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.

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.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease; Anemia, Sickle Cell; Sickle Cell Anemia; To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease; Sickle Cell Disorders
• Intervention / Treatment: Dietary supplement: AXA4010

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Florida
    • Hollywood, Florida, United States, 33021
      • Foundation for Sickle Cell Disease Research
    • Miami, Florida, United States, 33147
      • Advanced Pharma CR, LLC
  • Georgia
    • Atlanta, Georgia, United States, 30312
      • Primary Care Research
  • New Jersey
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Willing to participate in the study and provide written informed consent
• Male and female adolescent (ages 12 to 17 inclusive) and adult (≥ 18 years of age) subjects with a prior medically documented diagnosis of sickle cell disease (SS or β0 thalassemia)
• For Cohort 2 adult subjects only, a Screening cerebral blood flow/ velocity by transcranial doppler (TCD) should be >110 cm/sec

• Subjects must show active hemolysis at the time of screening based on each of the following parameters:

  • Lactate dehydrogenase (LDH) ≥ 2x above upper limits of normal (ULN) of the age- and gender-appropriate ranges
  • Indirect bilirubin > 2x of the age- and gender-appropriate ULN
  • Absolute reticulocyte counts > 2x of the age- and gender-appropriate ULN
• Able to ambulate (without assistance or walkers etc.) in a 6- minute walk test

Exclusion Criteria:

• Ten (10) or more Vascular-Occlusive Crisis (VOC)s that required hospital or emergency room (ER) visits within the past 12 months
• Any clinically significant changes abnormalities on the screening 12-lead ECG
• Estimated glomerular filtration rate <60mL/min/1.73m2 appropriately corrected
• Hemoglobin (Hb) ≤6.0 g/dL at Screening
• Subjects on chronic transfusion regimens, or who received a transfusion within the last 2 months prior to screening
• Experienced VOC or any other sickle crises (eg acute chest syndrome, splenic sequestration, dactylitis, stroke) within the last 2 months prior to Screening
• Current or history of significant alcohol consumption
• Other poorly controlled medical conditions as judged by the Investigator
• Known sensitivity and/or history of clinically significant food intolerance/allergies to proteins (including whey, soy, casein, amino acids, etc.)
• Unable or unwilling to adhere to contraception requirements
• Any contraindications to an MRI scan for Cohort 2 only
• Any other condition that, in the opinion of the Investigator, renders the subject at risk for compliance, compromises the well-being of the subject, or hinders study completion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: AXA4010

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]	Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)	Baseline to Week 12

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in Lactate Dehydrogenase (LDH)	Baseline to Week 12
Change Indirect Bilirubin	Baseline to Week 12
Change Absolute Reticulocyte Count	Baseline to Week 12
Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)	Baseline to Week 12

Collaborators and Investigators

• Sponsor: Axcella Health, Inc
• Investigators: Principal Investigator: Kimberly Cruz, MD, Advanced Pharma CR, LLC

Study record dates

Study Major Dates

• Study Start (Actual): September 25, 2019
• Primary Completion (Actual): January 11, 2021
• Study Completion (Actual): January 11, 2021

Study Registration Dates

• First Submitted: October 18, 2019
• First Submitted That Met QC Criteria: October 21, 2019
• First Posted (Actual): October 22, 2019

Study Record Updates

• Last Update Posted (Actual): April 1, 2021
• Last Update Submitted That Met QC Criteria: March 30, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Keywords: Amino Acid; Food Study
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia; Anemia, Sickle Cell
• Other Study ID Numbers: AXA4010-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No