Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.

Type: Observational [Patient Registry]

Start Date: Aug 2026

open study

This clinical trial studies whether a navigation program improves survivorship support service participation among survivors of breast cancer that has not spread from where it first started (primary site) to other places in the body (non-metastatic). Advances in treatment have caused the number of breast cancer survivors to grow. As this number increases, there are reported unmet supportive care needs in this population, including psychological distress and limitations in physical functioning. To address these needs, many cancer centers offer programming on a variety of topics including psychological services, exercise counseling, and nutrition counseling. Research has shown that while interest in these survivorship programs is high, participation remains low, especially among minority women. Navigation is a healthcare service that is designed to guide a patient through the healthcare system and reduce barriers to timely screening, follow-up, diagnosis, treatment, and supportive care. The navigation program in this trial is specifically focused on helping breast cancer survivors schedule and attend survivorship consultation appointments as well as providing additional support to underserved/vulnerable patients. A navigation program may be effective in improving survivorship support service participation among non-metastatic breast cancer survivors.

Type: Interventional

Start Date: Sep 2026

open study

This is an interventional, randomized, parallel group, treatment, Phase 3, double blind study to assess the effect of Rezpegaldesleukin in participants 12 years of age or older with moderate to severe atopic dermatitis, as compared to placebo. The estimated participant overall duration is approximately 15 months.

Type: Interventional

Start Date: Jun 2026

open study

Smell dysfunction significantly impacts quality of life and safety, with limited effective treatments. This open-label study evaluates the preliminary efficacy of combining non-invasive trigeminal nerve stimulation (TNS) with standard smell training (ST) to improve olfactory function. Participants will complete 8 weeks of at-home treatment and attend three in-person visits for assessment. Improvements in smell, mood, sleep, and quality of life will be measured.

Type: Interventional

Start Date: Jul 2026

open study

This is a randomized, placebo-controlled Phase 2 study to evaluate the efficacy and safety of SAR448851 in early Alzheimer's disease (AD) participants. The purpose of this study is to measure efficacy and safety with once daily oral SAR448851 compared to placebo in participants with mild cognitive impairment due to AD or mild AD dementia and with evidence of cerebral amyloid pathology. This Phase 2 study has 2 parts: Part A is a randomized, double-blind, parallel-group, placebo-controlled study with SAR448851 oral once daily. Part B is an open-label extension. All participants who complete Part A may continue to Part B. An optional dose 2 cohort will be considered to evaluate the efficacy and safety of SAR448851 dose 2 oral once daily. The study duration will be up to 111 weeks for Part A and B, and up to 63 weeks for the dose 2 cohort. The treatment duration will be up to 96 weeks for Part A and B, and up to 48 weeks for the dose 2 cohort. Up to 160 participants will be included in this study.

Type: Interventional

Start Date: Jul 2026

open study

This study will assess how different oral formulations of ubrogepant move through the body in healthy adult participants under fasting and fed conditions.

Type: Interventional

Start Date: Jul 2026

open study

This research is studying the use of low-intensity focused ultrasound (LIFU; a mild, noninvasive acoustic stimulation technique) in a small number of people to learn about its safety as a treatment for stuttering. LIFU is a small, safe sound signal that produces a gentle, pulsing flow of acoustic waves to help different parts of the brain communicate with each other. Researchers want to understand how the mild, non-invasive brain stimulation affects speech relevant brain areas, which may in turn affect speech fluency and speaking-related brain activity in people who stutter.

Type: Interventional

Start Date: Aug 2026

open study

The study is testing UBT251 in participants with type 2 diabetes. The purpose of this clinical study is to find out if UBT251 is effective and safe for treating participants with type 2 diabetes. Participants will either get UBT251, UBT251 placebo, semaglutide, or semaglutide placebo. Which treatment participants get is decided by chance. UBT251 is the treatment being tested and is not yet available for doctors to prescribe, while semaglutide is a medicine used to treat type 2 diabetes that doctors can already prescribe.

Type: Interventional

Start Date: Jun 2026

open study

This is an observational umbrella protocol evaluating toxicities after CAR-T therapy with ciltacabtagene autoleucel (cilta-cel) for RRMM, with a goal to identify key inflammatory features contributing to toxicities, define non-invasive biomarkers to guide clinical monitoring, and evaluate treatment strategies to reduce morbidity for patients. Toxicities of interest will include neurotoxicity, hematologic, and gastrointestinal events. Patients planned to receive cilta-cel as part of their standard of care multiple myeloma therapy will be enrolled. All patients will have baseline evaluation at the time of leukapheresis and cilta-cel infusion, as well as longitudinal blood, bone marrow, cerebrospinal fluid (CSF), and gastrointestinal (GI) samples collected for translational assessment. Patients who experience toxicities of interest as evaluated by their clinical team will undergo additional evaluation and sample collection, as guided by the involved organ system (e.g. CSF for neurologic toxicity, endoscopic evaluation with colonic biopsies for colitis), with monitoring for resolution of symptoms on therapy. Additional patients from Mount Sinai or other centers [University of California San Francisco (UCSF), Memorial Sloan Kettering Cancer Center (MSKCC)] who have previously been or will be treated with cilta-cel and are participating in institutional biobanks will similarly be included for ongoing sample collected per local protocols, and samples from patients experiencing toxicities of interest will be sent to Mount Sinai for analysis to supplement the prospective cohort.

Type: Observational

Start Date: Jul 2026

open study

The purpose of this study is to evaluate the efficacy, safety, and tolerability of elecoglipron, compared with placebo in adults with type 2 diabetes mellitus (T2DM) and impaired renal function, who are or will be on a background of sodium-glucose cotransporter 2 inhibitor (SGLT2i) dapagliflozin 10 mg as per guideline directed medical therapy (GDMT) for chronic kidney disease (CKD). Additionally, participants are on other glucose-lowering medication(s).

Type: Interventional

Start Date: Jul 2026

open study

The primary objective of the study is to assess the pharmacokinetics (PK) and safety of nemolizumab in pediatric participants (aged 6-23 months) with moderate-to-severe atopic dermatitis (AD) who are not adequately controlled with topical treatments.

Type: Interventional

Start Date: Jul 2026

open study

Alzheimer's Disease (AD) is associated with impairments in both gait and cognition, significantly increasing fall risk. Falls are a leading cause of injury-related disability in older adults, and individuals with AD experience a nearly threefold higher rate of falls compared to neurotypical older adults. There is an urgent need for fall prevention interventions tailored to the unique deficits of individuals with AD. Converging evidence suggests that interventions aiming to reduce fall risk in AD should target both gait and cognition. Rhythmic music interventions, such as Rhythmic Auditory Stimulation (RAS) can harness global brain activation and auditory-motor entrainment to facilitate high-intensity exercise to alleviate AD-related neurocognitive and gait dysfunction. This study aims to assess the neural correlates of gait dysfunction in people with AD, evaluate if baseline neurocognitive impairment is predictive of the effects of RAS, and evaluate RAS benefits for individuals with AD.

Type: Interventional

Start Date: Jun 2026

open study

The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

Type: Interventional

Start Date: Jul 2026

open study

This is a double-blind, randomized, multicenter, outpatient evaluation of the efficacy, safety, and tolerability of elismetrep, as compared with placebo, in the acute treatment of migraine.

Type: Interventional

Start Date: Jul 2026

open study

This study will assess the safety and efficacy of VS-7375 alone and in combination with cetuximab in patients with metastatic KRAS G12D - mutated Pancreatic Cancer

Type: Interventional

Start Date: Jun 2026

open study

The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.

Type: Interventional

Start Date: Aug 2026

open study

Researchers are looking for new ways to treat children with relapsed or refractory solid tumors: - Relapsed means the cancer came back after treatment - Refractory means the cancer did not respond (get smaller or go away) to treatment - Solid tumors are cancers mostly in body organs and tissues, not in the blood or other body liquids The study treatment I-DXd (also known as MK-2400 or ifinatamab deruxtecan) is an antibody-drug conjugate (ADC). An ADC attaches to a protein on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn: - About the safety of I-DXd and if children younger than 12 years old tolerate it - How many children who receive I-DXd have the cancer get smaller or go away

Type: Interventional

Start Date: Jul 2026

open study

The goal of this clinical trial is to evaluate the safety and efficacy of privosegtor, a neuroprotective candidate, in patients diagnosed with optic neuritis (ON). Researchers will compare privosegtor and the standard of care (methylprednisolone) to a placebo and standard of care (methylprednisolone).

Type: Interventional

Start Date: May 2026

open study

This study is being conducted to evaluate INCA033989 versus best available therapy in participants with essential thrombocythemia and a CALR mutation previously treated with cytoreductive therapy.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this research study is to examine how well a medication called lumateperone (Caplyta) works to relieve depression in older adults with treatment-resistant depression. Lumateperone (Caplyta) is approved by the U.S. Food and Drug Administration to treat Major Depressive Disorder in adults who are also taking another antidepressant medication. This study will compare lumateperone (Caplyta) to placebo (a sugar pill without medication).

Type: Interventional

Start Date: Aug 2026

open study

The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time. This study intends to collect information from participants diagnosed with fucosidosis; however, this study does not include any medication or treatment other than the usual medical care provided to study participants. The information collected in this study will be used to help understand the disease characteristics of fucosidosis; with this information potentially being able to help design future studies and treatments for this disease. There is currently no approved treatment for patients with fucosidosis. The study consists of 2 parts: a) Part A - retrospective data collection, and b) Part B - prospective data collection.

Type: Observational

Start Date: Mar 2026

open study

The goal of this study will be piloting the Parents and Babies perinatal depression prevention intervention to examine its feasibility and acceptability and to obtain preliminary data on its efficacy. This includes exploring/understanding users' experiences navigating the interventions (e.g., from an implementation perspective) and preliminary evaluations of clinical outcomes, including mental health and minority stress. With data gathered from this pilot study, we will make final adaptations to the Parents and Babies manuals and plan to develop a randomized-control trial to evaluate efficacy of the intervention.

Type: Interventional

Start Date: Aug 2026

open study

The goal of this clinical trial is to investigate biomarkers of non-IgE-mediated immediate hypersensitivity reactions during infusion of intravenous vancomycin. The main question it aims to answer is: • Identifying novel biomarkers in blood that occur during infusion reaction Participants will: - Have allergy skin testing for vancomycin - Receive an infusion of vancomycin

Type: Interventional

Start Date: May 2026

open study

This is a phase 2b, multicenter, randomized, double-blind, placebo-controlled study.

Type: Interventional

Start Date: May 2026

open study

The goal of this clinical trial is to learn if a single dose of the study drug, JST-018, is safe and tolerable when administered by injection into the arm or thigh muscle of healthy men and women aged 18 to 55. The main questions it aims to answer are: - Is a single administration of JST-018 safe? - What is the concentration of the JST-018 in the blood over time? - Do antibodies to JST-018 develop following a dose of JST-018? Researchers will compare JST-018 to Placebo to see if there are any differences in the safety and tolerability of a single dose at different dose levels. Participants will be confined to the clinic for the first 3 days. They will receive an injection on the second day, and then return for 9 more visits over the period of 1 year for: - Physical exam with vital signs - Electro-cardiogram (ECG) - Bood collection for clinical labs and research samples - Urine sample - Assessment of potential adverse effects and medications taken

Type: Interventional

Start Date: Jun 2026

open study