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Congenital, Hereditary, and Neonatal Diseases and Abnormalities
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- Anemia, Hemolytic, Congenital 3
- Anemia, Hypoplastic, Congenital 1
- Eye Diseases, Hereditary 1
- Genetic Diseases, X-Linked 6
- Hemoglobinopathies 4
- Heredodegenerative Disorders, Nervous System 2
- Loeys-Dietz Syndrome 1
- Marfan Syndrome 1
- Metabolism, Inborn Errors 5
- Neoplastic Syndromes, Hereditary 3
- Osteogenesis Imperfecta 3
- Skin Diseases, Genetic 2
Search Clinical Trials
Recruiting
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (External Link)
This is a phase I/II study of haploidentical HCT (HHCT) with ex vivo TCRαβ+ and CD19+ depletion using the CliniMACS device in patients with hematological malignancies and non-malignant disorders. HHCT …
Baylor Role:
Lead Sponsor
Recruiting
Marfan Syndrome Moderate Exercise Trial II (External Link)
Marfan syndrome (MFS) is a distinctive connective tissue disorder with clinical phenotypes that affect multiple organ systems including the heart, bones, ligaments, and eyes, and are associated with significant risk …
Baylor Role:
Lead Sponsor
Recruiting
Implementation of Whole Genome Sequencing as Screening in a Diverse Cohort of Healthy Infants (External Link)
The objective of this research protocol is to assess the impacts of genomic sequencing in healthy infants from ethnically and racially diverse communities as part of routine pediatric care. Investigators …
Baylor Role:
Collaborator
Recruiting
Rett Syndrome Registry (External Link)
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence …
Baylor Role:
Collaborator
Recruiting
Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708 (External Link)
This study is cross-sectional. At the participant's one study visit, data will be obtained at a single point in time and reflect the patients' current condition. Evaluations will include family …
Baylor Role:
Lead Sponsor
Recruiting
Clear Aligners for the Treatment of Dental Malocclusion in OI (External Link)
Osteogenesis imperfecta (OI) is a heritable connective tissue disorder leading to frequent fractures and bone deformities. In about 90% of individuals with OI, the disease is caused by dominant mutations …
Baylor Role:
Lead Sponsor
Recruiting
Oxytocin for Hypermobile Ehlers-Danlos Syndrome (External Link)
This will be a single-site study to evaluate the effect of IV oxytocin on chronic pain in female adult patients with hypermobile EDS. Oxytocin secretion in the body is dynamic …
Baylor Role:
Lead Sponsor
Recruiting
Hepatic Histopathology in Urea Cycle Disorders (External Link)
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 …
Baylor Role:
Lead Sponsor
Recruiting
The Rett Syndrome Global Registry (External Link)
The Rett Syndrome Global Registry is a fully remote, global, caregiver-reported registry intended to meet the needs of caregivers, clinicians and researchers, and therapeutic developers with the goal to increase …
Baylor Role:
Collaborator
Recruiting
Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors (External Link)
Approximately 15-24 subjects will participate in the treatment part of this study. Maximum of 180 mL of blood (not exceeding 3ml/kg/day) is collected from patients to grow the T cells …
Baylor Role:
Lead Sponsor