Clinical Effects of Two Dose Regimens of Aes-103 in Sickle cell
Research type
Research Study
Full title
A Phase 2, Exploratory, Placebo-Controlled, Multicenter, Double-Blind Evaluation of the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Effects of Two Dose Regimens of Aes-103 Given for 28 Days to Subjects with Stable Sickle Cell Disease
IRAS ID
129787
Contact name
Tim Mant
Contact email
Sponsor organisation
AesRx,LLC
Eudract number
2013-001534-18
Research summary
This study will investigate whether the drug AES-103, being developed by AesRx,LLC, is well tolerated and whether it may be of benefit for patients with sickle cell disease. The study will examine the clinical effects, safety, tolerability and the way the body handles (absorbs, distributes, breaks down and excretes) the drug AES-103 given by mouth for 28 days using 2 different dosing regimes in volunteers with stable sickle cell disease.
Sickle cell disease is an inherited genetic life-long blood disorder characterized by red blood cells that assume an abnormal, rigid, sickle shape when oxygen is at lower levels than normal in the body. Red blood cells contain haemoglobin which binds to oxygen and transports it around the body. In Sickle cell disease the red blood cells contain haemoglobin which is less able to bind to oxygen . As a result, the cells clump together and can block blood vessels, often causing pain and other symptoms.
In this study AES-103 or placebo (dummy drug) will be given to up to three groups of subjects. Some subjects will receive placebo throughout the entire study but all subjects will receive placebo on certain days. The first group will receive 1000mg four times a day and subsequent groups will receive a dose based on the results obtained from the first group.
Subjects will be recruited by Investigators at sites other than Quintiles who manage sickle cell patients. Eligible subjects will proceed to a 14 day outpatient run in period after screening, during which they will be required to take the study medication four times a day. On successful completion of the run in period they will be admitted to the clinical research unit at Quintiles for seven days of inpatient dosing. Post discharge they will self administer the study medication for a further 3 weeks. This will be followed by an observation period of 3 weeks during which no further study medication will be taken and subjects will visit the unit at specified times for study assessments.REC name
South Central - Oxford B Research Ethics Committee
REC reference
13/SC/0300
Date of REC Opinion
7 Aug 2013
REC opinion
Further Information Favourable Opinion