NCT01597401

A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease

A Phase 1, Placebo-Controlled, Randomized, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating, Single Oral Doses of Aes-103 in Subjects With Stable Sickle Cell Disease

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Baxalta now part of Shire · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural \[5-HMF\]) compared with placebo in subjects with stable sickle cell disease (SCD). Safety will be measured by monitoring adverse events (AEs), electrocardiograms (ECGs), vital signs, and laboratory values. Pharmacokinetics of Aes-103 will be measured over time in plasma, red blood cell hemolysate and binding of Aes-103 to hemoglobin. Pharmacodynamic effects will be assessed by measuring partial pressure of oxygen at which 50% of hemoglobin is saturated with oxygen (p50) while breathing normal air, blood oxygen levels (SpO2), ex-vivo antisickling effects in a hypoxic environment, and by imaging related changes in tissue blood flow and oxygen levels.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Aes-103	300 mg Aes-103 powder reconstituted in orange juice to a volume of 100 mL per single dose for oral administration.
DRUG	Aes-103	1000 mg Aes-103 powder reconstituted in orange juice to a volume of 100 mL per single dose for oral administration.
DRUG	Aes-103	2000 mg Aes-103 powder reconstituted in orange juice to a volume of 100 mL per single dose for oral administration.
DRUG	Aes-103	4000 mg Aes-103 powder reconstituted in orange juice to a volume of 100 mL per single dose for oral administration.
DRUG	Placebo	Orange juice vehicle, a solution that is highly similar in appearance to the Aes-103 orange juice solution.

Timeline

• Start date: 2012-05-12
• Primary completion: 2013-06-07
• Completion: 2013-06-07
• First posted: 2012-05-14
• Last updated: 2021-05-05

Locations

1 site across 1 country: United States