To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell DiseaseNCT04134299
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
A study testing AXA4010 in people with To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease and Anemia, Sickle Cell, run by Axcella Health, Inc at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease, Anemia, Sickle Cell, Sickle Cell Anemia
- What is being tested?
- AXA4010
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 4 sitesUnited States
- Study dates
- 25 Sep 2019to 11 Jan 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Primary Care research
Atlanta, Georgia, 30312
Foundation for Sickle Cell Disease Research
Hollywood, Florida, 33021
Advanced Pharma CR, LLC
Miami, Florida, 33147
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
About this study
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.
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
- Being tested
- AXA4010
- Keywords
- Amino Acid, Food Study
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AXA4010Dietary 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.
| Study group | Role | What they receive |
|---|---|---|
| AXA4010 | Experimental | AXA4010 Receives: Dietary Supplement: AXA4010 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ]
Incidence of study product emergent adverse events (AEs) and serious adverse events (SAEs)
Measured: Baseline to Week 12
Secondary outcomes (4)
Change in Lactate Dehydrogenase (LDH)
Measured: Baseline to Week 12
Change Indirect Bilirubin
Measured: Baseline to Week 12
Change Absolute Reticulocyte Count
Measured: Baseline to Week 12
Changes in Renal and Cerebral blood flow via MRI (in Adult Cohort 2 Only)
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2019
- Study start25 Sep 2019
- Primary completion11 Jan 2021
- Study completion11 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Axcella Health, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kimberly Cruz, MD, Principal investigator, Advanced Pharma CR, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04134299
- Sponsor's protocol ID
- AXA4010-001
- Status last verified
- Jan 2021
- First submitted
- 18 Oct 2019
- Last updated
- 1 Apr 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT04134299
- Acronym / sponsor ID
- AXA4010-001
- 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
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Axcella Health, IncIndustry (pharma, biotech, devices)
- Study start
- 25 Sep 2019 (actual)
- Primary completion
- 11 Jan 2021 (actual)
- Study completion
- 11 Jan 2021 (actual)
- First posted
- 22 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AXA4010 | Experimental | Dietary Supplement: AXA4010AXA4010 |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
- Able to ambulate (without assistance or walkers etc.) in a 6- minute walk test
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AXA4010The 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. | Dietary supplement | AXA4010 |
Primary outcome
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)
4 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2021.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kimberly Cruz, MD principal investigator
Advanced Pharma CR, LLC
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing AXA4010 in people with To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease and Anemia, Sickle Cell, run by Axcella Health, Inc at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Study Product-Emergent Adverse Events [Safety and Tolerability ] (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2021.