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121 Results
Terminated
Randomized Clinical Trial of Early Delivery in Fetal Gastroschisis vs. Routine Care
Gastroschisis is a congenital malformation in which abdominal organs protrude through an abdominal wall defect, leading to considerable morbidity and mortality during infancy. In contrast to the second class of …
Baylor Role: Lead Sponsor
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
Terminated
Pentoxifylline Therapy in Biliary Atresia
Biliary atresia (BA) is a devastating liver disease of infancy of unknown etiology, characterized by bile duct obstruction, live fibrosis, and cirrhosis. BA has no known medical treatments. The only …
Baylor Role: Lead Sponsor
Active, Not Recruiting
Phenylbutyrate Therapy for Maple Syrup Urine Disease
Maple syrup urine disease is a severe inborn error of amino acid metabolism caused by deficiency of the mitochondrial branched-chain alpha-ketoacid dehydrogenase complex (BCKDC) resulting in the accumulation of branched-chain …
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
Terminated
A Comparison Trial Between PCA and Epidural Analgesia for Pectus Excavatum Repair
Pectus excavatum is a cartilaginous deformity that is the most common congenital anterior chest wall defect in children [1]. Children with this disease process often complain of dyspnea, decreased exercise …
Baylor Role: Lead Sponsor
Active, Not Recruiting
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita (DC) is an inherited multisystem disorder, which classically presents with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. DC is part of a spectrum …
Baylor Role: Collaborator
Terminated
Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea
Despite the clear results of the STOP and the follow-up STOP II trials, the use of chronic erythrocyte transfusions for primary stroke prevention in children with Sickle Cell Anemia (SCA) …
Baylor Role: Collaborator
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
Terminated
Early Methicillin-resistant Staphylococcus Aureus (MRSA) Therapy in Cystic Fibrosis (CF)
The STAR-too study is a randomized, open label, multi-center study in CF patients with new MRSA isolated from the respiratory tract (sputum or oropharyngeal (OP) swab). The purpose of the …
Baylor Role: Collaborator