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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
Completed
Fetal Endotracheal Occlusion (FETO) in Severe and Extremely Severe Congenital Diaphragmatic Hernia (External Link)
Enrollment Women carrying fetuses with severe or extremely severe CDH and a normal karyotype will undergo routine clinical evaluation. The fetuses will be 27+0/7 to 29+6/7 weeks of gestational age …
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
Completed
Calcium Absorption in Patients With Rothmund-Thomson Syndrome (External Link)
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 (External Link)
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
39946-I Laser and Bevacizumab Treatment for Retinopathy of Prematurity (External Link)
This study will enroll preterm infants who were scheduled to receive treatment for ROP by laser or Bevacizumab. We will observe the patients in the hospital for the cure of …
Baylor Role:
Lead Sponsor
Completed
Fetal Surgery for Moderate Left Sided Congenital Diaphragmatic Hernia. (External Link)
Isolated Congenital Diaphragmatic Hernia (CDH) can be diagnosed in the prenatal period, and remains associated with a 30 % chance of perinatal death and morbidity mainly because of pulmonary hypoplasia …
Baylor Role:
Collaborator
Completed
Safe Passages: Ensuring Quality Transitions From NICU (Neonatal Intensive Care Unit) to Ambulatory Care (External Link)
Infants born prematurely or with complex congenital abnormalities are surviving to discharge in growing numbers and often require significant monitoring and coordination of care in the ambulatory setting. These complicated …