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

Axcella Health

Status

Completed

Conditions

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

Sickle Cell Disorders

Sickle Cell Anemia

Sickle Cell Disease

Anemia, Sickle Cell

Treatments

Dietary Supplement: AXA4010

Study type

Interventional

Funder types

Industry

Identifiers

NCT04134299

AXA4010-001

Details and patient eligibility

About

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.

Enrollment

9 patients

Sex

All

Ages

12+ years old

Volunteers

No Healthy Volunteers

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