Boston, MA Paid Clinical Trials

The main purpose of this study is to: * Learn about the safety of REGN4018 and to find out what dose of REGN4018 can be given alone or with cemiplimab to patients with ovarian cancer or cancer of the uterus * The study will also look at the levels of REGN4018 and/or cemiplimab in your body and measure how well your body can remove the study drug(s). This is called pharmacokinetics * The study will also look at any signs that REGN4018 alone or with cemiplimab can treat recurrent advanced ovarian cancer or cancer of the uterus * To find out how safe and tolerable the sarilumab pretreatment is, in combination with REGN4018 and cemiplimab Read Less

The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options. Read Less

The goal of this clinical trial is to evaluate the efficacy, safety and pharmacokinetics of elamipretide in subjects with dry age-related macular degeneration (AMD). The main questions it aims to answer are: what is the rate of change in the macular area of photoreceptor loss in subjects who receive a daily dose of elamipretide compared with those who receive a look-alike substance that contains no active drug, and what is the safety and tolerability of elamipretide daily subcutaneous injections. Participants will receive either once daily subcutaneous doses of 40mg elamipretide or placebo and the two treatment groups will be compared. Read Less

The primary objective is to determine the safety and efficacy of belzupacap sarotalocan (bel-sar) compared to sham control in patients with primary indeterminate lesions (IL) or small choroidal melanoma (CM). Read Less

This double-blind, randomized, placebo-controlled, multinational, multicenter, parallel-group, Phase 3, 2-arm, study will investigate the efficacy and safety of belumosudil compared with placebo, both administered on top of azithromycin and standard-of-care regimen of immunosuppression in male or female participants at least 1 year after bilateral lung transplant, who are at least 18 years of age and who have evidence of progressive CLAD despite azithromycin therapy. Study details include: The study duration will be up to 31 weeks for participants not entering the open-label extension (OLE) period and up to 57 weeks for participants entering the OLE period but not the long-term OLE. The treatment duration will be up to 26 weeks for participants not entering the OLE period and up to 52 weeks for participants entering the OLE period but not the long-term OLE. The number of visits will be up to 10 visits for participants not entering the OLE period and up to 16 visits for participants entering the OLE period but not the long-term OLE. For participants who enter the long-term OLE, treatment and study participation will continue with visits every 12 weeks per protocol specifications. Read Less

Ketamine, an NMDA antagonist, has been shown to have rapid anti- suicidal effects. However, its safety and efficacy and special populations has not been investigated and documented. Several reports in adults suggest rapid decrease of suicidal ideation. In the last decade there is an alarming increase of the number of suicide attempts in patients ages 14-30. Suicide is the second leading cause of death in this population. Patients with previous history of suicide attempt, are even in a higher risk category. The present study focus in this high risk group of suicide attempters. This will be a randomized controlled trial enrolling 62 youth between the ages 14-30 after a suicide attempt; patients will be randomized to receive Ketamine 0.5 mg/kg over 40 minutes or normal saline. Patients will receive all Ketamine or placebo infusions while admitted in the Inpatient Psychiatry Service. Patients will receive up to 6 ketamine or placebo infusions until, for 3 consecutive sessions, they have a clinician rated Scale for Suicidal Ideation (SSI) score of\<4, and \>50% decrease from baseline, and clinical assessment of patient not being suicidal, or they have been discharged from the inpatient unit. Patients will participate in weekly sessions of Collaborative Assessment for the management of Suicidality (CAMS), from the first week of the study while admitted to the hospital and will continue it on a weekly basis post-discharge until the patient has three consecutive outpatient CAMS sessions with an overall risk \< 2 (# 6 on the SSF Core Assessment) along with a positive response regarding their thoughts/feelings and clinician indicating behavioral stability (suicidal behavior). Read Less

This first-in-human (FIH) dose-escalation and dose-validation/expansion study will assess ziftomenib, a menin-MLL(KMT2A) inhibitor, in patients with relapsed or refractory acute myeloid leukemia (AML) as part of Phase 1. In Phase 2, assessment of ziftomenib will continue in patients with NPM1-m AML. Read Less

This is a Phase 3, open-label, randomized trial designed to evaluate the RFS of TURBT followed by cretostimogene grenadenorepvec versus TURBT followed by observation for the treatment of participants with IR-NMIBC. Read Less

The main purpose of this study is to learn more about the safety, side effects, and effectiveness of LOXO-435 by itself or when it is combined with other standard medicines that treat cancer. LOXO-435 may be used to treat cancer of the cells that line the urinary system and other solid tumor cancers that have a change in a particular gene (known as the FGFR3 gene). Participation could last up to 30 months (2.5 years) and possibly longer if the disease does not get worse. Read Less

The primary purpose of the study is to understand how well the study drug can eliminate abnormal plasma cells and laboratory signs of high-risk monoclonal gammopathy of undetermined significance (HR-MGUS) and non high-risk smoldering multiple myeloma (NHR-SMM). This requires understanding the safety and tolerability of the study drug (how the body reacts to linvoseltamab) as well as the effectiveness of the study drug (how well linvoseltamab eliminates plasma cells). All participants will start treatment with gradually increasing doses of linvoseltamab (step-up doses) before they start receiving the assigned full dose. The study is split into 2 parts: * In Part 1, separate groups of 3-6 patients will receive different full doses of linvoseltamab to evaluate the short-term side effects (safety) and tolerability of the study drug within the first 5 weeks after starting treatment. The data collected will help to make a decision about the dosing regimens chosen for Part 2. * In Part 2, a larger number of participants will be randomized to different dosing regimens to further assess the side effects of linvoseltamab, and to evaluate the ability of linvoseltamab to eliminate abnormal plasma cells in HR-MGUS and NHR-SMM. The study is looking at several other research questions, including: * How many participants treated with linvoseltamab have improvement of their HR-MGUS or NHR-SMM? * What side effects may happen from taking the study drug? * How much study drug is in the blood at different times? * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects). Read Less

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Read Less

The purpose of this Phase 1/2 master protocol study is to evaluate if DCC-3009 is safe, tolerable and works effectively in the treatment of GIST. The study will use a modular approach with each module being defined according to therapy: DCC-3009 alone or DCC-3009 in combination with other anticancer therapies. Each module will be conducted in 2 parts: Part 1 (Dose Escalation) and Part 2 (Dose Expansion). Participants will be treated in 28-day treatment cycles with an estimated duration of up to 2 years. Read Less