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NCT Number: NCT04134299

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

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.

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Key information

Conditions

To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Sickle Cell Anemia Sickle Cell Disease Sickle Cell Disorders

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Foundation for Sickle Cell Disease Research, Hollywood, Florida, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Primary outcomes

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

    Time frame: Baseline to Week 12

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

Secondary outcomes

  1. Change in Lactate Dehydrogenase (LDH)

    Time frame: Baseline to Week 12

  2. Change Indirect Bilirubin

    Time frame: Baseline to Week 12

  3. Change Absolute Reticulocyte Count

    Time frame: Baseline to Week 12

  4. Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)

    Time frame: Baseline to Week 12

Sponsors and collaborators

Lead sponsor

Axcella Health, Inc

Industry

Registry information

Official study 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.

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Oct 22, 2019
Registry last updated
Apr 1, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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