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Found 24 Birth Defects trials

A listing of Birth Defects medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

18 years - 99 years
All genders
Interventional
This research study is being conducted to collect information on those who have been diagnosed with a disease related to a heart valve dysfunction or abnormality, needing surgical repair, or replacement of the affected valve. This research study is collecting information about the disease and the treatment. Participants will be …
99 years or below
All genders
With vastly improved survival for babies born with congenital heart disease (CHD), there is increasing attention on long-term outcomes in adulthood, especially those born with severe forms of CHD. One area of intense research is liver disease in Fontan patients, the nature of which is incompletely understood. It is known …
 Clinical Transcriptomics in Systemic Vasculitis (CUTIS)
99 years or below
All genders
This study employs a multi-center approach to evaluate cutaneous vasculitis across several forms of idiopathic vasculitis. Patients with cutaneous manifestations of vasculitis will be evaluated by teams of primary vasculitis care providers and Dermatologists in order to facilitate optimal selection of patients and sampling of lesions. A punch skin biopsy …
18 years - 99 years
All genders
Mortality related to cardiovascular disease in the US is improving. Yet the rate of decline is less for women than for men. Womens health is unique due to the varying reproductive stages, and differences are even greater due to gender-specific characteristics of the heart and vasculature. While women have some unique risk factors, …
99 years or below
All genders
Phase 1
Amphivena Therapeutics, Inc. (Amphivena) is studying an investigational drug called AMV564, which may help to treat acute myeloid leukemia (AML). The purpose of this clinical research study is to find the highest tolerable dose that can be given to patients with AML, and to recommend a dose to be used …
99 years or below
All genders
Phase 1
The purposes of this study are to see if the study drugs, venetoclax and gilteritinib can be safely and effectively combined for the treatment of patients with acute myeloid leukemia (AML) that has returned after prior treatment, or has failed to respond to prior treatment and identify potential biomarkers.
 Determination of B-cell responsiveness to the incretin hormones GLP-1 and GIP in Cystic Fibrosis (PRIME)
18 years - 99 years
All genders
The mechanisms underlying cystic fibrosis related diabetes (CFRD) pathophysiology must be better defined if care for people with CFRD is to be optimized or if development of CFRD is to be interrupted. Detailed studies of insulin secretion in CF are required to achieve this goal since 1) subtle insulin secretion …
 Study Evaluating Safety and Efficacy of UCART123 in Patients With Relapsed/ Refractory Acute Myeloid Leukemia (AMELI-01)
18 years - 65 years
All genders
Phase 1
Interventional
This study is evaluating the safety and efficacy of UCART targeting CD123 in patients with relapsed/refractory acute myeloid leukemia (AML). The purpose of this study is to evaluate the safety and clinical activity of UCART123v1.2 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D).
 Phase I Trial of Programmed Allogeneic CRISPR-edited T cells (PACE) Gene Edited to Eliminate Endogenous TCR  HLA-class I and HLA-class II and Engineered to Express anti-CD19 Chimeric Antigen Receptor (PACE CART19) in Patients with Relapsed Or Refractory CD19+ Leukemia and Lymphoma
18 years - 99 years
All genders
Phase 1
Interventional
This research study is being conducted to evaluate an experimental drug called PACE CART19 in patients with leukemia or lymphoma. PACE CART19 consists of cells from a healthy donor that are genetically modified and designed to target your cancer.
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