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33 Results
Completed
Hypotension in Children With Down Syndrome With Anesthesia Induction
Children generally undergo induction of anesthesia by inhalation of sevoflurane. This is particularly relevant for children with developmental delays as to avoid placement of an IV while awake. However, children …
Baylor Role: Lead Sponsor
Completed
Marfan Syndrome Moderate Exercise Pilot
Marfan syndrome (MFS) affects multiple organ systems including the heart, bones, ligaments, and eyes, and is associated with significant risk of aortic dissection. Given anecdotal reports of aortic dissection, limited …
Baylor Role: Lead Sponsor
Completed
Pimavanserin Treatment in TS
Tourette Syndrome is typically treated with drugs that block or reduce the function of dopamine (a brain chemical involved in movement). However, many of these drugs have adverse effects including …
Completed
Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid …
Baylor Role: Collaborator
Completed
Chronic Liver Disease in Urea Cycle Disorders
Urea cycle disorders (UCDs) are among the most common inborn errors of liver metabolism.With early diagnosis and improved treatments, the survival of individuals with UCDs has improved, and this improved …
Baylor Role: Lead Sponsor
Completed
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in …
Baylor Role: Lead Sponsor
Completed
Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD
Argininosuccinate lyase deficiency (ASLD; also known as argininosuccinic aciduria) is the second most common urea cycle disorder (UCD) and accounts for 15-20% of all disorders of ureagenesis. Individuals with ASLD …
Baylor Role: Lead Sponsor
Completed
Fetal Hemoglobin Induction Treatment Metformin
This is a dose escalation, pilot study for subjects with sickle cell anemia (SCA) disease to determine if metformin has a beneficial effect on the treatment and quality of life …
Baylor Role: Lead Sponsor
Completed
Genomic Sequencing for Childhood Risk and Newborn Illness
The objective of this research protocol is to conduct a randomized clinical trial to assess the benefits and risks of adding the information from a genomic sequencing report to physician-mediated …
Baylor Role: Collaborator
Completed
Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders
The investigators will study and compare how effectively sodium phenylbutyrate, sodium benzoate, and a combination of the two, help excrete nitrogen in healthy volunteers. Subject participation will require three, separate, …
Baylor Role: Lead Sponsor