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

Not Applicable

Completed

• Conditions: To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease; Sickle Cell Disorders; Sickle Cell Disease; Anemia, Sickle Cell; Sickle Cell Anemia
• Interventions: Dietary Supplement: AXA4010

• Registration Number: NCT04134299
• Lead Sponsor: Axcella Health, Inc

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-25
• Study First Submitted: 2019-10-18
• Study First Submitted QC: 2019-10-21
• Study First Posted: 2019-10-22
• Status Verified: 2021-01
• Primary Completion: 2021-01-11
• Study Completion: 2021-01-11
• Last Update Submitted: 2021-03-30
• Last Update Posted: 2021-04-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
AXA4010	AXA4010	AXA4010

Primary Outcome Measures

Name	Time	Method
Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]	Baseline to Week 12	Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)

Secondary Outcome Measures

Name	Time	Method
Change Absolute Reticulocyte Count	Baseline to Week 12
Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)	Baseline to Week 12
Change in Lactate Dehydrogenase (LDH)	Baseline to Week 12
Change Indirect Bilirubin	Baseline to Week 12

Trial Locations

Locations (4)

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