Featured

Status

Health Topics

Any Category
Diseases
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Clear Filters

Search Clinical Trials

80 Results
Terminated
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
If clinically feasible (no aplasia, no active malignancy), the recipients marrow will be harvested and cryopreserved as a back up for use if non-engraftment/rejection is followed by failure to undergo …
Baylor Role: Lead Sponsor
Terminated
Oral Magnesium Pidolate, Hemoglobin SC Disease, MG Pidolate
This is a placebo-controlled study which means that the effectiveness of the magnesium medication (the chemical form of which is known as Mg pidolate) will be compared to placebo. A …
Baylor Role: Lead Sponsor
Terminated
Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
To do the stem cell transplant, we must first kill most of the cells in the bone marrow that make the sickle hemoglobin or abnormal blood cells of severe beta …
Baylor Role: Lead Sponsor
Terminated
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe …
Baylor Role: Lead Sponsor
Unknown Status
Study of Treatment and Metabolism in Patients With Urea Cycle Disorders
PROTOCOL OUTLINE: This protocol involves 2 clinical studies. Part A is a metabolic study of glutamine conversion to urea at different levels of protein intake, while on and off medications. …
Baylor Role: Collaborator
Completed
Study of Phenotype and Genotype Correlations in Patients With Contiguous Gene Deletion Syndromes
PROTOCOL OUTLINE: Patients undergo clinical, cytogenetic, and molecular studies. These include radiographic, neurologic, developmental, and 24 hour sleep studies, ophthalmologic, otolaryngologic, speech and language, and audiologic exams, echocardiogram, and renal …
Baylor Role: Collaborator
Completed
Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome
PROTOCOL OUTLINE: Patients receive an electrocardiogram at baseline and every 6 months for 3 years. Clinical stage II patients undergo 24-hour Holter monitoring on the same schedule; age-matched controls are …
Baylor Role: Collaborator
Completed
Study of the Nutritional, Metabolic, and Body Composition Profile in Children and Adolescents With Epidermolysis Bullosa
PROTOCOL OUTLINE: Data collected from a 5-day food diary are analyzed for protein, nutrient, and calorie intake. Nutritional, metabolic, and body composition are evaluated by indirect calorimetry, total body electrical …
Baylor Role: Collaborator
Completed
Nutritional Aspects of Rett Syndrome
PROTOCOL OUTLINE: All participants undergo extensive nutritional and metabolic assessment with body composition evaluation. Studies include macronutrient intake, body fat, lean muscle mass, metabolic rate, and time-action studies; and 24-hour …
Baylor Role: Collaborator
Completed
Delta Hepatitis and Liver Disease in Hemophiliacs
BACKGROUND: Patients with classical hemophilia (hemophilia A) and Christmas disease (hemophilia B) were exposed to many hepatotropic viruses during the course of their therapy. Severe chronic hepatitis among these patients …
Baylor Role: Lead Sponsor