Clinical trials

The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is being funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases. ASSESS protocol is specific for symptomatic ALS and control participants. This protocol includes both on-site and off-site(remote) participants. The participants will be followed for 24 months (2 years), and will include collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once a month. Participants who are coming into clinic may also provide optional Cerebrospinal Fluid (CSF) samples.

The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is begin funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases. PREVENT protocol is specific for asymptomatic participants who are genetically at risk for ALS. The participants will be followed for up to 36 months (3 years), and will include 4 in-person on-site visits once a year and 6 off-site(remote) visits once in 4 months. The study includes collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once in 4 months. Participants may also provide optional Cerebrospinal Fluid (CSF) samples.The participants may also opt into a sub-study if they are interested in genetic testing for ALS causative genes. The sub-study will involve a minimum of 3 visits over a course of 2-3 months. This will include a screening/pre-test genetic counseling visit, a return of genetic results and a post-test counseling visit.

Collaboration with multiple sclerosis (MS) specialty colleagues led us to formulate the central hypothesis that Siponimod could lower the rate of brain atrophy in Alzheimer's disease (AD) subjects. To test our central hypothesis, we will carry out an 18-month Phase II, double-blind, randomized, twoarmed, placebo controlled, proof-of-concept clinical study in early AD subjects (i.e. mild AD) who will be receiving an escalating dose of Siponimod or placebo in the ratio 2:1 for 12 months, followed by a 6-month washout period. The primary outcome measures are safety and tolerability of Siponimod in mild AD subjects. The secondary outcome measures are the rates of brain atrophy derived from volumetric MRI (vMRI) as a proxy for neurodegeneration conducted at baseline, 6, 12, and 18 months. The tertiary outcome measures are the changes in cognition and the levels of AD-associated (e.g., Aβ and tau) and inflammatory biomarkers in CSF after Siponimod exposure. In an exploratory effort, we will also measure plasma inflammatory markers during the entire duration of the study to investigate whether one or more of these markers can be used as dynamic surrogate markers of treatment response. Using our unique experience with the repurposing of immunomodulatory drugs for AD (and NCT #04032626), in the present project we are using elements of clinical trial design that we believe were successful and made some adjustments to fit the pharmacologic and toxic properties of Siponimod.

This study will administer the investigational drug, BDTX-1535 to eligible patients with recurrent high-grade glioma (HGG) and newly-diagnosed glioblastoma (nGBM). BDTX-1535 was designed to block a growth signal important to some cancers. BDTX-1535 is being tested in this study to see if it can be given safely to people who have tumors that can be dependent on that growth signal because of changes in a protein called EGFR. These gene changes are called amplifications, mutations, fusions or alterations and are found only in the tumors. The study design includes a Phase 0 component with PK/PD-trigger for participant enrollment into an Expansion Phase 1 component. The primary objective of the Phase 0 component is to evaluate the PK endpoints of BDTX-1535. The primary objective of the Phase 1 component is to establish the safe dose of BDTX-1535 to be used in participants with a specified treatment regimen, three of which include standard of care radiotherapy for nGBM participants.

Urinary tract infections in catheterized patients is an unacceptable complication and has been termed a 'never event' by the Center for Medicare \& Medicaid Services (CMS); however there is not consensus among clinicians on how to best prevent CAUTIs. We propose a prospective randomized controlled trial to test the efficacy of prophylactic gentamicin bladder irrigation in elimination of CAUTIs.

This pilot and feasibility study will enable the research team to determine the feasibility of implementing a time-restricted eating regimen among adults with mild cognitive impairment (MCI) and the impact of time-restricted eating on cognitive performance and biomarkers of metabolic health in this population. Study staff will execute the specific aims using a pre-post, non-randomized study design in which all participants receive the intervention. The intervention is a 16/8 time-restricted eating regimen characterized by fasting for 16 hours and eating within an 8-hour window on 5 days per week for 3 months. Assessments will be performed at baseline and after the 3-month time-restricted eating intervention with the following outcome measures. Outcome measures for feasibility include participant recruitment, retention and metrics of acceptability, safety, and adherence to the intervention. Outcome measures for cognitive performance and metabolic health include neuropsychological tests, blood biomarkers, and surveys of psychological well-being.

This is a randomized, double-blind, placebo-controlled, parallel group study. The use of placebo is appropriate to minimize bias related to treatment expectations of the subject, study partner, and site investigator, as well as to changes in the relationship between the subject and study partner that might occur with the initiation of treatment and expectation of improvement in motor symptoms or cognition. Changes in subject/study partner interactions can impact subject mood and might introduce biases that cannot be quantified. The double-blind use of placebo will also prevent bias in the clinical and scientific assessments.

The purpose of this study is to test whether a special memory training program, called CogMed, can help people with early memory problems. The Investigators want to see if this training improves memory and also helps reduce stress. The Investigators also want to see if CogMed results in changes to a blood biomarker called p-Tau 217, which possibly indicate Alzheimer's disease (AD).

To find out whether oral antibiotics given after transsphenoidal endoscopic surgery for pituitary adenoma improves sinus and nasal symptoms, reduces the incidence of infection (sinusitis), and helps mucosal healing in the nasal passages.