Mass General - Division of Clinical Research

This study is open to adults aged 18 and older or above legal age who have systemic sclerosis. People can participate if they have a specific subtype called diffuse cutaneous systemic sclerosis. People with another subtype called limited cutaneous systemic sclerosis can also participate if they are anti Scl-70 antibody positive. Systemic sclerosis is also called scleroderma. The purpose of this study is to find out whether a medicine called Avenciguat (BI 685509) helps people with scleroderma who have symptoms due to lung fibrosis or vascular problems. Participants are put into 2 groups by chance. One group takes Avenciguat (BI 685509) tablets 3 times a day and the other group takes placebo tablets 3 times a day. Placebo tablets look like BI 685509 tablets but do not contain any medicine. Participants take the tablets for at least 11 months. Afterwards, participants can continue to take the tablets until the last participant has completed the 11-months treatment period. This means that the time in the study and duration of treatment is different for each participant, depending on when they start the study. At the beginning of the study, participants visit the study site every 2 weeks. The time between the visits to the study site gets longer over the course of the study. After the 11-months treatment period, participants visit the study site every 3 months. During the study, participants regularly do lung function tests. The results are compared between the 2 groups to see whether the treatment works. The participants also regularly fill in questionnaires about their scleroderma symptoms. The doctors regularly check participants' skin condition and general health and take note of any unwanted effects.

Type: Interventional

Start Date: Nov 2022

open study

B-cell Lymphoma is an aggressive and rare cancer of a type of immune cells (a white blood cell responsible for fighting infections). Classic Follicular Lymphoma is a slow-growing type of non-Hodgkin lymphoma. The purpose of this study is to assess the safety of epcoritamab in adult participants in relapsed or refractory (R/R) diffuse large b-cell lymphoma (DLBCL) who have received at least 1 prior line of systemic antilymphoma therapy including at least 1 anti-CD20 monoclonal antibody-containing therapy or R/R classic follicular lymphoma (cFL). Adverse events will be assessed. Epcoritamab is an investigational drug being developed for the treatment of R/R DLBCL and R/R cFL. Study doctors will assess participants in a monotherapy treatment arm of epcoritamab. Participants will receive escalating doses of epcoritamab, until full dose is achieved. Approximately 184 adult participants with R/R DLBCL and R/R cFL will be enrolled in the study in approximately 80 sites in the United States of America. Participants will receive escalating doses of subcutaneous epcoritamab, until full dose is achieved, in 28-day cycles. 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 an approved institution (hospital or clinic). The effect of the treatment will be frequently checked by medical assessments, blood tests, questionnaires and side effects.

Type: Interventional

Start Date: Aug 2022

open study

The purpose of this study is to find out whether the study drug, LY4052031, is safe, tolerable and effective in participants with advanced, or metastatic solid tumors including urothelial cancer. The study is conducted in two parts - phase Ia (dose-escalation, dose-optimization) and phase Ib (dose-expansion). The study will last up to approximately 4 years.

Type: Interventional

Start Date: Jul 2024

open study

This is a Phase 2, randomized, double-blind, placebo-controlled, multiple dose study to assess the safety, tolerability, and efficacy of JK07 in participants aged 18-85 with heart failure. There will be 2 cohorts in this study: Cohort 1: Heart failure (HF) participants with left ventricular ejection fraction (LVEF) of ≤ 40%. Cohort 2: Heart failure (HF) participants with left ventricular ejection fraction (LVEF) > 40% and ≤ 65%. Participants in both cohorts will be randomized into either low dose JK07, high dose JK07 or placebo. Participants will have a 2:1 chance of receiving JK07 versus placebo.

Type: Interventional

Start Date: Mar 2024

open study

This Phase 2 clinical trial will study RVP-001, a new manganese-based MRI contrast agent, in people who are known to have gadolinium-enhancing central nervous system (CNS) lesions, for example stable brain tumors or multiple sclerosis. The goal of this study is to assess safety, efficacy, and pharmacokinetics of RVP-001 at three dose levels. The study will also compare RVP-001 imaging to gadolinium-based contrast agent (GBCA) imaging. A single dose of RVP-001 will be administered to each subject. Subjects will have known gadolinium-enhancing CNS lesions and will be due to have a routine gadolinium-based contrast agent-enhanced MRI of the brain a few days before receiving RVP-001 with imaging. The ultimate goal of this research program is development of a gadolinium-free alternative to current general purpose MRI contrast agents.

Type: Interventional

Start Date: Mar 2024

open study

The Epilepsy Learning Health System (ELHS) is a quality improvement and research network to improve outcomes for people with epilepsy. The ELHS is designed as a model of value-based chronic care for epilepsy as envisioned by the National Academies of Medicine Committee in their landmark reports "The Learning Health System" and "Epilepsy Across the Spectrum: Promoting Health and Understanding". The ELHS network is a collaboration among clinicians, patients and researchers that promotes the use of data for multiple purposes including one-on-one clinical care, population management, quality improvement and research. The ELHS Registry includes data on children and adults with epilepsy collected during the process of standard epilepsy care. These data are used to create population health reports and to track changes in outcomes over time. ELHS teams use quality improvement methods, such as Plan-Do-Study-Act (PDSA) cycles, to continuously learn how to improve care.

Type: Observational [Patient Registry]

Start Date: Mar 2019

open study

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Systemic Lupus Erythematosus

Type: Interventional

Start Date: Feb 2024

open study

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

Type: Interventional

Start Date: Aug 2023

open study

The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use. Participants will apply either Patidegib Gel 2% or a Vehicle Gel (with no active drug substance) to their face twice a day for a year (12 months). The number of new BCCs on the face will be compared between participants who used Patidegib Gel 2% or Vehicle Gel after 12 months.

Type: Interventional

Start Date: Mar 2024

open study

The primary objective of this study is to evaluate the long-term safety and tolerability of ecopipam tablets in children (greater than or equal to [>=] 6 and less than [<] 12 years of age), adolescents (>=12 and <18 years of age), and adults (>=18 years of age) with Tourette's Syndrome (TS).

Type: Interventional

Start Date: Aug 2023

open study

The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986278 in participants with Idiopathic Pulmonary Fibrosis.

Type: Interventional

Start Date: Sep 2023

open study

Crohn's Disease (CD) is a gastrointestinal disease that can cause chronic diarrhea with or without gross bleeding, abdominal pain, weight loss, and fever. This study will assess the pharmacokinetics, efficacy, and safety of risankizumab in pediatric participants with moderately to severely active CD aged 2 to < 18 years old who have had intolerance or inadequate response to other therapies. Risankizumab is an approved drug for adults with plaque psoriasis, psoriatic arthritis, and CD and is being developed for the treatment of CD in pediatrics. This study is comprised of 3 cohorts that may participate in 3 substudies (SS). Cohort 1 will enroll participants with ages from 6 to less than 18 years. Cohort 2 will enroll participants with ages from 2 to less than 6 years. Cohort 3 will enroll participants with ages from 2 to less than 18 years. SS1 is an open-label induction period where participants will receive a weight-based induction regimen of risankizumab. SS2 is a double-blind maintenance period where participants will be randomized to receive 1 of 2 doses of weight-based induction regimen of risankizumab. SS3 is an open-label extension period where participants will receive risankizumab based off of their response in SS2. Around 110 pediatric participants with CD will be enrolled at around 100 sites worldwide. Participants in SS1 will receive risankizumab intravenously during the 12-week induction period. Participants in SS2 will receive risankizumab subcutaneously during the 52-week randomized maintenance period. Participants in SS3 will receive risankizumab subcutaneously during the 208-week open label period. Participants will be followed-up for approximately 140 days. 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: Dec 2023

open study

The investigators will conduct a 12,500-patient randomized multi-center trial to determine (i) which general anesthesia technique yields superior patient recovery experiences in any of three surgical categories ((a) major inpatient surgery, (b) minor inpatient surgery, (c) outpatient surgery) and (ii) whether TIVA confers no more than a small (0.2 %) increased risk of intraoperative awareness than INVA in patients undergoing both outpatient and inpatient surgeries

Type: Interventional

Start Date: Sep 2023

open study

This study will evaluate the efficacy of TNX-103 (levosimendan) compared with placebo in subjects with PH-HFpEF as measured by the change in 6-Minute Walk Distance (6 MWD; Day 1 to Week 12).

Type: Interventional

Start Date: Jan 2024

open study

The purpose of the study is to test the safety and dosing of [177Lu]Lu-FF58, a radioligand therapy for patients with advanced or metastatic tumors that express proteins known as integrins: alpha-v beta-3 integrin (αvβ3) and alpha-v beta-5 integrin (αvβ5). The study will also further characterize the radioligand imaging agent [68Ga]Ga-FF58 including its ability to identify tumor lesions and its safety profile.

Type: Interventional

Start Date: Oct 2023

open study

This study evaluates the efficacy and safety of a single injection of iltamiocel (300 x 10^6 cells) compared to a placebo in the reduction of fecal incontinence episode frequency in adult female participants with chronic fecal incontinence and a history of obstetric anal sphincter injury. Half of the participants will receive iltamiocel (injections with cells) and the other half will receive placebo.

Type: Interventional

Start Date: Mar 2024

open study

The goals of this study are to compare the amount of study drug, bulevirtide (BLV), that gets into the bloodstream and how long it takes for the body to eliminate it, measure the effect of BLV on bile acids, and evaluate the safety and tolerability of multiple doses of BLV in participants with normal or impaired renal (kidney) function.

Type: Interventional

Start Date: Mar 2023

open study

CKJX839D12302 is a pivotal Phase III study designed to test the hypothesis that treatment with inclisiran sodium 300 milligram (mg) subcutaneous (s.c.) administered on Day 1, Day 90, and every 6 months thereafter in patients at high cardiovascular (CV) risk without a prior major atherosclerotic cardiovascular disease (ASCVD) event will significantly reduce the risk of 4-Point-Major Adverse Cardiovascular Events (4P-MACE) defined as a composite of CV death, non-fatal myocardial infarction (MI), non-fatal ischemic stroke, and urgent coronary revascularization, compared to placebo.

Type: Interventional

Start Date: Mar 2023

open study

This is a randomized, double-blind, placebo-controlled, parallel-group, international, Phase 3 study in patients with newly diagnosed H3 K27M-mutant diffuse glioma to assess whether treatment with ONC201 following frontline radiotherapy will extend overall survival and progression-free survival in this population. Eligible participants will have histologically diagnosed H3 K27M-mutant diffuse glioma and have completed standard frontline radiotherapy.

Type: Interventional

Start Date: Jan 2023

open study

Vorasidenib in combination with pembrolizumab in participants with recurrent or progressive enhancing isocitrate dehydrogenase-1 (IDH-1) mutant Glioma.

Type: Interventional

Start Date: Jan 2023

open study

The purpose of this study is to assess the safety and tolerability of zilovertamab vedotin as monotherapy and in combination in participants with select B-cell lymphomas including mantle cell lymphoma (MCL), Richter's transformation lymphoma (RTL), follicular lymphoma (FL), and chronic lymphocytic leukemia (CLL). This study will also evaluate zilovertamab vedotin as monotherapy and in combination with respect to objective response rate. - Cohort A: Participants with relapsed or refractory MCL relapsed or refractory disease after at least 2 prior systemic therapies including a Bruton's tyrosine kinase inhibition/inhibitor (BTKi), and post therapy chimeric antigen receptor T (CAR-T) cell therapy or ineligible for CAR-T cell therapy - Cohort B: Participants with relapsed or refractory RT disease after at least 1 prior systemic therapy - Cohort C: Participants with relapsed or refractory MCL relapsed or refractory disease after at least 1 prior systemic therapy and no prior exposure to a non-covalent BTKi - Cohort D: Participants with relapsed or refractory FL and CLL relapsed or refractory disease after at least 2 prior systemic therapies and have no other available therapy - Cohort E: Participants with relapsed or refractory FL after at least 2 prior systemic therapies and have no other available therapy - Cohort F: Participants with relapsed or refractory CLL after at least 2 prior systemic therapies and have no other available therapy The primary study hypothesis is that zilovertamab vedotin monotherapy has an increased Objective Response Rate (ORR) per Lugano Response Criteria as assessed by blinded independent central review (BICR).

Type: Interventional

Start Date: Jul 2022

open study

This study aims to understand how safe and well-tolerated a drug called BGB-24714 is when used alone, or in combination with chemotherapy or radiation therapy, for people with advanced or spreading solid tumors. The main objective is to identify the highest tolerable dose or the highest administered dose of BGB-24714. Additionally, the study aims to identify the most suitable doses for further investigation in larger groups of participants.

Type: Interventional

Start Date: Jul 2022

open study

This is a first in human study in patients with advanced or metastatic solid tumors known to have an MTAP deletion. The first part of the study is an open-label, dose escalation and the second part is an open label dose expansion in specific MTAP-deleted tumor types. The study drug, TNG908, is a selective PRMT5 inhibitor administered orally. The study is planned to treat up to 192 participants.

Type: Interventional

Start Date: Mar 2022

open study

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.

Type: Interventional

Start Date: Jun 2022

open study

The specific aims of this study are to: 1. Determine if a painless and quick measurement of muscle activity using surface electrodes can help with the diagnosis of ALS. Specifically, we ask if a measure of intermuscular coherence (IMC-βγ), when added to current diagnostic criteria (Awaji criteria), can differentiate ALS from mimic diseases more accurately and earlier than currently possible. 2. Characterize IMC-βγ in neurotypical subjects by age, sex, race, and ethnicity. 3. Follow a cohort of ALS patients longitudinally to determine if IMC-βγ changes with ALS disease progression and whether such changes correlate with functional and clinical scores, or survival.

Type: Observational

Start Date: Mar 2021

open study