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

NCT04134299 | Completed

• 1 other identifier: AXA4010-001
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 4

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.

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

Outcome Measures

Primary Outcomes (1)

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

  Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)

  Baseline to Week 12

Secondary Outcomes (4)

• Change in Lactate Dehydrogenase (LDH)

  Baseline to Week 12

• Change Indirect Bilirubin

  Baseline to Week 12

• 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

Study Arms (1)

AXA4010

EXPERIMENTAL

AXA4010

Dietary Supplement: AXA4010

Interventions

AXA4010 DIETARY_SUPPLEMENT

The study will initiate with approximately eight (8) adult subjects (≥18 y/o) with SCD (Cohort 1). Each subject will receive AXA4010 twice a day (BID) for 12 weeks. Safety and tolerability will be monitored, and study procedures will be performed. Analysis of safety, tolerability and laboratory assessments at week 12 would be performed. Based on the 12-week observations from at least three subjects in Cohort 1, subsequent Cohorts 2 and 3 in Part II may be initiated.

AXA4010

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Axcella Health, Inc: lead

Study Sites (4)

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33021, United States

Location

Advanced Pharma CR, LLC

Miami, Florida, 33147, United States

Location

Primary Care research

Atlanta, Georgia, 30312, United States

Location

Newark Beth Israel Medical Center

Newark, New Jersey, 07112, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Kimberly Cruz, MD

  Advanced Pharma CR, LLC

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 2019
• First Posted: October 22, 2019
• Study Start: September 25, 2019
• Primary Completion: January 11, 2021
• Study Completion: January 11, 2021
• Last Updated: April 1, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)