NCT04134299

To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease

An Open-Label 12-Week Food Study to Assess the Safety, Tolerability, And Physiological Effects of An Amino Acid Food Product, AXA4010, In Subjects With Sickle Cell Disease.

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Axcella Health, Inc · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed.

Conditions

• To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease
• Anemia, Sickle Cell
• Sickle Cell Anemia
• Sickle Cell Disorders
• Sickle Cell Disease

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	AXA4010	The study will initiate with approximately eight (8) adult subjects (≥18 y/o) with SCD (Cohort 1). Each subject will receive AXA4010 twice a day (BID) for 12 weeks. Safety and tolerability will be monitored, and study procedures will be performed. Analysis of safety, tolerability and laboratory assessments at week 12 would be performed. Based on the 12-week observations from at least three subjects in Cohort 1, subsequent Cohorts 2 and 3 in Part II may be initiated.

Timeline

• Start date: 2019-09-25
• Primary completion: 2021-01-11
• Completion: 2021-01-11
• First posted: 2019-10-22
• Last updated: 2021-04-01

Locations

4 sites across 1 country: United States