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

Sickle Cell Disease Clinical Trial

Official title:

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04134299
• Other study ID #: AXA4010-001
• Status: Completed
• Phase: N/A
• Start date: September 25, 2019
• Est. completion date: January 11, 2021

Study information

• Verified date: January 2021
• Source: Axcella Health, Inc
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: January 11, 2021
• Est. primary completion date: January 11, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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 ß0 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

Related Conditions & MeSH terms

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

Intervention

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.

Locations

Country	Name	City	State
United States	Primary Care research	Atlanta	Georgia
United States	Foundation for Sickle Cell Disease Research	Hollywood	Florida
United States	Advanced Pharma CR, LLC	Miami	Florida
United States	Newark Beth Israel Medical Center	Newark	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Axcella Health, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in Lactate Dehydrogenase (LDH)		Baseline to Week 12
Secondary	Change Indirect Bilirubin		Baseline to Week 12
Secondary	Change Absolute Reticulocyte Count		Baseline to Week 12
Secondary	Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)		Baseline to Week 12