Mass General - Division of Clinical Research

The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.

Type: Interventional

Start Date: Apr 2026

open study

The goal of this observational study is to learn about the natural progression of Angelman syndrome (AS) in children and adults with a confirmed genetic diagnosis of AS. The main questions it aims to answer are: - How do developmental skills, such as communication, motor abilities, and adaptive behaviors, change over a 1-year period in people with AS? - Are there specific patterns in brain activity or sleep that are associated with changes in AS symptoms over time? Participants will: - Visit the study site 5 times over 1 year (approximately every 3 months) for assessments. - Complete tests and questionnaires about development, behaviors, and sleep with the help of their caregivers. - Undergo electroencephalograms (EEGs) to measure brain activity and wear a sleep-monitoring device at home (to collect actigraphy data).

Type: Observational

Start Date: Jun 2026

open study

PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Two escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.

Type: Interventional

Start Date: Dec 2025

open study

The purpose of this study is to evaluate how well JNJ-79635322 works (efficacy) in participants with Relapsed or Refractory Multiple Myeloma (RRMM; a cancer that forms in a type of white blood cells called a plasma cell. Cancer is called relapsed if it comes back after treatment and is called 'refractory' if does not respond to treatment) who have received at least 3 prior lines of therapy.

Type: Interventional

Start Date: Feb 2026

open study

This is a study of LY4337713 in participants with certain types of cancer that is advanced or has spread. Participants must have cancer with high levels of a protein called fibroblast activation protein (FAP). The purpose of this study is to evaluate safety, side effects, and efficacy of LY4337713. In addition, this study will evaluate how much LY4337713 gets into the bloodstream, how it is broken down, and how long it takes the body to get rid of it. For each participant, the study will last about 5 years.

Type: Interventional

Start Date: Oct 2025

open study

This study is researching an experimental drug called dupilumab (called "study drug"). The study is focused on children with active eosinophilic esophagitis (EoE; an inflammatory disease of the esophagus) which impacts feeding and nourishment. The aim of the study is to see how safe, tolerable, and effective the study drug is when given for 24 weeks to children with active EoE. The study is looking at several other research questions, including: - 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)

Type: Interventional

Start Date: Nov 2025

open study

This is a multi-center, randomized, open label study that will assess the efficacy and safety of ACTEMRA(R) or one of its FDA-approved biosimilars Tocilizumab (TCZ) maintenance versus withdrawal in Giant cell arteritis (GCA) patients who are in remission after at least 12 months of high dose TCZ treatment. Eligible participants will also have discontinued glucocorticoids (e.g., prednisone (or equivalent)) entirely at least three months before randomization. High dose TCZ treatment includes 6-8 mg/kg intravenously (IV) monthly or 162 mg subcutaneously (SC) weekly, which are two forms of administration that are commonly used in clinical practice and are equally efficacious in controlling GCA This research study has three parts: 1. The screening phase (up to 42 days) consists of collecting information about your health and your GCA, a physical exam, and blood tests to see If you qualify to enroll in the study 2. The study treatment phase (withdrawal/step down dosing phase study months 0 - 18) consists of you either completely stopping or decreasing your current dose of tocilizumab while collecting information about your health and your GCA as well as blood samples every two months at clinic visits 3. The safety follow-up phase (months 19-30) consists of collecting information about your health and your GCA as well as blood samples every three months The primary objective is to determine the rate of disease relapse at 18 months in participants with GCA who receive low-dose TCZ compared to those who discontinue TCZ

Type: Interventional

Start Date: Dec 2025

open study

This study will evaluate the safety, efficacy, optimal dose, and pharmacokinetics (PK) of BNT326 as monotherapy (Part 1) and as combination treatment with immunotherapeutic agents (Part 2) in participants with histologically or cytologically confirmed solid tumors that are advanced (i.e., either metastatic or recurrent tumors with no further definitive treatment possible) and/or have relapsed/progressed after prior therapy.

Type: Interventional

Start Date: Aug 2025

open study

This study will evaluate the safety and efficacy of INCB123667 in Participants With Platinum-Resistant Ovarian Cancer (PROC) With Cyclin E1 Overexpression.

Type: Interventional

Start Date: Nov 2025

open study

The overall aim of this study is to develop an intervention that can help recovery in patients surviving severe brain injury but failing to fully recover. In particular, this multicenter project aims to (1) establish short-term efficacy of tFUS as a therapeutic to promote recovery in patients with prolonged DoC as compared to sham treatment, (2) establish dose-related safety and efficacy of tFUS as a therapeutic intervention in prolonged DoC patients and (3) explore preliminary predictors and biomarkers of susceptibility and response to thalamic sonication.

Type: Interventional

Start Date: Apr 2025

open study

This is a randomized controlled trial testing the efficacy of Resilience Training in college students with elevated transdiagnostic risk for developing a serious mental illness.

Type: Interventional

Start Date: Feb 2025

open study

The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)

Type: Interventional

Start Date: Mar 2025

open study

Women living with HIV have been shown to experience more frequent and severe hot flashes and night sweats (collectively known as vasomotor symptoms) as compared to women living without HIV. This correlates with disturbed sleep, increased depressive symptoms, increased anxiety, worse mental function, interference with activities of daily living including work, and worse overall quality of life. Hormone therapy is considered to be the most effective therapy for hot flashes and night sweats and the most appropriate choice to prevent bone loss at the time of menopause for women without HIV. However, the usefulness of hormone therapy has not been specifically studied in women living with HIV. This trial is being done to see if: - There is evidence to support the use of hormone therapy (estradiol with or without progesterone) for the treatment of hot flashes and night sweats in women living with HIV - Hormone therapy improves mental function, mood, sleep, quality of life, bone health, heart health, and inflammation in women living with HIV - Hormone therapy is safe and tolerable for women living with HIV

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this study is to determine the proportion of participants who achieve undetectable measurable residual disease (uMRD) in previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

Type: Interventional

Start Date: May 2025

open study

This is a first-in-human study of MEN2312, a lysine acetyltransferase 6 (KAT6) inhibitor, in adult participants with advanced breast cancer.

Type: Interventional

Start Date: Oct 2024

open study

The goal of this study is to learn if people who receive intismeran autogene and pembrolizumab after surgery are cancer-free longer than people who receive placebo and pembrolizumab. Researchers want to know if giving intismeran autogene and pembrolizumab after surgery can help prevent the cancer from coming back in people with non-small cell lung cancer (NSCLC) whose tumors did not respond completely to treatment before surgery (neoadjuvant treatment).

Type: Interventional

Start Date: Oct 2024

open study

This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).

Type: Interventional

Start Date: Jan 2025

open study

This is a randomized, open-label study in adult patients who have completed standard first line therapy for large B-cell lymphoma (LBCL) and achieved a complete response or partial response suitable for observation, but who have minimal residual disease (MRD) as detected by the Foresight CLARITY™ Investigational Use Only (IUO) MRD test, powered by PhasED-Seq™. The purpose of the trial is to assess the efficacy and safety of consolidation with cemacabtagene ansegedleucel (cema-cel), an allogeneic CD19 CAR T product, as compared to standard of care observation. In this study, participants with MRD are randomized 1:1 to treatment with cema-cel or an observation arm. Treatment includes cema-cel following a lymphodepletion regimen of fludarabine and cyclophosphamide. Prior to August 2025, participants may also have received an anti-CD52 monoclonal antibody, ALLO-647, as part of their lymphodepletion regimen.

Type: Interventional

Start Date: Jun 2024

open study

This study is designed to characterize the safety, tolerability, and anti-tumor activity of MDX2001 in patients with advanced solid tumors.

Type: Interventional

Start Date: Jun 2024

open study

This clinical trial is studying solid tumor cancers. A solid tumor is one that starts in part of your body like your lungs or liver instead of your blood. Once they've grown bigger in one spot or spread to other parts of the body, they're harder to treat. This is called advanced or metastatic cancer. Participants in this study must have breast cancer or gastric cancer. Participants must have tumors that have HER2 on them. This allows the cancer to grow more quickly or spread faster. There are few treatment options for patients with advanced or metastatic solid tumors that express HER2. This clinical trial uses an experimental drug called disitamab vedotin (DV). Disitamab vedotin is a type of antibody drug conjugate or ADC. ADCs are designed to stick to cancer cells and kill them. This clinical trial uses a drug called tucatinib, which has been approved to treat cancer in the United States and some other countries. This drug is sold under the brand name TUKYSA®. This study will test how safe and how well DV with tucatinib works for participants with solid tumors. This study will also test what side effects happen when participants take these drugs. A side effect is anything a drug does to the body besides treating the disease.

Type: Interventional

Start Date: May 2024

open study

TRANSFORM is a prospective, randomized, open blinded endpoint (PROBE), event-driven, pragmatic trial in patients who are at increased risk for atherosclerotic cardiovascular (CV) disease but with no known symptomatic CV disease. The trial tests the hypothesis that a Cleerly Coronary Artery Disease (CAD) Staging System-based care strategy reduces CV events compared with risk factor-based care.

Type: Interventional

Start Date: Mar 2024

open study

The purpose of this study is to evaluate the efficacy and safety of adjuvant autogene cevumeran plus atezolizumab and modified leucovorin, 5-fluorouracil (5-FU), irinotecan, and oxaliplatin (mFOLFIRINOX) versus mFOLFIRINOX alone in participants with resected pancreatic ductal adenocarcinoma (PDAC) who have not received prior systemic anti-cancer treatment for PDAC and have no evidence of disease after surgery.

Type: Interventional

Start Date: Oct 2023

open study

Study RIN-PF-305 is designed to evaluate the safety and efficacy of inhaled treprostinil in subjects with progressive pulmonary fibrosis (PPF) over a 52-week period.

Type: Interventional

Start Date: Oct 2023

open study

This study is to prospectively compare clinical effectiveness between clinically- matched incremental hemodialysis and conventional hemodialysis in patients with incident kidney dysfunction requiring dialysis and residual kidney function. The study will enroll 350 patients on chronic hemodialysis and 140 caregivers of enrolled patients. Patients will be randomized in 1:1 ratio to either incremental start hemodialysis or conventional hemodialysis. Caregivers will be followed along with patients for an average period of 2 years post randomization.

Type: Interventional

Start Date: Feb 2024

open study

The goal of this observational study is to evaluate the role of blood pressure (BPV) variability in patients suffering from acute ischemic stroke. The main questions it aims to answer are: 1. To determine the association of BPV with functional/cognitive outcome after ischemic stroke. 2. To determine a pathophysiologic mechanism of BPV's deleterious effect on functional outcome. 3. To evaluate potential treatment targets to pharmacologically reduce BPV after ischemic stroke.

Type: Observational

Start Date: Jul 2024

open study