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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
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
Recruiting
Liver Disease 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
Interleukin-15 Armored Glypican 3-specific Chimeric Antigen Receptor Expressed in Autologous T Cells for Solid Tumors (External Link)
Approximately 118-27 subjects will participate in the treatment part of this study. Maximum of 180 mL of blood (approximately 12 tablespoons) is collected from patients to grow the T cells …
Baylor Role:
Lead Sponsor
Recruiting
L-Citrulline Dose Finding Safety Study in MELAS (External Link)
The human body is made of many cells and each cell contains many mitochondria. Mitochondria are called the powerhouses of the cell, because they produce the energy needed for a …
Baylor Role:
Lead Sponsor
Recruiting
Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress (External Link)
This research study looks to enroll as many people with LFS or TP53 gene variants as possible in order to: - Better estimate cancer risks in individuals with TP53 variants …
Baylor Role:
Collaborator
Recruiting
Technology Development for Noninvasive Prenatal Genetic Diagnosis Using Whole Fetal Cells From Maternal Peripheral Blood (External Link)
This is a revision to a project entitled "Prenatal Genetic Diagnosis by Genomic Sequencing: A Prospective Evaluation." This study proposes to test the utility and accuracy of a new form …
Baylor Role:
Collaborator
Recruiting
Genetic Disorders of Obesity Program Database (External Link)
Children and adolescents who have suffered from severe weight gain prior to 5 years of age and who are seen in the Genetic Disorders of Obesity Program at Texas Children …
Baylor Role:
Lead Sponsor
Recruiting
Clinical and Basic Investigations Into Congenital Disorders of Glycosylation (External Link)
The investigators are conducting a natural history study of patients with congenital disorders of glycosylation (CDG). The study will look into the progression of the disease amongst the participants and …
Baylor Role:
Collaborator
Recruiting
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease (External Link)
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a …
Baylor Role:
Collaborator
Recruiting
Investigation of the Genetics of Hematologic Diseases (External Link)
Participants will be individuals (proband) receiving therapy or expert consultation regarding a non-malignant hematologic disorder. We propose to use genomics, transcriptomics, proteomics and metabolomic analysis coupled with family linkage studies …
Baylor Role:
Collaborator