is to characterize the efficacy and safety of the combination of ribociclib and standard adjuvant endocrine therapy (ET) on invasive breast cancer-free survival (iBCFS), in a close to clinical practice patient population with HR-positive (HR+), HER2-negative (HER2-), Anatomic Stage Group III, IIB, and a subset of Stage IIA Early Breast Cancer (EBC).

The main purpose of this study is to evaluate the safety and effectiveness of LY4268989 when compared to placebo in adult participants with moderately to severely active ulcerative colitis (UC). The study drug will be administered orally. The study will last up to approximately 108 weeks, excluding screening.

Ovarian cancer (OC) is a lethal disease. The purpose of this study is to assess the safety, pharmacokinetics and efficacy of ABBV901, alone or in combination with other anticancer drugs, in participants with ovarian cancer. ABBV901 is an investigational drug for the treatment of ovarian cancer. This study has 6 parts where participants will receive ABBV-901, alone or in combination with other anticancer therapies. Around 285 participants will be enrolled in the study at approximately 45 sites around the world. In part 1, participants will receive escalating doses of intravenous (IV) ABBV-901 alone. In part 2, participants will receive 1 of 3 doses of IV ABBV-901 alone to determine the optimized dose. In part 3, participants will receive escalating doses of IV ABBV-901 in combination with IV bevacizumab. In part 4, participants will receive recommended doses for expansion of IV ABBV-901 combination with IV bevacizumab. in Part 5, participants will receive escalating doses of IV ABBV-901 in combination with IV carboplatin and IV bevacizumab and in part 6, participants will receive recommended doses for expansion of IV ABBV-901 combination with IV carboplatin and IV bevacizumab. The total study duration will be approximately 3 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic and may require frequent medical assessments, blood tests, and scans.

The purpose of this study is to find out if baricitinib can preserve beta-cell function in participants newly diagnosed with type 1 diabetes. Participation in the study will last about 60 weeks.

The purpose of this study is to find out if baricitinib can delay the onset of clinical type 1 diabetes (T1D) in people who are at high risk to develop T1D. Participation in the study will last up to approximately 5 years.

The main purpose of the SYNERGY-OUTCOMES study is to find out whether retatrutide and tirzepatide can prevent major adverse liver outcomes (MALO) in people with high-risk metabolic dysfunction-associated steatotic liver disease (MASLD). The study will enroll adults who have MASLD based on non-invasive tests (NITs), which indicate they are more likely to develop MALO. Participants will be randomly assigned within a Master Protocol to receive either retatrutide (N1T-MC-RT01), tirzepatide (N1T-MC-TZ01) or placebo. The trial plans to enroll about 4,500 adults and will run for approximately 224 weeks. Participants may have up to approximately 25 to 30 clinic visits throughout the study to monitor their health, complete study procedures, and assess liver function and disease progression. Once the study is complete, eligible participants may participate in an optional 2-year extension study, in which all participants will receive either retatrutide or tirzepatide, even if they received placebo in the main study.

The primary objective of this trial is to demonstrate that maridebart cafraglutide is superior to placebo when given as an adjunct to standard of care with respect to reducing cardiovascular (CV) morbidity and mortality.

The purpose of this study is to evaluate the efficacy and safety of KarXT + KarX-EC for cognitive impairment in Alzheimer's Disease

Migraine is a disease that most often causes moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms. The goals of the study are to evaluate adverse events and how well treatment of atogepant works compared to placebo (looks like the study treatment but contains no medicine) in preventing chronic migraine in participants between 12 and 17 years of age. Atogepant is a medicine currently approved in the United States and Europe for the preventive treatment of migraine in adult patients with migraine and is being studied for the preventative treatment of chronic migraine in participants between the ages of 12 and 17 years. Participants will be randomly assigned to one of the 2 groups to be treated with either atogepant or placebo. This study is double-blinded, which means that neither the patients nor the study doctors know who is given which study treatment. Approximately 420 participants 12 to 17 years of age with chronic migraine will be enrolled at approximately 70 sites across the world. Participants will receive oral tablets of atogepant or placebo once daily for 12 weeks and will be followed for 4 weeks. Participants will attend regular visits during the study at a hospital or clinic and the effects of treatment will be checked by completion of a daily diary, medical assessments, blood tests, checking for side effects, and completing questionnaires.

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib cream in children (6 to \< 12 years old) with nonsegmental vitiligo.

The X-TOLE3 Phase 3 clinical trial is a randomized, double-blind, placebo-controlled study that will evaluate the clinical efficacy, safety and tolerability of XEN1101 administered as adjunctive therapy in focal-onset seizures.

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the clinical efficacy, safety, and tolerability of XEN1101 administered as adjunctive treatment in primary generalized tonic-clonic seizures (PGTCS).

The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.

This study will evaluate de-identified (anonymous) data in subject medical charts to review the clinical outcomes of various treatment approaches in the treatment of chronic pain.

The purpose of this study is to: * Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels

The purpose of the study is to assess the efficacy, safety and tolerability of PLENVU® by measuring its bowel cleansing success rate compared to sodium picosulfate in paediatric participants aged less than 18 years who are scheduled to undergo a colonoscopy. Colonoscopy is a crucial procedure that helps to diagnose and manage various gastrointestinal (GI) conditions in paediatric patients. An adequate level of bowel preparation and cleansing is essential for effective colonoscopy to allow proper visualisation of the bowel.

Neovascular age-related macular degeneration (nAMD), also known as "wet" AMD, is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. The purpose of this study is to assess how safe and effective Surabgene Lomparvovec is in treating participants with Neovascular age-related macular degeneration (nAMD). Surabgene Lomparvovec (ABBV-RGX-314) is an investigational gene therapy being developed for the treatment of neovascular age-related macular degeneration (nAMD). Participants will be placed into 1 of 3 groups, called treatment arms. Each group receives different treatment. Adult participants aged 50 and older years with a diagnosis of previously treated nAMD will be enrolled. Around 561 participants will be enrolled in the study at approximately 150 sites worldwide. Participants in groups 1 and 2 will receive a single subretinal dose of ABBV-RGX-314. Participants in group 3 will receive Ranibizumab as needed throughout the study. Ranibizumab will be given as an intravitreal injection (injection into the jelly-like tissue that fills the eyeball injection), and ABBV-RGX-314 will be given as a subretinal (between the retina and the back of the eye) injection. The Assessment Period begins after randomization (1:1:1) to one of the ABBV-RGX-314 treatment groups or control at Week -2 and lasts up to 5 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular monthly visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

The main purpose of this study is to assess if olomorasib in combination with pembrolizumab is more effective than the pembrolizumab and placebo combination in part A in participants with resected KRAS G12C-mutant NSCLC and to assess if olomorasib in combination with durvalumab is more effective than the durvalumab and placebo combination in part B in participants with unresectable KRAS G12C-mutant non-small cell lung cancer. The study may last up to 3 years for each participant.

The purpose of this pediatric, chronic weight management, Phase 3 Master Protocol (PWMP) is to create a framework to evaluate the safety and efficacy of pharmacologic agents for the treatment of obesity or overweight in pediatric participants.

The main purpose of this study is to evaluate the efficacy and safety of lebrikizumab in participants with chronic rhinosinusitis and nasal polyps treated with intranasal corticosteroids. The study will last about 18 months.