To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
Completed · Not applicable
Conditions studied: 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
In brief
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.
Key facts
- Study ID
- NCT04134299
- Run by
- Axcella Health, Inc
- People needed
- 9
- Starts
- 2019-09-25
- Expected to finish
- 2021-01-11
- Last updated by the study team
- 2021-04-01
Who can join
Age: 12 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Foundation for Sickle Cell Disease Research — Hollywood, Florida, United States
- Advanced Pharma CR, LLC — Miami, Florida, United States
- Primary Care research — Atlanta, Georgia, United States
- Newark Beth Israel Medical Center — Newark, New Jersey, United States
Full record on ClinicalTrials.gov
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