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Genetic Disease, Inborn clinical trials

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NCT ID: NCT05425745 Active, not recruiting - Clinical trials for Hypercholesterolemia

Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.

BROOKLYN
Start date: July 25, 2022
Phase: Phase 3
Study type: Interventional

This study will be a placebo-controlled, double-blind, randomized, phase 3 study to Evaluate the Efficacy, Safety, and Tolerability of Obicetrapib in Participants with a History of Heterozygous Familial Hypercholesterolemia (HeFH).

NCT ID: NCT02916888 Completed - Dermatitis, Atopic Clinical Trials

A Study Comparing the Quality of Life of Patients in the Treatment of Eczema by Pediatric Generalists and Specialists

Start date: September 2016
Phase: N/A
Study type: Observational

The purpose of this study is to investigate the differences in the quality of life of patients and caregivers who are treated by general pediatricians versus pediatric dermatologists for eczema (atopic dermatitis or AD).

NCT ID: NCT00001405 Recruiting - Granuloma Clinical Trials

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

Start date: February 27, 1994
Phase:
Study type: Observational

The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that will be used in the laboratory and/or in the clinic to develop new cell therapies for patients with inherited or acquired disorders of immunity or blood cells. Development of novel cellular therapies requires access to HSC, Mononuclear Cells and/or granulocytes as the essential starting materials for the pre-clinical laboratory development of gene therapies and other engineered cell products. HSC or blood cells from healthy adult volunteers serve both as necessary experimental controls and also as surrogates for patient cells for clinical scale-up development. HSC or blood cells from patients serve both as the necessary experimental substrate for novel gene therapy and cellular engineering development for specific disorders and as pre-clinical scale up of cellular therapies. Collection of cells from adult patients collected in the NIH Department of Transfusion Medicine (DTM) under conditions conforming to accepted blood banking clinical practice may also be used directly in or cryopreserved for future use in other NIH protocols that have all required regulatory approvals allowing such use. In summary, the research goal of this protocol is the collection of HSC or blood cells that may be used for both laboratory research and/or for clinical treatment in other approved protocols.