Mass General - Division of Clinical Research

Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.

Type: Observational

Start Date: Feb 2015

open study

The proposed research will test the hypothesis that objective social isolation and loneliness are linked to neurobehavioral mechanisms involved in social perception and motivation in individuals with and without serious mental illness. Moreover, it will investigate the specific dynamic interactions among these experiences in daily life and how they, and their neurobehavioral predictors, are linked to day-to-day functioning. The findings of this project could provide novel targets for therapeutics aimed at improving functioning and overall quality of life in individuals with serious mental illnesses, as well as quantitative phenotypes for use in early detection efforts.

Type: Interventional

Start Date: Jul 2022

open study

This research study is studying a combination of drugs as a possible treatment for Hepatocellular Carcinoma or Biliary Tract Cancer. The interventions involved in this study are: - Durvalumab - Tremelimumab - Radiation Therapy

Type: Interventional

Start Date: May 2018

open study

The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.

Type: Interventional

Start Date: Nov 2019

open study

Patients with metastatic breast cancer who are approaching the end of life often experience high symptom burden and acute hospitalizations without sufficient communication with care teams about their end-of-life care wishes or the support of palliative or hospice services. The purpose of this multi-site trial is to test the efficacy of a targeted, five-session palliative care intervention designed specifically for those with poor prognosis metastatic breast cancer and their caregivers to improve communication with clinicians and the quality of end-of-life care. This study will lay the groundwork for ultimately delivering scalable, timely, and tailored palliative care interventions for patients with advanced cancer who have long disease trajectories.

Type: Interventional

Start Date: Oct 2025

open study

The purpose of this trial is to evaluate the safety, tolerability, pharmacokinetics (PK), anti-tumor activity and immunogenic potential of GEN1286 in participants with advanced solid tumors. This trial consists of 2 parts: - Part A: Dose escalation and dose level expansion - Part B: Tumor-specific expansion with dose optimization

Type: Interventional

Start Date: Nov 2024

open study

The main purpose of the study is to assess whether the study drug, LY4066434, is safe and tolerable when administered to participants with locally advanced or metastatic solid tumors with certain KRAS mutations. LY4066434 will be given alone or in combination with other treatments. The study will have 2 parts: monotherapy dose escalation and dose optimization. The study is expected to last up to approximately 5 years.

Type: Interventional

Start Date: Oct 2024

open study

The main purpose of this study is to find out whether the study drug, LY4050784, is safe, tolerable and effective in participants alone or in combination with other anticancer agents. In addition, with locally advanced or metastatic solid tumors with a BRG1 (Brahma-related gene 1, also known as SMARCA4) alteration who have previously received, do not qualify for, or are refusing standard of care treatments, or there is no standard therapy available for the disease. The study is conducted in two parts - phase Ia (dose-escalation) and phase Ib (dose-optimization, dose-expansion). The study will last up to approximately 4 years.

Type: Interventional

Start Date: Sep 2024

open study

The goal of this pilot clinical trial is to test efgartigimod alfa against placebo in adults with first-time optic neuritis (optic nerve inflammation). The main questions it aims to answer are: - Is it feasible to use efgartigimod alfa for optic neuritis? - Is it feasible to run a larger trial testing efgartigimod alfa in optic neuritis? - Does efgartigimod alfa work better than placebo in improving how quickly and how much vision returns? Participants will: - have their vision and blood tested - be asked questions about their vision - will receive standard of care treatment with steroids regardless of whether they are receiving efgartigimod alfa or not - will have periodic visits over 6 months

Type: Interventional

Start Date: Aug 2025

open study

Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). This study will assess how safe and effective Upadacitinib is in treating pediatric participants with UC. Adverse events and change in disease activity will be assessed. Upadacitinib (RINVOQ) is a drug approved in adults for moderate- to severely active UC and is being developed for moderate- to severely active UC in pediatric participants. This study is conducted in 2 periods: Period 1 is comprised of two phases: an 8-week open-label induction phase which means that the study doctor and patients know that participants will receive UPA Dose-A (or the adult equivalent based on body weight) followed by a 44-week double-blind maintenance phase meaning that neither the participants nor the study doctors will know which dose of upadacitinib will be given(UPA Dose B or Dose C). Period 2 is a 260 week open-label extension (OLE) of Period 1. Approximately 110 pediatric participants with moderate to severely active UC will be enrolled at up to 100 sites worldwide. Participants will receive upadacitinib oral tablets once daily or oral solution twice daily at approximately the same time each day, with or without food. Participants will be followed up for 30 days after each phase (i.e. after induction, maintenance, OLE) and only if a participant doesn't continue into the next phase. 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. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Type: Interventional

Start Date: Nov 2023

open study

The goal of this clinical trial is to assess the efficacy, safety and tolerability of the combination of lasofoxifene and abemaciclib compared to fulvestrant and abemaciclib for the treatment of pre- and postmenopausal women and men who have previously received ribociclib or palbociclib-based treatment and have locally advanced or metastatic estrogen receptor positive (ER+)/human epidermal growth factor 2 negative (HER2-) breast cancer with an estrogen receptor 1 (ESR1) mutation. The main question the study aims to answer is: • To compare the efficacy of the combination of lasofoxifene and abemaciclib with that of fulvestrant and abemaciclib Participants will receive either receive 5 mg/d of oral lasofoxifene plus oral abemaciclib 150 mg twice a day or the combination of fulvestrant 500 mg intramuscular (IM) on Days 1, 15, and 29 and then once monthly thereafter plus oral abemaciclib 150 mg twice a day.

Type: Interventional

Start Date: Oct 2023

open study

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.

Type: Interventional

Start Date: Jan 2023

open study

The primary goal of the trial is to determine if the experimental arms (rivaroxaban or ticagrelor or both) are superior to the clopidogrel arm for lowering the 1-year rate of ischemic stroke, intracerebral hemorrhage, or vascular death.

Type: Interventional

Start Date: Aug 2022

open study

Phase I of this research study will assess what doses of Sacituzumab Govitecan and Enfortumab Vedotin can be safely combined in the treatment of metastatic urothelial carcinoma (mUC). In Phase II of the study, patients in one of the two cohorts will receive Sacituzumab Govitecan, Enfortumab Vedotin, and Pembrolizumab to assess the efficacy of this drug combination. The names of the study drugs in these investigational combinations are: - Enfortumab Vedotin - Sacituzumab Govitecan - Pembrolizumab

Type: Interventional

Start Date: May 2021

open study

The study is a global, multi-center safety and efficacy trial of epcoritamab, an antibody also known as EPKINLY™ and GEN3013 (DuoBody®-CD3xCD20). Epcoritamab will be tested either in Relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) as: - Monotherapy, or - Combination therapy: - epcoritamab + venetoclax - epcoritamab + pirtobrutinib In Non-United States (US) Participants Only: Treatment-naïve (TN) high risk (HR) (CLL): • epcoritamab + pirtobrutinib Combination therapy for Richter's Syndrome (RS): - epcoritamab + lenalidomide - epcoritamab + R-CHOP (i.e., rituximab, cyclophosphamide, doxorubicin hydrochloride, vincristine [Oncovin®] and prednisone). The study includes participants with R/R or TN HR CLL (non-US participants only)/small lymphocytic lymphoma (SLL) and participants with RS. The trial consists of two parts, a dose-escalation phase (phase Ib) and an expansion phase (phase II). Participants with RS are only included in the expansion phase. Epcoritamab will be injected subcutaneously (under the skin). Standard-of-care and combination treatments (venetoclax, pirtobrutinib, lenalidomide, and R-CHOP) will be given either orally (by mouth) or intravenously (in a vein). Study details include: - Study duration will be up to 5 years after the last participant's first treatment in the trial. - The treatment duration for each participant will be between 12 months (1 year) and 24 months (2 years), depending upon the treatment arm assigned. - The visit frequency will be either weekly, every other week, or monthly, depending upon the part of the study. All participants will receive active drug; no one will be given placebo.

Type: Interventional

Start Date: Nov 2020

open study

We have developed novel Brillouin microscopy and we are testing its potential for keratoconus and ectasia diagnostics. We plan to perform axial scans of the cornea in human volunteers in order to compare biomechanical properties of Keratoconus vs. Normal corneas and compare biomechanical properties of post-LASIK ectasia vs. normal corneas.

Type: Observational

Start Date: Jun 2013

open study

The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin amyloidosis with cardiomyopathy (ATTR-CM), and in control subjects without heart failure or cardiomyopathy. The main question it aims to answer is: • Can SWV be used to measure myocardial and liver stiffness in the study's target populations? Researchers will compare patients with ATTR-CM, patients without ATTR-CM, and a control group to determine the distributions of SWV in each population. Participants will: - Have a standard cardiology assessment, including a physical exam, blood work, and an echocardiogram. - Undergo an investigational assessment with the eMyosound LYRA device to measure SWV in their myocardium and liver. - Have the investigational assessment repeated by a second observer to assess measurement reliability.

Type: Observational

Start Date: Sep 2025

open study

The purpose of this study is to pilot two resilience interventions for cardiac arrest survivors and their informal caregivers, Recovering Together after Cardiac Arrest 1 and Recovering Together after Cardiac Arrest 2 . The data the investigators gather in this study will be used to further refine the interventions.

Type: Interventional

Start Date: Oct 2025

open study

Study MTR-601-201 is an 8-week, randomized, placebo-controlled study to examine the safety, tolerability, and efficacy of MTR-601 in participants with cervical dystonia.

Type: Interventional

Start Date: Feb 2025

open study

This is a Phase 2 dose-finding study in adult participants with symptomatic HFpEF.

Type: Interventional

Start Date: Feb 2025

open study

This study is researching an experimental drug called ubamatamab, also referred to as "study drug". The study is focused on patients who have advanced ovarian cancer. The aim of the study is to see how safe, tolerable, and effective the study drug is on its own and in combination with other anti-cancer drugs (bevacizumab, cemiplimab, fianlimab and a standard chemotherapy drug, pegylated liposomal doxorubicin [PLD]), referred to as "combination drugs'. The study is looking at several other research questions, including: - What side effects may happen from taking the study drug and its experimental combinations - How much study drug and fianlimab 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) and its combinations

Type: Interventional

Start Date: May 2025

open study

This is a multi-center, hospital based, cross-sectional study based on data from patient medical records, including laboratory results. The study will include adult patients with chronic (> 1 year duration) primary Immune Thrombocytopenia (ITP) only. Patients will be identified based on a laboratory confirmed diagnosis of ITP in the medical records, where there is also a physician-confirmed diagnosis of primary ITP. If patients consent to take part in the study, a routine clinical visit will also serve as the study visit. All study participants will have a routine blood test as part of their standard clinical care at the study visit, and this same procedure will be used to collect supplementary blood samples to assess a variety of biomarkers. Data will be collected using a combination of chart review, clinical outcome assessment administration, and laboratory results through blood tests.

Type: Observational

Start Date: Jan 2025

open study

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

Type: Interventional

Start Date: Apr 2025

open study

This study will compare the effects of Quizartinib versus placebo in combination with chemotherapy in participants with newly diagnosed FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) negative acute myeloid leukemia (AML).

Type: Interventional

Start Date: Nov 2024

open study

The purpose of this study is to evaluate the safety, tolerability, dosimetry and preliminary efficacy of [177Lu]Lu-NNS309 and the safety and imaging properties of [68Ga]Ga-NNS309 in patients aged ≥ 18 years with locally advanced or metastatic pancreatic ductal adenocarcinoma (PDAC), non-small cell lung cancer (NSCLC), HR+/HER2- ductal and lobular breast cancer (BC), triple negative breast cancer (TNBC) and colorectal cancer (CRC).

Type: Interventional

Start Date: Oct 2024

open study