Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
Recruiting
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated biomarkers and potential targets with immune and multi-omics profiling. The disease sample collection and analysis will be the foundation for an extensive network of biospecimen access and linked datasets for future translational research.
Gender:
All
Ages:
Between 18 years and 85 years
Locations: Sanguine Biosciences, Woburn, Massachusetts
Conditions: Fanconi Anemia
Study to Assess Adverse Events, Change in Disease Activity and How Oral ABBV-552 Capsules Moves Through the Body of Participants Aged 50 to 90 Years With Mild Alzheimer's Disease
Recruiting
Alzheimer's disease (AD) is a progressive, irreversible neurological disorder and is the most common cause of dementia in the elderly population. Clinical symptoms of the disease may begin with occasional forgetfulness such as misplacement of items, forgetting important dates or events, and may progress to noticeable memory loss, increased confusion and agitation, and eventually, loss of independence and non-responsiveness. This study will assess how safe and effective ABBV-552 is in treating sy... Read More
Gender:
All
Ages:
Between 50 years and 90 years
Locations: Adams Clinical /ID# 248358, Watertown, Massachusetts +11 locations
Conditions: Alzheimer's Disease (AD)
CAR- PRISM (PRecision Intervention Smoldering Myeloma)
Recruiting
The goal of this research study is to test if ciltacabtagene autoleucel (cilta-cel) is safe and effective in treating participants with high-risk, smoldering myeloma. The names of the treatment interventions used in this study are: Cilta-cel (or chimeric antigen receptor T cells) Cyclophosphamide (a lymphodepleting chemotherapy) Fludarabine (a lymphodepleting chemotherapy)
Gender:
All
Ages:
18 years and above
Locations: Dana Farber Cancer Institute, Boston, Massachusetts
Conditions: Multiple Myeloma, Smoldering Multiple Myeloma
A Study to Investigate the Safety, Tolerability, and Processing by the Body of Intravenous RO7121932 in Participants With Multiple Sclerosis.
Recruiting
The primary purpose of the study is to evaluate the safety and tolerability of single ascending intravenous (IV) doses of RO7121932 in participants with multiple sclerosis (MS)
Gender:
All
Ages:
Between 18 years and 65 years
Locations: University of Massachusetts Medical School, Worcester, Massachusetts +22 locations
Conditions: Multiple Sclerosis
Studying the Safety and Determining the Optimal Dose of Novobiocin in Patients With Tumors That Have Alterations in DNA Repair Genes
Recruiting
This phase I trial tests the safety, side effects, and best dose of novobiocin in treating cancer patients with alterations in deoxyribonucleic acid (DNA) repair genes. Novobiocin is an antibiotic that blocks the activity of a protein called DNA polymerase theta, which helps repair DNA that has become damaged as cells grow and divide. Cancer cells that cannot repair their damaged DNA die. This medication may help shrink or stabilize cancer with a mutation in DNA repair genes.
Gender:
All
Ages:
18 years and above
Locations: Dana-Farber - Harvard Cancer Center LAO, Boston, Massachusetts +2 locations
Conditions: Metastatic Malignant Solid Neoplasm, Unresectable Malignant Solid Neoplasm
NHLBI Transmural Electrosurgery LeafLet Traversal And Laceration Evaluation (TELLTALE) BASILICA-TAVR Trial
Recruiting
Background: Replacing a valve in the heart can save many people s lives. The procedure is called transcatheter aortic valve replacement (TAVR). In rare cases, a flap of tissue called a leaflet can shift during TAVR; the leaflet may block blood flow to the coronary arteries, which supply blood to the heart muscle. About 50% of people who experience this problem will die. To reduce this risk, doctors will cut this leaflet during TAVR. But the tools used to cut the leaflet were originally designed... Read More
Gender:
All
Ages:
Between 21 years and 109 years
Locations: Beth Israel Deaconess Medical Center, Boston, Massachusetts +9 locations
Conditions: Valvular Heart Disease, Aortic Valve Failure
Siplizumab in T1DM
Recruiting
This is a multicenter, Phase Ib, open-label, siplizumab dose-finding study in individuals aged 8-45 years with a Type 1 diabetes mellitus (T1DM) diagnosis. within 18 months of V0. Participants will be randomized 1:1:1:1 to one of four possible siplizumab dosing arms. All dosing arms will receive weekly siplizumab doses for a total of 12 weeks. After the completion of treatment, participants will undergo follow-up visits at weeks 12, 24, 36 and 52 which include longitudinal MMTTs. Blood samples f... Read More
Gender:
All
Ages:
Between 8 years and 45 years
Locations: Joslin Diabetes Center: Joslin Clinic, Boston, Massachusetts +19 locations
Conditions: Type 1 Diabetes Mellitus
A Study to Investigate the Safety, Tolerability, PK, PD, and Efficacy of ONO-7018 in Patients With R/R NHL or CLL
Recruiting
This is a Phase 1, open-label, multicenter study. This will be the first-in-human clinical study for ONO-7018 and will be conducted in two phases: a Dose Escalation Phase (Part 1) and a Dose Expansion Phase (Part 2).
Gender:
All
Ages:
18 years and above
Locations: Dana-Farber Cancer Institute, Boston, Massachusetts +9 locations
Conditions: Lymphoma, Non-Hodgkin, Leukemia, Lymphocytic, Chronic, B-Cell
Longitudinal Endotyping Of Atopic Dermatitis Through Transcriptomic Skin Analysis
Recruiting
This is a multi-center, longitudinal study which will characterize the gene expression profiles and transcriptomic endotypes that underlie mild and moderate-severe Atopic dermatitis (AD) and will determine changes in these expression patterns and endotypes in response to standard-of-care treatment. Participants will complete up to ten study visits with assessment of topical steroid response and dupilumab response (if uncontrolled with topical steroids). Skin samples will be collected at all stud... Read More
Gender:
All
Ages:
6 years and above
Locations: Boston Children's Hospital: Department of Immunology, Boston, Massachusetts +9 locations
Conditions: Atopic Dermatitis
The PREDICTOR Study: Assessing Diagnostic Predictors of Airway Collapse in Patients With Obstructive Sleep Apnea
Recruiting
This study is an exploratory, multicenter study of up to 300 subjects diagnosed with obstructive sleep apnea who are being evaluated for airway surgery. Subjects will undergo standard evaluation for airway surgery as part of standard of care. In addition to the standard airway assessment, a simple, non-invasive measurement of the width of the inside of each subjects mouth will be performed. This measurement takes 2-3 minutes to perform.
Gender:
All
Ages:
18 years and above
Locations: Massachusetts Eye and Ear Infirmary, Boston, Massachusetts +9 locations
Conditions: Sleep Apnea, Obstructive
Study to Evaluate the Safety, PK, and Dose Response of Paltusotine in Subjects With Carcinoid Syndrome
Recruiting
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and exploratory dose response of paltusotine treatment in subjects with carcinoid syndrome. This study consists of a Randomized Treatment Phase followed by an Open-Label Extension (OLE) Phase.
Gender:
All
Ages:
18 years and above
Locations: Crinetics Study Site, Boston, Massachusetts +29 locations
Conditions: Carcinoid Syndrome, Carcinoid, Carcinoid Tumor, Carcinoid Tumor of Ileum, Carcinoid Tumor of Cecum, Carcinoid Syndrome Diarrhea, Carcinoid Intestine Tumor, Carcinoid Tumor of Liver, Carcinoid Tumor of Pancreas
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Recruiting
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen or risdiplam or have a history of onasemnogene abeparvovec-xioi, compared to placebo.
Gender:
All
Ages:
Between 4 years and 21 years
Locations: Boston Children's Hospital - Harvard, Boston, Massachusetts +58 locations
Conditions: Spinal Muscular Atrophy, Neuromuscular Diseases, SMA