Augusta

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

Researchers are evaluating the experimental antibody COM701 in participants with relapsed platinum sensitive ovarian cancer (PSOC). This trial aims to find out if COM701, given as a maintenance treatment, can delay the progression of ovarian cancer, delay the need for new anti-cancer treatments, and to assess its safety. The study is part of an adaptive-platform trial with multiple sub-studies, focusing initially on COM701 alone compared to a placebo. Participants are randomly assigned in a 1:2 ratio to receive either a placebo or COM701 via intravenous infusion every 3 weeks. The trial includes a double-blind, randomized, placebo-controlled design for the first sub-study. Future sub-studies will explore COM701 combined with other anti-cancer drugs. During the study, participants will visit the clinic every three weeks for treatment and monitoring. Health checks include physical exams, vital signs, ECGs, blood and urine tests, and pregnancy tests if applicable. Disease response will be assessed with CT or MRI scans and tumor marker tests using tumor tissue samples. The primary measure is progression-free survival, tracking time from randomization until disease progression or death, assessed up to two years.

Researchers are investigating BGB-16673, a targeted protein degrader aimed at treating various B-cell cancers including marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia, Waldenström macroglobulinemia, and diffuse large B-cell lymphoma. The study includes both Phase 1 and Phase 2 parts to determine safe and effective dosing and to evaluate the drug's response in patients. The trial is conducted under the new company name BeOne Medicines, previously known as BeiGene. The treatment involves oral administration of BGB-16673. Phase 1 focuses on dose escalation and safety expansion to identify the maximum tolerated dose and recommended dose for expansion over approximately 28 days to 3 years. Phase 2 includes expansion cohorts to assess overall response rates over about 3 years. Participants may have prior treatments including Bruton tyrosine kinase inhibitors and other anticancer therapies depending on their cancer type and study phase. Participants will be monitored closely with assessments of adverse events from the first dose until 30 days after the last dose or before starting new therapy, whichever comes first, for up to 47 weeks. The study measures tolerability, dosing recommendations, and treatment response. Eligibility assessments include performance status and measurable disease, with safety and response evaluations continuing through both phases for up to three years.

Researchers are evaluating the safety of aerosolized RSP-1502 in people with cystic fibrosis who have chronic lung infections caused by Pseudomonas aeruginosa. This phase 1b/2a study compares different doses of RSP-1502 to an active control, aiming to find the maximum tolerated dose (MTD) and assess safety outcomes. Participants must meet specific lung function and infection criteria to join the study. The study involves administering RSP-1502 or an active control solution by inhalation using a nebulizer for 14 days. RSP-1502 contains tobramycin and CaEDTA in a sterile solution, while the active control is a tobramycin inhalation solution. After dose escalation to identify the MTD, a dose expansion phase compares the MTD of RSP-1502 to the active control for another 14 days. Participants will then be followed for 14 days after treatment ends. Participants will have their lung function tested with spirometry and undergo electrocardiograms on specific days during treatment. Researchers will monitor for any treatment-related adverse events and serious adverse events throughout the 28-day treatment and follow-up period. They will also track pulmonary exacerbations and other safety measures. The total participation includes dosing and a 14-day follow-up after treatment completion.

This research involves both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood and immune system. It focuses on using unlicensed cryopreserved cord blood units (CBUs) for transplantation, aiming to study how well these unlicensed CBUs support recovery after transplant. The study also looks at important outcomes such as infection transmission, infusion reactions, survival rates, and graft-versus-host disease. Participants will receive transplants using these unlicensed cord blood units as part of a multicenter access and distribution protocol. The study is conducted at multiple U.S. transplant centers under the care of transplant physicians. The transplantation process involves administering these CBUs to patients with hematologic malignancies and other relevant conditions. Patients will be monitored for neutrophil recovery at 60 and 100 days post-transplant to assess engraftment success. Researchers will also evaluate infection rates, serious infusion reactions, survival one year after transplant, and incidences of acute and chronic graft-versus-host disease. Platelet recovery will be tracked as well. The study involves regular assessments to follow patients’ health and transplant outcomes over time.

Researchers are evaluating the safety, tolerability, how the body processes, and effectiveness of TERN-701, a selective allosteric inhibitor targeting BCR-ABL1, in adults with chronic phase chronic myeloid leukemia (CP-CML) who have been previously treated. The study is divided into two parts: Part 1 focuses on dose escalation to find safe dosage levels, and Part 2 involves randomized dose expansion to further assess the chosen doses and includes a mutation cohort for participants with certain resistance mutations. Participants in both parts will take TERN-701 orally once daily in 28-day cycles. Part 1 involves sequential dose escalation cohorts, while Part 2 evaluates two recommended dose levels selected from Part 1. The mutation cohort (Part 2m) will assess a specific 500 mg dose in participants with particular resistance mutations. Scheduled visits occur frequently during the first treatment cycle and then regularly throughout the study to monitor treatment effects. During the study, participants will have regular visits for evaluations including safety checks and laboratory tests. Researchers will measure dose-limiting toxicities, adverse events, hematologic response, molecular response, and changes in BCR-ABL1 transcript levels up to three years. The trial plans to enroll about 180 participants, with up to 80 in Part 1, about 80 in Part 2, and around 20 in the mutation cohort. All participants will receive the active treatment throughout the study duration.

Researchers are studying BHV-1510, a Trop-2 directed antibody-drug conjugate, in people with previously treated, advanced solid tumors. This Phase 1/2 open-label trial aims to evaluate the safety, tolerability, and preliminary effectiveness of BHV-1510 alone and combined with cemiplimab. The study includes a Phase 1 dose escalation to find safe doses and a Phase 2 dose expansion to explore response rates in specific tumor groups. BHV-1510 is given in different schedules, including every 2 or 3 weeks, and sometimes on Days 1 and 8 every 3 weeks. Cemiplimab is administered by intravenous infusion at 350 mg on similar schedules when used in combination. The trial first determines recommended doses and maximum tolerated dose during Phase 1, then proceeds to Phase 2 for further evaluation of treatment response and safety in larger groups. Participants will be monitored for side effects, treatment response, and duration of response over approximately 47 months. Assessments include physical exams, blood tests, imaging to measure tumors, and safety evaluations. The study tracks adverse events and measures objective response rates to understand how well BHV-1510 works alone and with cemiplimab in advanced solid tumors.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of DISC-3405 in adults with sickle cell disease (SCD) in this Phase 1b open-label, multicenter study. The study focuses on participants who have either HbSC or HbSS types of SCD and have experienced various SCD-related complications, such as vaso-occlusive crises or organ complications. The purpose is to assess DISC-3405 at up to three different dose levels to understand its effects and safety profile in this population. DISC-3405 is administered as a subcutaneous injection. The study uses a within-participant dose-escalation design, meaning each participant will receive increasing doses of DISC-3405 to evaluate safety and tolerability at each level. The study will continue for up to 36 weeks to monitor the drug's effects. Participants must maintain stable doses of any current SCD treatments such as hydroxyurea during the study. Participants will be closely monitored throughout the study with assessments including blood tests to measure hemoglobin levels, iron status, and reticulocyte counts. Safety and tolerability will be tracked for up to 36 weeks. Additional evaluations will include pregnancy testing for females of childbearing potential and monitoring for any adverse events or complications related to SCD or the study drug. Participants must be able to understand and comply with the study procedures and provide informed consent to join the trial.

Researchers are studying the effects of NEU-411 in men and women aged 40 to 80 years who have early Parkinson's Disease (PD) with increased activity in the LRRK2 pathway, identified through a genetic test. This Phase 2 trial aims to evaluate how well NEU-411 works and its safety in this specific group compared to a placebo. The study involves participants with clinically established or probable PD and focuses on those with LRRK2-driven PD. Participants will be randomly assigned to receive either NEU-411, a brain-penetrant oral drug that inhibits LRRK2 activity, or a matching placebo once daily for 52 weeks. After completing this treatment phase, participants will have a safety follow-up visit within two weeks. Those eligible may join an open-label extension to receive NEU-411 for an additional 26 weeks. During the study, researchers will monitor changes in a digital biomarker score using a Parkinson's Disease app from enrollment through 52 weeks, along with recording any treatment-emergent or serious adverse events until the study ends at 54 weeks. Assessments include genetic testing, clinical evaluations of PD status, safety monitoring, and tracking of side effects to understand the drug's effects and tolerability over time.

Researchers are evaluating the effectiveness, safety, tolerability, and pharmacodynamics of multiple doses of APL-3007 combined with Syfovre/Pegcetacoplan (APL-2) in patients aged 60 years and older diagnosed with geographic atrophy secondary to age-related macular degeneration. This Phase 2, randomized, placebo-controlled, multicenter, masked study focuses on measuring changes in retinal pigment epithelium lesions using advanced artificial intelligence-based SD-OCT imaging. Participants will receive either the combination of APL-3007 with pegcetacoplan (APL-2) or a placebo. The study includes a treatment period with multiple doses administered, aiming to assess the impact on geographic atrophy lesions over a 12-month period. Syfovre injections at 6-8 week intervals prior to enrollment are part of the inclusion criteria. During the study, participants will undergo various eye imaging assessments such as OCT and FAF to monitor lesion size and progression. Researchers will evaluate changes in lesions at month 12 compared to baseline. Safety and tolerability will be closely monitored through laboratory tests, clinical evaluations, and vaccination status requirements. The study duration includes regular visits for treatment administration and monitoring over at least one year.