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NCT ID: NCT04239625 Enrolling by invitation - Stargardt Disease Clinical Trials

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Start date: December 20, 2019
Phase: Phase 2
Study type: Interventional

The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only. Funding Source - FDA OOPD

NCT ID: NCT04804176 Enrolling by invitation - Parkinson Disease Clinical Trials

Study on the Mode and Mechanism of Ultra-low Frequency rTMS for Sleep Disorders in Patients With Parkinson's Disease

Start date: December 20, 2019
Phase: N/A
Study type: Interventional

The study by giving a sleep disorder Parkinson's disease patients with different patterns of ultra-low frequency transcranial magnetic stimulation or sham stimulation, scale for assessment of the patients were observed, hematology and imaging changes before and after therapy, clear ultra-low frequency transcranial magnetic stimulation for sleep disorder Parkinson's disease treatment, to explore the mechanism of action, compare the difference between different modes.

NCT ID: NCT06072079 Enrolling by invitation - Genetic Disease Clinical Trials

Structural Chromosome Rearrangements and Brain Disorders

Start date: December 20, 2019
Phase:
Study type: Observational

The project is focused on the detailed study of structural genomic variants (SVs). Such genetic mutations are in fact alterations in the DNA molecule structure and include copy number variants, inversions and translocations. A single event may affect many genes as well as regulatory regions and the specific phenotypic consequences will depend on the location, genetic content and type of SV. Many times, the specific disease-causing mechanism is not known. Here, we plan to study the molecular genetic behavior of structural variants as well as the underlying mutational mechanisms involved. First, we will use genome sequencing to pinpoint the chromosomal breakpoints at the nucleotide level, characterize the genomic architecture at the breakpoints and study the relationship between structural variants and SNVs. Second, we will study how structural variants impact gene expression. Finally, we will functionally explore the disease mechanisms in vivo using zebrafish and in vitro using primary patient cells and induced pluripotent stem cells. Our studies will focus on the origin, structure and impact of structural variation on human disease. The results will directly lead to a higher mutation detection rate in genetic diagnostics. Through a better understanding of disease mechanisms our findings will also assist in the development of novel biomarkers and therapeutic strategies for patients with rare genetic disorders.

NCT ID: NCT04724993 Enrolling by invitation - Physical Activity Clinical Trials

Effects of Online Aerobic Exercise Training and Physical Activity Counseling in Juvenile Familial Mediterranean Fever

Start date: December 20, 2020
Phase: N/A
Study type: Interventional

The aim of the study is to investigate comparison the effectiveness of online aerobic dance exercises versus physical activity counselling in patients with Juvenile Familial Mediterranean Fever.

NCT ID: NCT05210803 Enrolling by invitation - AMD Clinical Trials

Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD

Start date: December 20, 2021
Phase:
Study type: Observational

This is a prospective, observational study designed to evaluate the long-term safety and efficacy of RGX-314. Eligible participants are those who were previously enrolled in a clinical study of nAMD in which they received suprachoroidal space (SCS) administration of RGX-314. Enrollment of each participant in the current study should occur after the participant has completed either the end of study or early discontinuation visit in the previous (parent) clinical study. Participants will be followed for up to 5 years after RGX-314 administration (inclusive of the parent study). As such, the total study duration for each participant may vary depending on when they enroll in the current study following RGX-314 administration in the parent study.

NCT ID: NCT05334784 Enrolling by invitation - Clinical trials for High-Risk Percutaneous Coronary Intervention

Impella ECP Study (ECP Study) and Impella ECP Continued Access Protocol

Start date: December 20, 2022
Phase: N/A
Study type: Interventional

The Impella ECP Study is a prospective, multi-center, single-arm study evaluating the rate of major adverse cardiovascular and cerebrovascular events (MACCE) with the Impella ECP device in adult patients undergoing elective or urgent high-risk percutaneous coronary intervention. The above applies to Impella ECP Continued Access Protocol

NCT ID: NCT05422573 Enrolling by invitation - Genetic Counseling Clinical Trials

Clinical Trial of the Sequence of Cardiovascular Genetic Counseling and Testing

RESEQUENCEGC
Start date: December 20, 2022
Phase: N/A
Study type: Interventional

Although pre-test genetic counseling is widely recommended and has come to dominate genetic counseling practice, tailored results-focused genetic counseling could both increase genetic counseling efficiency and improve genetic counseling outcomes for the growing number of patients seeking genetic testing for recommended genome-guided medical management. This study will test that hypothesis in adults referred for cardiovascular genetic counseling and testing at the Johns Hopkins Center for Inherited Heart Diseases. This study is a three-arm randomized clinical trial to evaluate two complementary approaches to shifting the primary genetic counseling session to post-test for 510 adults with two broad cardiovascular genetic counseling indications: diagnostic panel testing and family-specific variant testing. The investigators will compare usual care (pre-test genetic counseling appointment, results returned by phone / electronic health record) with online video-based pre-test tailored genetic education with an optional (efficiency arm) or required (flipped arm) phone call with a genetic counselor followed by a post-test genetic counseling appointment. The investigators hypothesize that post-test genetic counseling will: 1) increase efficiency, 2) promote patient empowerment and adherence, and 3) have similar genetic test-associated psychosocial impact.

NCT ID: NCT05839847 Enrolling by invitation - Anxiety Clinical Trials

LIFU for Anxiety Management

Start date: December 20, 2023
Phase: N/A
Study type: Interventional

This study is examining the effects of noninvasive brain stimulation on anxiety. Low-intensity focused ultrasound (LIFU) will be utilized to neuromodulate the brain prior to a task (NPU). Physiological recordings will be taken throughout (EEG, EMG, HR, BP, RR, GSR).

NCT ID: NCT06058728 Enrolling by invitation - Clinical trials for Uterine Fibroids (UF)

A Study to Evaluate Changes in Hair in Adult Participants Taking Oral Oriahnn Capsules With Heavy Menstrual Bleeding (HMB) Associated With Uterine Fibroids (UF)

Start date: December 20, 2023
Phase:
Study type: Observational

Uterine Fibroids (UF) are benign smooth muscle neoplasms of the uterus that affect women of reproductive age. UFs are one of the leading causes of hospitalizations for gynecological disorders and often lead to hysterectomy. In this study, women with heavy menstrual bleeding (HMB) due to UF who are being treated with Oriahnn will be followed to establish the incidence rate, time to onset, extent, pattern, and resolution of meaningful hair loss, as well as any racial differences. Oriahnn is an approved drug for the management of HMB associated with UF. All study participants will receive Oriahnn as prescribed by their study doctor in accordance with approved local label. Study Participants will be followed for up 24 months (part 1). Approximately 1600 participants aged 18-50 years will be enrolled at 1 site in the United States. Participants will receive oral Oriahnn as prescribed by their physician according to their routine clinical practice and local label. Participants will be followed for 24 months. There is expected to be no additional burden for participants in this trial. Participants will complete questionnaires on a quarterly basis.

NCT ID: NCT06173635 Enrolling by invitation - Healthy Volunteers Clinical Trials

A Comparison Study of the Maxi Move 5 (MM5) Device

Start date: December 20, 2023
Phase: N/A
Study type: Interventional

This trial aims to evaluate the added improvements from a caregiver and patient benefit perspective. The trial should identify the forces involved for the caregiver when manoeuvring the lift.