Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04134299
Recruitment Status : Recruiting
First Posted : October 22, 2019
Last Update Posted : October 22, 2019
Sponsor:
Information provided by (Responsible Party):
Axcella Health, Inc

Tracking Information
First Submitted Date  ICMJE October 18, 2019
First Posted Date  ICMJE October 22, 2019
Last Update Posted Date October 22, 2019
Actual Study Start Date  ICMJE September 25, 2019
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 21, 2019)
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)
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: October 21, 2019)
  • Change in Lactate Dehydrogenase (LDH) [ Time Frame: Baseline to Week 12 ]
  • Change Indirect Bilirubin [ Time Frame: Baseline to Week 12 ]
  • Change Absolute Reticulocyte Count [ Time Frame: Baseline to Week 12 ]
  • Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only) [ Time Frame: Baseline to Week 12 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
Official Title  ICMJE 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.
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE
  • 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
Intervention  ICMJE Dietary Supplement: AXA4010
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.
Study Arms  ICMJE Experimental: AXA4010
AXA4010
Intervention: Dietary Supplement: AXA4010
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 21, 2019)
24
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2020
Estimated Primary Completion Date September 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Margaret Koziel, MD 857-320-2200 regulatory@axcella.com
Contact: Manu Chakravarthy, MD 857-320-2200 regulatory@axcella.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04134299
Other Study ID Numbers  ICMJE AXA4010-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Axcella Health, Inc
Study Sponsor  ICMJE Axcella Health, Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kimberly Cruz, MD Advanced Pharma CR, LLC
PRS Account Axcella Health, Inc
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP