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

NCT04134299 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2021-04-01

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Axcella Health, Inc

    lead INDUSTRY

Principal Investigators

  • Kimberly Cruz, MD · Advanced Pharma CR, LLC

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-25
Primary Completion
2021-01-11
Completion
2021-01-11

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04134299 on ClinicalTrials.gov