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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…
Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies
The purpose of this study is to identify genes associated with impaired development and function of the cranial nerves and brainstem, which may result in misal…
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has …
CHAMPION: Combining HIV And Meth Prevention and Treatment Interventions Optimized for HIV-Negative MSM
The goal of this randomized controlled trial is to pilot test new mobile health (mHealth) interventions to improve PrEP adherence among HIV-negative men who ha…
TMS (Transcranial Magnetic Stimulation) for Methamphetamine Use Disorder in People Living With HIV
The purpose of this study is to learn more about how accelerated theta burst stimulation affects methamphetamine craving and brain activity. Theta burst stimul…
A Pilot Study in North Louisiana to Assess the Tolerability of Psilocybin as Well as Its Capacity to Promote Abstinence From Methamphetamine
The primary purpose of this study is to preliminarily determine if the use of psilocybin to promote abstinence from methamphetamine is feasible and well tolera…
Phentermine/Topiramate in Children, Adolescents, and Young Adults With Hypothalamic Obesity: a Pilot and Feasibility Study
Hypothalamic obesity (HO) refers to the substantial weight gain that often complicates hypothalamic brain tumors. Children with this treatment-recalcitrant for…
International Rapid-onset Obesity with Hypothalamic Dysfunction, Hypoventilation, and Autonomic Dysregulation (ROHHAD) REDCap Registry
The Center for Autonomic Medicine in Pediatrics (CAMP) has collaborated with doctors from around the world to build the first International ROHHAD (Rapid-onset…
A Clinical Comparison in Wound Healing of Bikini Versus Longitudinal Incision in Direct Anterior Approach Total Hip Arthroplasty in the Obese Patient Population: A Randomized Control Trial
The study team is conducting this study to see if there is a difference between the wound healing and participant satisfaction rates between two incision types…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
Rural Community Peer Partnerships for Improving Methamphetamine-Associated Heart Failure Screening and Engagement
The goal of PEER-Heart is to implement and assess feasibility and preliminary effectiveness of a community peer-facilitated screening for methamphetamine-assoc…
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…