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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04134299
Other study ID # AXA4010-001
Secondary ID
Status Completed
Phase N/A
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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