Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Axcella Health, Inc
Age
12–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
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
