October, 10 2019

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.

Locations

4 United States sites

Age

> 12 Years

Genotypes

SS

Phase

N/A

About the study

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 Type

Interventional

Phase

N/A

Age

> 12 Years

Genotype

SS

Gender

All

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

Interventions

  • Dietary Supplement

Outcome measures

  • Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]

Location

  • Foundation for Sickle Cell Disease Research, Hollywood, Florida, United States, 33021 [Not yet recruiting]
  • Advanced Pharma CR, LLC, Miami, Florida, United States, 33147 [Recruiting]
  • Primary Care research, Atlanta, Georgia, United States, 30312 [Recruiting]
  • Newark Beth Israel Medical Center, Newark, New Jersey, United States, 07112 [Not yet recruiting]

Contact Research Team

  • Manu Chakravarthy, MD
    857-320-2200
  • Margaret Koziel, MD
    857-320-2200

More info

View on ClinicalTrials
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