Mass General - Division of Clinical Research

Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.

Type: Observational

Start Date: Sep 2015

open study

This clinical trial is being conducted to learn about safety and tolerability of digoxin in ALS individuals. Additionally, this trial aims to better understand if digoxin has an effect on slowing neurodegeneration in ALS.

Type: Interventional

Start Date: May 2025

open study

The goal of this research is to learn how a new device called the wireless thermal capsule (WTC) can collect thermal data to help see diseases that happen in the gastrointestinal (GI) tract, such as Crohn's Disease.

Type: Interventional

Start Date: Jun 2025

open study

The goal of this clinical trial is to learn if a new screening approach including an artificial intelligence algorithm that analyzes fundus photographs, measurement of eye pressure and visual field testing works to screen for glaucoma. Participants will: Have an image of their fundus (back of the eye) taken as part of their diabetic eye screening Have a measurement of their eye pressure If needed, have a test of their side vision using a headset

Type: Interventional

Start Date: Jul 2025

open study

The complex and unclear relationship between language and executive function (EF) creates barriers to developing effective interventions for children with developmental language disorder (DLD) whose language difficulties often co-occur with impaired EF. Children and adults with typical language development (TD) facilitate their EF by using self-directed language, or verbal mediation, to guide conscious reflection and override habitual behaviors. Conversely, children with DLD do not use verbal mediation to support EF efficiently or effectively. Promising evidence suggests that language-based training can shape verbal mediation and improve EF task performance in children with TD, which makes it pertinent to determine whether verbal mediation training benefits children with DLD. Specifically, modeling interventions have been shown to promote learning of language forms without taxing the cognitive resources required for learning such as attention or working memory, which are known to be impaired among children with DLD. The long-term goal of the proposed work is to optimize intervention outcomes for children with DLD by elucidating the complex relationship between language and executive functions. The objective of this project is to determine the impact of modeling verbal mediation on shifting task performance in school-aged children with DLD. Shifting, also known as cognitive flexibility, is the ability to alternate between operations or mental sets. It is an important EF because it is the pivot point between multiple goal-directed tasks when language use is critical for guiding action. Children aged 8-10 years will complete three versions of a shifting task over three phases: pre-intervention, intervention, and post-intervention. During the intervention phase, half of the participants with DLD will be exposed to a task model with verbal mediation (training), while the other half will be exposed to a silent task model (control). The investigators will determine the effect of modeling verbal mediation on the subsequent use of verbal mediation (Aim 1) and behavioral and electrophysiological measures of shifting ability (Aim 2). Indirect measures of shifting (i.e., accuracy and reaction time) will be supplemented with an electrophysiological marker of shifting that reflects real-time cue processing. This combination of methods provides insight to changes in processing following intervention that may precede and predict subsequent changes in behaviors. Our central hypothesis is that modeling verbal mediation will facilitate more effective use of verbal mediation and improve shift cue processing in children with DLD. The project will provide a theoretical framework for the role of language in shaping goal-directed behavior and the first examination of electrophysiological change in shifting following a verbal mediation intervention. Results will have a significant impact on clinical practice by advancing knowledge about a promising language-based intervention to support EF and other goal-directed tasks.

Type: Interventional

Start Date: Jul 2025

open study

A Phase 2 study to evaluate the safety and efficacy of TORL-1-23 in patients with advanced ovarian cancer.

Type: Interventional

Start Date: Nov 2024

open study

This Phase 3 study is an Open Label Extension of the APG-20 Study To Evaluate the Long-term Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects with Partial Lipodystrophy

Type: Interventional

Start Date: Oct 2024

open study

First in human study to evaluate the safety, tolerability, and pharmacokinetics (PK) of BBO-10203, a PI3Kα:RAS breaker, alone and in combination with other anti-cancer agents in patients with advanced solid tumors.

Type: Interventional

Start Date: Oct 2024

open study

AJX-101 is a first-in-human (FIH), phase 1, non-randomized, multi-center, open-label clinical trial designed to investigate the safety, tolerability, pharmacokinetics (PK), clinical activity and changes in biomarkers of an orally administered type II JAK2 inhibitor, AJ1-11095, in subjects with primary or secondary myelofibrosis previously treated with at least one type I JAK2 inhibitor.

Type: Interventional

Start Date: Oct 2024

open study

The purpose of this study is to evaluate the effectiveness and safety of Arlocabtagene Autoleucel (BMS-986393) in participants with relapsed or refractory multiple myeloma.

Type: Interventional

Start Date: Mar 2024

open study

The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG).

Type: Interventional

Start Date: Mar 2024

open study

This is a trial in which 350 primary lung transplant recipients will be randomized (1:1) to receive either Tocilizumab (six doses over 20 weeks) plus standard triple maintenance immunosuppression or placebo (sterile normal saline) plus standard triple maintenance immunosuppression (Tacrolimus, Mycophenolate Mofetil, corticosteroids). The primary objective is to test the hypothesis that treatment with triple maintenance immunosuppression plus Tocilizumab (TCZ) is superior to triple maintenance immunosuppression plus placebo (saline) as defined by a composite endpoint of a) CLAD, b) listed for re-transplantation, and c) death

Type: Interventional

Start Date: Feb 2024

open study

This pilot study in healthy volunteers aims to determine if biological sex has an impact on recovery from dexmedetomidine-induced unconsciousness, and if transcranial magnetic stimulation combined with electroencephalography (TMS-EEG) can be used to measure brain complexity during dexmedetomidine sedation without arousing study participants.

Type: Interventional

Start Date: Nov 2024

open study

The purpose of this Ph2b study is to characterize the dose-response relationship and to evaluate the safety and efficacy of three different single doses of TIN816 in hospitalized adult participants in an intensive care setting with a diagnosis of sepsis-associated acute kidney injury (SA-AKI).

Type: Interventional

Start Date: Jan 2024

open study

The goal of this clinical trial is to demonstrate that the OPTIMIZER® Integra CCM-D System (the "CCM-D System") can safely and effective convert induced ventricular fibrillation (VF) and spontaneous ventricular tachycardia and/or ventricular fibrillation (VT/VF) episodes in subjects with Stage C or D heart failure who remain symptomatic despite being on guideline-directed medical therapy (GDMT), are not indicated for cardiac resynchronization therapy (CRT), and have heart failure with reduced left ventricular ejection fraction (LVEF ≤40%). Eligible subjects will be implanted with the CCM-D System. A subset of subjects will be induced into ventricular fibrillation "on the table" in the implant procedure room. During the follow-up period, inappropriate shock rate and device-related complications will be evaluated. The follow-up period is expected to last at least two years.

Type: Interventional

Start Date: May 2023

open study

This Phase 1 study will assess the safety, tolerability, and preliminary antileukemic activity of ziftomenib in combination with venetoclax and azacitidine (ven/aza), ven, and 7+3 for two different molecularly-defined arms, NPM1-m and KMT2A-r.

Type: Interventional

Start Date: Jul 2023

open study

The goal of this follow-up study is to learn about long-term patient survival and graft function in highly sensitized patients who have received desensitization treatment with imlifidase or standard of care (SoC) in order to enable kidney transplantation in clinical study ConfIdeS (20-HMedIdeS-17, NCT04935177).

Type: Observational

Start Date: Apr 2023

open study

This is a first-in-human, Phase 1/2, open-label, dose escalation, dose expansion and combination study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of Peluntamig (PT217) as a monotherapy and in combination with chemotherapy.

Type: Interventional

Start Date: Sep 2023

open study

The goal of this observational study is to evaluate the Thoraflex Hybrid device alone and in combination with the RelayPro NBS stent-graft in the treatment of aortic disease affecting the aortic arch and descending aorta with or without involvement of the ascending aorta. Patients who undergo treatment with the Thoraflex Hybrid device with or without extension with a RelayPro NBS stent-graft will be eligible for enrolment and study activities and follow-up regime will follow standard care at each participating site. Participant involvement in the study will last for a total of 10 years from the point at which the Thoraflex Hybrid device is placed.

Type: Observational

Start Date: Mar 2023

open study

The main purpose of this study is to measure how well imlunestrant works compared to standard hormone therapy in participants with early breast cancer that is estrogen receptor positive (ER+) and human epidermal receptor 2 negative (HER2-). Participants must have already taken endocrine therapy for two to five years and must have a higher-than-average risk for their cancer to return. Study participation could last up to 10 years.

Type: Interventional

Start Date: Oct 2022

open study

This is a 2-part (phase 2b/3) prospective, interventional, multicenter, randomized, double-blind, placebo-controlled study. Part 1 (phase 2b) is a dose-finding study for CSL300 vs placebo. Part 2 (phase 3) aims to assess the efficacy of CSL300 on cardiovascular (CV) outcomes and safety in subjects with systemic inflammation and either atherosclerotic cardiovascular disease (ASCVD) or diabetes with end stage kidney disease (ESKD) undergoing maintenance dialysis.

Type: Interventional

Start Date: Oct 2022

open study

This is a global Phase 2, open-label, single-arm, multicohort, multicenter study to evaluate efficacy and safety of JCAR017 in adult subjects with r/r FL or MZL. The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This study is divided into three periods: - Pretreatment, which consists of screening assessments, leukapheresis and the Pretreatment evaluation; - Treatment, which starts with the administration of lymphodepleting (LD) chemotherapy and continues through JCAR017 administration at Day 1 with follow-up through Day 29; - Posttreatment, which includes follow-up assessments for disease status and safety for 5 years.

Type: Interventional

Start Date: Jul 2020

open study

RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).

Type: Interventional

Start Date: Jan 2025

open study

This study is referred to as the "umbrella master protocol" for pembrolizumab (MK-3475) in the treatment of non-small cell lung cancer (NSCLC). This pembrolizumab NSCLC umbrella master protocol uses a platform design and consists of this master screening study and seven substudies. Each substudy will enroll a different population of NSCLC participants. The goal of this umbrella master protocol is to screen potential participants with NSCLC for enrollment into 1 of 7 substudies. Participants must first enroll in this pembrolizumab master protocol study and undergo screening for NSCLC that will be used to assign them to participation in 1 of 7 pembrolizumab substudies.

Type: Observational

Start Date: Dec 2019

open study

The purpose of this study is to test the safety and tolerability of PMD-026 in patients with metastatic breast cancer. PMD-026 is a targeted oral agent designed to kill tumor cells in metastatic breast cancer.

Type: Interventional

Start Date: Nov 2019

open study