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A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…
Phase 1/2 Study: CD45RA Depleted Peripheral Stem Cell Addback to Prevent Viral and Fungal Infections Following Alternative Donor TCRab/CD19 Depleted Hematopoietic Stem Cell Transplant
The major morbidities of allogeneic hematopoietic stem cell transplant (HSCT) using donors that are not human leukocyte antigen (HLA) matched siblings are graf…
The Effects of Disulfiram on Visual Acuity in Patients With Retinal Degeneration
Oral disulfiram (Antabuse®) has been shown to improve image-forming vision in animal models with retinal degeneration due to its ability to decrease Retinoic A…
Prospective Observational Study of the ICD in Sudden Cardiac Death Prevention (PROSe-ICD)
The overall hypothesis of this study is that subtle interactions between structural (substrate) and functional (trigger) abnormalities of the heart, some of wh…
A High-Performance ECoG-based Neural Interface for Communication and Neuroprosthetic Control
Test the feasibility of using electrocorticography (ECoG) signals to control complex devices for motor and speech control in adults severely affected by neurol…
Qualification for Cone-Optogenetics (EyeConic)
This study aims to prepare for the first-in-human clinical trial of cone optogenetics vision restoration. As a first step, this worldwide multicenter ocular im…
The Utility of pGz in Primary Mitochondrial Disorders
This is a multi-aim study, studying the effects of conventional exercise (measured through Cardiopulomary Exercises Testing or an in-bed pedal exercise) and pa…
A Multicenter, Prospective, Randomized Controlled Modified Platform Trial Assessing the Efficacy of Human Placental Membrane Products and Standard of Care Versus Standard of Care Alone in the Management of Nonhealing Diabetic Foot Ulcers and Venous Leg Ulcers.
The purpose of the study is to evaluate the efficacy of multiple human placental membrane products and Standard of Care (SOC) versus SOC alone in the managemen…
Modifiable Variables in Parkinsonism (MVP) [Formerly CAM Care in PD]
We are trying to identify factors associated with improved quality of life and fewer PD symptoms. We are attempting to identify practices, beliefs, and therapi…
Prospective Randomized Pilot Trial of Formoterol in Patients With Diabetic Kidney Disease
The purpose of the study is to evaluate if formoterol fumarate is effective in treating patients with diabetic kidney disease. Study participants will be rando…
Cannabidiol for the Treatment of Diabetic Peripheral Neuropathy: Pilot Study
The "Cannabidiol for the Treatment of Diabetic Peripheral Neuropathy: Pilot study (CBD-DPN1)" is a double-blinded, placebo-controlled, crossover pilot study ev…
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…