Clinical Trial

Disease: Sickle Cell Trait, (NCT06071377)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Carriers of the disease, or individuals with sickle cell trait, have only one copy of the sickle mutation and produce variable amounts of the abnormal sickle hemoglobin (25-45% of total hemoglobin).

Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide.
Official title:
Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)


Name: Julie Kanter, MD

Phone: (202) 596-1548



National Alliance for Sickle Cell Centers


United States, Alabama 
University of Alabama, Birmingham, Alabama, United States, 35294

United States, California 
Loma Linda University Health Care, Loma Linda, California, United States, 92354

United States, Indiana 
Indiana University, Indianapolis, Indiana, United States, 46202

United States, Maryland 
Johns Hopkins University, Baltimore, Maryland, United States, 21224

United States, North Carolina 
University of North Carolina, Chapel Hill, North Carolina, United States, 27599
East Carolina University, Greenville, North Carolina, United States, 27834

Study start:
Apr. 1, 2023
1000 participants
Gene editing method:
Delivery method:
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.

Last updated: Jan. 16, 2024
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