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

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

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.

Sponsors & Collaborators

• SAIC-Frederick, Inc.

  collaborator INDUSTRY

• Therapeutics for Rare and Neglected Diseases (TRND)

  collaborator NIH

• QS Pharma

  collaborator UNKNOWN

• National Chung Cheng University

  collaborator OTHER

• Infrared Imaging and Thermometry Unit, Biomedical Engineering and Physical Science Shared Resource (NIBIB)

  collaborator UNKNOWN

• ClinPharm Consulting, LLC

  collaborator UNKNOWN

• Ricerca Biosciences LLC

  collaborator UNKNOWN

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Cato Research

  collaborator INDUSTRY

• Baxalta now part of Shire

  lead INDUSTRY

Principal Investigators

• Study Director · Takeda

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

65 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-05-12

Primary Completion

2013-06-07

Completion

2013-06-07

Countries

• United States

Study Locations