Clinical trial NCT04134299: To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: 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
Keywords: Amino Acid
Food Study
Lead sponsor: Axcella Health, Inc
Sponsor class: INDUSTRY
First posted: 2019-10-22
Enrollment: 9
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: BASIC_SCIENCE

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]
timeFrame: Baseline to Week 12
Secondary outcomes:
measure: Change in Lactate Dehydrogenase (LDH)
timeFrame: Baseline to Week 12
measure: Change Indirect Bilirubin
timeFrame: Baseline to Week 12
measure: Change Absolute Reticulocyte Count
timeFrame: Baseline to Week 12
measure: Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)
timeFrame: Baseline to Week 12
Other outcomes:
Locations:
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Miami
country: United States
facility: Advanced Pharma CR, LLC
state: Florida
city: Atlanta
country: United States
facility: Primary Care research
state: Georgia
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
Study officials:
affiliation: Advanced Pharma CR, LLC
name: Kimberly Cruz, MD
role: PRINCIPAL_INVESTIGATOR
References: