Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests....

Type: Interventional

Start Date: Oct 2019

open study

Background: - Salivary glands in and around the mouth and throat make saliva. Salivary gland disorders can affect a person s quality of life. Studying people who have a disease that affects their salivary gland(s) may teach researchers about the disorders and their genetics. Objectives: - To study salivary gland diseases and disorders. To collect data and samples from people with salivary gland problems and their relatives. Eligibility: - People more than 4 years old who have or are suspected to have a disease involving salivary glands. - Their relatives more than 4 years old. - Healthy volunteers 18 years or older. Design: - Participants may be screened with: - Medical history - Physical exam - Blood and urine tests - General oral and dental history and exam - Saliva collection - Eye exam and test for dry eyes - Health questionnaires (adults) - Biopsy of some minor salivary glands. A small incision will be made on the inside of the lower lip and several tiny salivary glands will be removed. - Participants will have 2-3 visits. These may include: - Repeats of some screening tests - Ultrasounds of some glands. Researchers will put some gel on the face, then press on it with a smooth wand. - Adults may have other biopsies - A small catheter inserted into the opening of the parotid gland duct on the inside of the cheek. A saline solution (in a syringe) will fill the duct. - Swishing a saltwater solution in the mouth for 10 seconds and then spitting into a cup - Scrapings collected from teeth, tongue, and cheeks

Type: Observational

Start Date: Apr 2015

open study

Background: - Spondyloarthritis (SpA) is a group of bone and joint disorders that may cause back and joint pain and stiffness. In some cases, SpA can lead to abnormal bone growth affecting the joints and spine. Some patients have SpA without ever developing these growths, while others develop them after only a few years. Researchers are interested in studying people with SpA and their relatives to determine which people are more likely to develop more severe conditions. Objectives: - To identify symptoms and medical tests that can help determine whether a person with SpA is at risk for developing more severe forms of the disease. Eligibility: - Individuals of any age who have been diagnosed with SpA. - Healthy volunteer relatives (at least 6 years of age) of the individuals with SpA. Design: - Participants will be screened with medical records and family medical histories, and will be invited to the clinical center for the study. - Participants with SpA will have a physical exam and medical history, including a study of joint movement, blood and urine tests, and questionnaires about pain and quality of life. - Participants with SpA will have imaging studies, including magnetic resonance imaging (MRI). Other samples such as skin tissue and bone marrow may also be collected for study. - Healthy volunteers will provide a blood sample and cheek cell samples. - No treatment will be provided, although treatment options will be discussed....

Type: Observational

Start Date: Aug 2011

open study

We will investigate the clinical manifestations and molecular genetic defects of heritable urologic malignant disorders. Families with urologic malignancy with known or suspected genetic basis will be enrolled. Affected individuals or individuals suspected of having a germline urologic malignant disorder will undergo periodic clinical assessment and genetic analyses for the purpose of: 1) definition and characterization of phenotype, 2) determination of the natural history of the disorder, and 3) genotype/phenotype correlation. Genetic linkage studies may be performed in situations in which the genetic basis of the disorder has not been elucidated. ...

Type: Observational

Start Date: Dec 1990

open study

The goal of this study is to learn more about the brain activity underlying Parkinson's disease risk taking and reward seeking behaviors. The investigators will utilize neural recordings from corticostriatal structures performed during deep brain stimulation surgery to measure neural activity underlying nonmotor symptoms of Parkinson's disease.

Type: Interventional

Start Date: Sep 2024

open study

This is a United States (US) based, prospective, non-interventional, provider-referral study to evaluate the real-world effectiveness and patient-centered outcomes of remibrutinib in chronic spontaneous urticaria (CSU) patients using validated patient reported outcome (PRO) tools.

Type: Observational

Start Date: Jan 2026

open study

Many individuals with severe motor impairments rely on Assistive Technologies (ATs) or Brain-Computer Interfaces (BCIs) to interact with digital devices such as their computers. Clinicians and researchers currently lack a common framework to objectively quantify how much a given AT or BCI improves real-world function or to compare across tools. This project seeks to address this gap by developing a standardized method to objectively assess or compare the functional benefit of these tools on digital independence, i.e., the ability to independently operate computers, phones, and other digital systems, by creating a unique Digital Assessment Interface (DAI). This assessment will be a simulation of online and digital activities that prior work has determined is important to functional daily living in the digital domain. Participants will complete this assessment with various ATs and BCIs, and these scores will be used to create an index, which will be comprised of performance outcomes, clinician-reported outcomes, and patient-reported outcomes. The tool aims to quantify and compare digital task performance across devices and user populations. The primary objective of this study is to develop an index. The index will quantify functional performance of individuals using various ATs and BCIs. The secondary objectives are to extensively evaluate the psychometric properties of the index, such as the validity, responsiveness, reliability, and floor/ceiling effects both globally and across different devices and impairment levels, ensuring that it can reliably measure the impact of an AT or BCI on a user's ability to independently operate digital systems; and to characterize the familiarization and use of specific BCI and AT systems with reference to a normative healthy control population.

Type: Interventional

Start Date: Jan 2026

open study

In order to systematically improve code status communication and documentation while clarifying how providers understand code status decisions, we propose the Choice of Diction's Effect Electronic Measures of Resuscitation Study Inpatient (CODE-EMRS.I) with 3 aims: Aim 1 - Determine rate of patient utilization of code status invitation (research) via the Portal (Hypothesis 1: Participants with an existing Portal are more likely to participate in research than new signups); Aim 2 - Evaluate different phrasings in code status prompts with and without death language (Hypothesis 2: Participants are more likely to pick no code with death language than without); Aim 3 - Determine how objective data drives physician agreement on code status decision (Hypothesis 3: Physicians are more likely to disagree with full code decisions for poor GO-FAR, but not CCI). Participants will fill out all study questionnaires electronically, but have options within these to ask to speak to a study physician/their own physician for clarification. After completing the surveys, the research associate will deliver the patient's code status decision to the attending of record and ask their views on it. Once a week, participants who have expressed interest in the study (by clicking the "I am interested" button) but have not completed the study will receive a reminder to complete the study as well as an offer to withdraw from the study in that same communication.

Type: Interventional

Start Date: Feb 2026

open study

The purpose of this study is to determine the feasibility of administering a predetermined amount of normal saline into the intrathecal or subarachnoid space via a small spinal catheter to reduce or eliminate the effects of previously injected spinal anesthetic following lower extremity orthopedic surgery.

Type: Interventional

Start Date: Feb 2026

open study

The purpose of this study is to evaluate the pharmacokinetics, safety, and tolerability of NSHO-101 in Healthy Participants.

Type: Interventional

Start Date: Jan 2026

open study

The researchers are doing this study to find out whether obecabtagene autoleucel (obe-cel) is an effective treatment for people with B-cell acute lymphoblastic leukemia (ALL) that is in complete remission (CR, meaning all signs of cancer are gone) with no measurable residual disease (MRD-negative, meaning there are no detectable cancer cells). Participants in this study will have received past treatment for their B-cell ALL, and their disease will be in MRD-negative CR for the first time (first MRD-negative CR).

Type: Interventional

Start Date: Feb 2026

open study

A Study of ALV-100 to Assess Safety, Tolerability, and PK/PD in Overweight/Obese Participants with or without Type 2 Diabetes

Type: Interventional

Start Date: Dec 2025

open study

The study is a Phase 3, double-blind, placebo-controlled, randomized withdrawal study to assess the efficacy and safety of AXS-14 in the management of fibromyalgia.

Type: Interventional

Start Date: Jan 2026

open study

The main purpose of this study is to evaluate the efficacy and safety of eloralintide compared with placebo in participants with persistent obesity or overweight, with or without type 2 diabetes, and on stable incretin background therapy. Participation in the study will last about 80 weeks.

Type: Interventional

Start Date: Feb 2026

open study

This is a pilot clinical study designed to evaluate the effects of a dietary supplement on selected blood-based nutritional biomarkers in healthy children aged 4 to 15 years. Approximately 24 children will be screened and enrolled to account for potential dropouts, with a target of 20 participants completing the study. Eligible participants will attend a screening and baseline visit, followed by daily intake of the study supplement for 4 weeks. After the supplementation period, participants will return to the clinic for an end-of-study visit. Blood samples will be collected at baseline and after 4 weeks to measure nutritional biomarkers, including vitamin D, vitamin B12, folate, omega-3 fatty acids, and standard blood count parameters. Additional assessments include height, weight, body mass index (BMI), and a socioeconomic questionnaire. Throughout the study, participants or their caregivers will record daily supplement intake, any medications taken, and any adverse events in a study diary. The results of this pilot study will provide preliminary data on the effects of the dietary supplement on serum biomarkers in children.

Type: Interventional

Start Date: Jan 2026

open study

The objective of this study is to identify determinants for implementing the i-STAT Alinity whole blood traumatic brain injury (TBI) test for its Food and Drug Administration (FDA)-indicated use and to evaluate other potential outcomes with clinical implications. The main questions it aims to answer are: 1. Determine the number of patients with non-elevated (i.e., below clinical cutoff) whole blood iSTAT Alinity tests who also receive CT scans. 2. What are the obstacles for using the i-STAT Alinity for its FDA-indicated use

Type: Observational

Start Date: Jan 2026

open study

This observational study aims to identify the factors that influence whether cancer patients accept or decline supportive care services after screening positive for health-related social needs (HRSNs) at the Sidney Kimmel Comprehensive Cancer Center. The study focuses on adult oncology patients who previously reported at least one HRSN-such as transportation, food, housing, or utility needs-but declined assistance when it was offered. The main questions it aims to answer are: - What factors shape cancer patients' intentions to use supportive care services for HRSNs? - Why do patients who screen positive for HRSNs choose not to accept help offered by the healthcare system? Participants will: - Complete a one-time electronic survey delivered via MyChart or email. - Optionally participate in a semi-structured Zoom interview (for a subset of 20-30 survey respondents) to explore decision-making in greater depth.

Type: Observational

Start Date: Dec 2025

open study

The main goal of this study is to evaluate whether a manually determined sleep extension intervention is effective at improving sleep and related outcomes among adults who find it difficult to get enough sleep.

Type: Interventional

Start Date: Mar 2026

open study

The purpose of this study is to determine the safety and tolerability of monotherapy CR-001 and identify the maximum tolerated dose (MTD), and/or optimal biologic dose and Recommended Phase 2 Dose(s) (RP2D) in participants with locally advanced or metastatic solid tumors.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this observational study is to 1) better understand and predict biological processes before disease begins or is identified, 2) study genomic and environmental contributors to disease, 3) identify ways to stop disease advancement before it becomes serious or complex, and 4) identify potential targets for disease therapy. Participants will be asked to: - collect biological samples, - download a mobile app, - collect speech (voice) recordings, and - complete surveys

Type: Observational

Start Date: Jan 2026

open study

A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and provide information relevant to clinically meaningful functional improvement. This has been a particular challenge in rare and slowly progressive diseases such as Duchenne Muscular Dystrophy (DMD). The investigators hypothesize that urinary N-terminal fragment of titin (NTFT) corresponding to activity level/intensity will define a high-precision, non-invasive biomarker of systemic muscle injury to enable serial measurements of efficacy and safety in the clinical investigation of gene therapy for DMD and other myopathies. This should provide a valuable exploratory, secondary and eventually primary outcome measure of therapeutic efficacy to minimize the enrollment size in informative early phase and pivotal clinical trials.

Type: Interventional

Start Date: Mar 2026

open study

This study will look at how centanafadine works when taken together with stimulant medicines in healthy adults, and whether combining them affects how the body responds.

Type: Interventional

Start Date: Feb 2026

open study

This is a prospective, single arm, open-label, interventional, pilot study to evaluate the accuracy and precision of the continuous glucose monitoring (CGM) system of i-SENS, Inc., CareSens Air 3, in adult patients with type 1 diabetes.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. Food and Drug Administration to treat SOD1-ALS. The main questions it aims to answer are: - Does tofersen lower the levels of neurofilament light chain (NfL) in the blood and CSF of adult participants with non-SOD1 ALS? - Is tofersen safe and tolerable for adult participants with non-SOD1 ALS? - Does tofersen affect other measurements such as clinical outcomes and quality-of-life measures in participants with non-SOD1 ALS? Participants will : - Receive 100mg tofersen via lumbar puncture for 24 weeks. The doses are at the following time points: Weeks 0, 2, 4, 8, 12, 16, 20, and 24. - Complete 2 follow-up visits following the end of the dosing period at Weeks 28 and 32. - Complete a variety of questionnaires and outcome measurements such as strength and breathing testing.

Type: Interventional

Start Date: Dec 2025

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