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

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

Type: Interventional

Start Date: Oct 2024

open study

The investigator aims to conduct an open pilot study (N=12; 10 completers) to test the feasibility, acceptability, and credibility of an asynchronous web-based mind-body intervention (Toolkit for Resilient Life beyond Pain and Substance Use; Web-TIRELESS) for adult patients with a comorbidity of non-traumatic painful upper-extremity condition(s) (NPUC) and risky substance use. Deliverables: 1) Adapt and refine open pilot protocol, patient recruitment, and other study materials. 2) Assess the feasibility, acceptability, and credibility of Web- TIRELESS in preparation for a future feasibility RCT. Participants will complete 4 on-demand video sessions at their own pace (approximate pace of 1 session per week) and complete baseline and post-test assessments. participants may also partake in an exit interview to provide feedback on Web-TIRELESS to further refine the program and study protocol for future iterations.

Type: Interventional

Start Date: Oct 2024

open study

This is a two-part (Phase 2/Phase 3) study of V940, an individualized neoantigen therapy (INT), plus pembrolizumab in participants with locally resectable advanced cutaneous squamous cell carcinoma (LA cSCC). Phase 2 has three arms V940 plus pembrolizumab given as neoadjuvant and adjuvant treatment with standard of care (SOC), standard of care (surgical resection with/without adjuvant radiation therapy (RT) only at investigator's discretion) and pembrolizumab monotherapy given as neoadjuvant and adjuvant treatment with SOC. This phase will assess the safety and efficacy of V940 in combination with pembrolizumab as neoadjuvant and adjuvant therapy in participants with resectable LA cSCC as compared to standard of care SOC only. The primary hypothesis is that V940 plus pembrolizumab with SOC is superior to SOC only with respect to event free survival (EFS) as assessed by the investigator. Phase 3 expansion will be determined by prespecified Go-No-Go decision in which 412 additional participants will be randomized to V940 plus pembrolizumab with SOC and SOC only, without changing the inclusion/exclusion criteria for the additional enrollment or study endpoints.

Type: Interventional

Start Date: Apr 2024

open study

This seamless phase 2/3 randomized controlled study will evaluate the efficacy and safety of the hexavalent OX40 agonist antibody INBRX-106 combined with the anti-PD-1 antibody pembrolizumab versus pembrolizumab (+ placebo in phase 3) as first-line treatment for patients with locally advanced recurrent or metastatic head and neck squamous cell carcinoma (R/M HSNSCC) incurable by local therapies, expressing PD-L1 with a combined proportion score (CPS) ≥20.

Type: Interventional

Start Date: May 2024

open study

This research study aims assess whether the Difference Frequency Generation (DFG) laser could be a better alternative to the CO2 laser in terms of reduced side effects and patient downtime.

Type: Interventional

Start Date: Oct 2024

open study

The primary purpose of this substudy is to determine if collagen-targeted PET using the type 1 collagen-targeted PET probe, Gallium-68 (68Ga)-labeled collagen binding probe 8 (CBP8) can inform as to drug effect of EGCG and assist in dose selection.

Type: Interventional

Start Date: Feb 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) or Progressive Forms of Multiple Sclerosis (PMS).

Type: Interventional

Start Date: Mar 2024

open study

This is a global, open-label, multicenter Phase 1/2 study to evaluate the safety, tolerability, PK, and antitumor activity of PRO1107 in patients with advanced solid tumors. This study consists of 2 parts, Part A: dose escalation and dose level expansion, and Part B: tumor specific expansion.

Type: Interventional

Start Date: Jan 2024

open study

This is a parallel group, Phase 3, multinational, multicenter, randomized, double blind, placebo-controlled, 3-arm monotherapy study for treatment of participants diagnosed with moderate to severe atopic dermatitis (AD), whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. The purpose of this study is to measure the efficacy and safety of treatment with amlitelimab solution for SC injection compared with placebo in participants with moderate to severe AD aged 12 years and older. Study details include: At the end of the treatment period, participants will have an option to enter a separate study: the blinded extension study EFC17600 (ESTUARY). For participants not entering the blinded extension Study EFC17600 (ESTUARY), the study duration will be up to 44 weeks including a 2 to 4-week screening, a 24-week randomized double-blind period, and a 16-week safety follow-up. For participants entering the blinded extension Study EFC17600 (ESTUARY), the study duration will be up to 28 weeks including a 2 to 4-week screening and a 24-week randomized double-blind period. The total treatment duration will be up to 24 weeks. The total number of visits will be up to 10 visits (or 9 visits for those entering the blinded extension study EFC17600] (ESTUARY).

Type: Interventional

Start Date: Nov 2023

open study

The primary goal of this study is to test the effectiveness of the iDECIDE (Drug Education Curriculum: Intervention, Diversion, and Empowerment) curriculum, a novel drug education and diversion program, in approximately 300 middle and high school students, who have violated their school substance use policies in the past month, as an alternative to punitive school responses for school-based substance use infractions. This randomized controlled trial will test the hypothesis that adolescents randomized to the iDECIDE curriculum will have improved substance use outcomes (i.e., knowledge, attitudes, and behavior) compared to adolescents in a waitlist control group. The outcomes of this study will measure knowledge of drug effects and brain development, perceptions of harm from substance use, willingness to quit or reduce use, and substance use behavior.

Type: Interventional

Start Date: Nov 2023

open study

This is a prospective multicenter biomarker study evaluating the prognostic impact of ctDNA detection at diagnosis in patients with Ewing sarcoma or osteosarcoma.

Type: Interventional

Start Date: May 2018

open study

The goal of this prospective cohort study is to assess potential differences in sleep biomarkers in older adult patients undergoing major orthopedic surgery. The main questions it aims to answer are: 1. To define sleep/circadian biomarkers of delirium (sleep duration, regularity, stability and timing of rhythm) in a prospective observational study. 2. To determine if plasma Alzheimer's disease (AD) pathology/inflammatory burden interacts with or moderates the relationship between a sleep/circadian biomarker and post-operative delirium (POD) risk. 3. To determine whether sleep/circadian regulation interacts with the genetic risk of AD to influence POD/cognitive decline. Participants will be asked to: 1. Donate several blood samples both intraoperatively and postoperatively 2. Complete baseline and postoperative neurocognitive assessments 3. Wear an actigraphy data collection watch for the two weeks prior to their surgery

Type: Observational

Start Date: Sep 2023

open study

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

Type: Interventional

Start Date: Oct 2023

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: Oct 2024

open study

The goal of this clinical trial is to assess the effect of PRP/PPP/BMAC application on functional outcomes after hip arthroscopy to address acetabular labral pathologies. The main questions it aims to answer are: 1. Does PRP/PPP/BMAC application improve functional outcomes after hip arthroscopy to address acetabular labral pathologies? 2. Does PRP/PPP/BMAC application reduce the arthritic burden as measured by functional outcomes following hip arthroscopy to address labral pathologies with concomitant PRP/PPP/BMAC application?

Type: Interventional

Start Date: Aug 2023

open study

This goal of this study is to test an information and support intervention for patients with malignant (or "high-grade") brain tumors. This study was developed to help patients cope after a brain tumor diagnosis. The main question this study aims to answer is whether this intervention (which includes access to an information guide and one-on-one coaching sessions) is feasible (i.e., possible to carry out) and acceptable (i.e., considered helpful) to patients. Participants will be asked to take part in the coaching sessions, use the guide as desired, and complete a small group of short surveys at three different points in time; some participants will be asked to share feedback via exit interviews.

Type: Interventional

Start Date: Oct 2023

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

Subjects include: aged 18 to 75 years, inclusive, have biopsy-confirmed disease that is clinically inactive as determined by negative celiac disease (CeD) serology and histology (determined via endoscopy at time of screening), have followed a gluten-free diet (GFD) for ≥6 months as reported by the subject, and be human leukocyte antigen (HLA)-DQ2.5 and/or HLA-DQ8 positive. Study involves the following randomized intervention; 10g gluten + 200mg of Ritlecitinib or placebo

Type: Interventional

Start Date: Mar 2023

open study

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary anti-tumor activity of RO7616789. The study will have 3 parts: Dose Escalation (Parts 1 and 2) and Dose Expansion (Part 3). Participants with advanced stage small cell lung cancer (SCLC) and neuroendocrine carcinoma (NEC) will be enrolled in the study.

Type: Interventional

Start Date: Jan 2023

open study

This study will test the safety, including side effects, and determine the characteristics of a drug called PRO1184 in participants with solid tumors. Participants will have solid tumor cancer that has spread through the body (metastatic) or cannot be removed with surgery (unresectable).

Type: Interventional

Start Date: Dec 2022

open study

This is a multicenter, Phase 1b/2 trial. The phase 1b part of the trial aims to determine the RP2D of elacestrant when administered in combination with alpelisib, everolimus, palbociclib, abemaciclib, and ribociclib. The Phase 2 part of the trial will evaluate the efficacy and safety of the various combinations in patients with ER+/HER2- advanced/metastatic breast cancer.

Type: Interventional

Start Date: Jan 2023

open study

The purpose of this study is to characterize the safety and tolerability of KFA115 and KFA115 in combination with pembrolizumab in patients with select advanced cancers, and to identify the maximum tolerated dose and/or recommended dose.

Type: Interventional

Start Date: Oct 2022

open study

A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of tecovirimat for the treatment of people with laboratory-confirmed or presumptive HMPXV disease.

Type: Interventional

Start Date: Sep 2022

open study

The primary goal of this study is to assess the safety and tolerability of mRNA-4359 administered alone and in combination with pembrolizumab.

Type: Interventional

Start Date: Aug 2022

open study