Search Clinical Trials

80 Results

Recruiting
Sickle Cell Clinical Research and Intervention Program (External Link)
The St. Jude Pediatric SCD Program has developed a comprehensive plan of care that spans the ages of 0 to 25, and provides the structure for screening and monitoring disease …
Baylor Role: Collaborator
Completed
Novel Dose Escalation to Predict Treatment With Hydroxyurea (External Link)
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 (External Link)
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 (External Link)
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
Active, Not Recruiting
Phenylbutyrate Therapy for Maple Syrup Urine Disease (External Link)
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) (External Link)
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
Active, Not Recruiting
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita (External Link)
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 (External Link)
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 (External Link)
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) (External Link)
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