Status:
COMPLETED
To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease
Lead Sponsor:
Axcella Health, Inc
Conditions:
To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease
Anemia, Sickle Cell
Eligibility:
All Genders
12+ years
Phase:
NA
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 ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 25 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 11 2021
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT04134299
Start Date
September 25 2019
End Date
January 11 2021
Last Update
April 1 2021
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
Foundation for Sickle Cell Disease Research
Hollywood, Florida, United States, 33021
2
Advanced Pharma CR, LLC
Miami, Florida, United States, 33147
3
Primary Care research
Atlanta, Georgia, United States, 30312
4
Newark Beth Israel Medical Center
Newark, New Jersey, United States, 07112