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

Primary Purpose

To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

AXA4010

About this trial

This is an interventional basic science trial for To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease focused on measuring Amino Acid, Food Study

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Foundation for Sickle Cell Disease Research
Advanced Pharma CR, LLC
Primary Care research
Newark Beth Israel Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

AXA4010

Arm Description

AXA4010

Outcomes

Primary Outcome Measures

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

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

Secondary Outcome Measures

Change in Lactate Dehydrogenase (LDH)

Change Indirect Bilirubin

Change Absolute Reticulocyte Count

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

Full Information

NCT ID

NCT04134299

First Posted

October 18, 2019

Last Updated

March 30, 2021

Sponsor

Axcella Health, Inc

1. Study Identification

Unique Protocol Identification Number

NCT04134299

Brief Title

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

2. Study Status

Record Verification Date

January 2021

Overall Recruitment Status

Completed

Study Start Date

September 25, 2019 (Actual)

Primary Completion Date

January 11, 2021 (Actual)

Study Completion Date

January 11, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Axcella Health, Inc

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Basic Science

Study Phase

Not Applicable

Interventional Study Model

Sequential Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

AXA4010

Arm Type

Experimental

Arm Description

AXA4010

Intervention Type

Dietary Supplement

Intervention Name(s)

AXA4010

Intervention Description

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 Outcome Measure Information:

Title

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

Description

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

Time Frame

Baseline to Week 12

Secondary Outcome Measure Information:

Title

Change in Lactate Dehydrogenase (LDH)

Time Frame

Baseline to Week 12

Title

Change Indirect Bilirubin

Time Frame

Baseline to Week 12

Title

Change Absolute Reticulocyte Count

Time Frame

Baseline to Week 12

Title

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

Time Frame

Baseline to Week 12

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Kimberly Cruz, MD

Organizational Affiliation

Advanced Pharma CR, LLC

Official's Role

Principal Investigator

Facility Information:

Facility Name

Foundation for Sickle Cell Disease Research

City

Hollywood

State/Province

Florida

ZIP/Postal Code

33021

Country

United States

Facility Name

Advanced Pharma CR, LLC

City

Miami

State/Province

Florida

ZIP/Postal Code

33147

Country

United States

Facility Name

Primary Care research

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30312

Country

United States

Facility Name

Newark Beth Israel Medical Center

City

Newark

State/Province

New Jersey

ZIP/Postal Code

07112

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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