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33 Results
Completed
Nitric Oxide Supplementation in Argininosuccinic Aciduria
The investigators will perform a double blind, randomized, placebo-controlled, crossover study of NO supplementation in ASA patients assessing endothelial function and blood pressure as primary endpoints, and markers of nitric …
Baylor Role: Lead Sponsor
Completed
Novel Dose Escalation to Predict Treatment With Hydroxyurea
To be eligible to participate in this study, patients must have decided that they want to begin hydroxyurea therapy. Patients choosing to participate in this study will be assigned randomly …
Baylor Role: Lead Sponsor
Completed
A Phase II Trial of Regadenoson in Sickle Cell Anemia
If you are willing to participate in this research study you will be asked to undergo some screening tests and procedures to confirm your eligibility. Many of these tests and …
Baylor Role: Collaborator
Completed
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
Rett Syndrome is a developmental disorder primarily if not exclusively affecting females. The disorder is characterized by apparent normal development in early infancy (6-18 months), followed by a period of …
Baylor Role: Collaborator
Completed
Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD)(Citrullinemia I)
This is an interventional, case control study to investigate the production of nitric oxide in patients with citrullinemia type I. Through the infusion of isotopes [15N2-ureido] arginine, [5-13C,4, 4, 5, …
Baylor Role: Lead Sponsor
Completed
Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
Subjects with AS, RTT or PWS and normal siblings (controls) will be recruited for study participation. Subjects will be recruited from the Rare Disease Clinical Research Network (RDCRN) consortium registries …
Baylor Role: Lead Sponsor
Completed
Calcium Absorption in Patients With Rothmund-Thomson Syndrome
This is an evaluation of bone calcium deposition in patients with Rothmund-Thomson Syndrome (RTS). This study includes one study visit during which subjects will be admitted to the inpatient unit …
Baylor Role: Lead Sponsor
Completed
A Trial of Levodopa in Angelman Syndrome
Levodopa is a prodrug that "delivers" dopamine to the brain. It is usually given with carbidopa, a peripheral decarboxylase inhibitor, to increase the bioavailability of levodopa. Animal studies have suggested …
Baylor Role: Collaborator
Completed
Nitric Oxide Production in MELAS Syndrome
What does the study involve? Individuals with MELAS syndrome will be admitted twice to the General Clinical Research Center (GCRC) at Texas Children's Hospital. Each time they will stay for …
Baylor Role: Lead Sponsor
Completed
The Role of Dietary Calcium for the Treatment of Osteopenia in Girls With Rett Syndrome
Objective: The investigators conducted a randomized, double-blind, placebo-controlled trial to determine the safety and efficacy of oral calcium supplementation for the treatment of bone mineral deficits in individuals with Rett …
Baylor Role: Lead Sponsor