Status:
COMPLETED
Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients
Lead Sponsor:
Emmaus Medical, Inc.
Conditions:
Sickle Cell Disease
Pharmacokinetics
Eligibility:
All Genders
5+ years
Phase:
PHASE4
Brief Summary
L-glutamine has been approved in the US to reduce the acute complications of sickle cell disease (SCD) in adult and pediatric patients 5 years of age and older. The purpose of this single-center, open...
Detailed Description
Sickle cell disease (SCD) is associated with a mutation in the β-hemoglobin gene that results in abnormal polymerization of hemoglobin. Polymerization of hemoglobin causes the red blood cell to sickle...
Eligibility Criteria
Inclusion
- 5 years of age and older at Screening.
- Has documented diagnosis of SCD with known genotype (HbSS, HbSβ0 and HbSC).
- Written informed consent provided by patient or the patient's legally authorized representative.
- Non-pregnant females of childbearing age must agree to avoid pregnancy during the study and to practice a recognized form of birth control during the course of the study (e.g., barrier, birth control pills, or abstinence).
- Inclusion Criteria for Healthy Volunteers:
- No known hematologic illness.
- No known renal impairment.
- 18 Years of age or older at screening.
- Written informed consent provided by patient or the patient's legally authorized representative.
- African American and Hispanic participants preferred.
Exclusion
- Recent significant medical condition that required hospitalization (other than sickle cell crisis) within 2 months prior to starting L-glutamine therapy.
- History of chronic kidney disease Stage 4 (glomerular filtration rate \[GFR\]=15-29) or Stage 5 (GFR\<15 mL/min/1.73 m2).
- History of chronic liver disease Child Pugh class C (10-15 points).
- Received any blood products 3 months prior to starting L-glutamine therapy.
- Currently pregnant or lactating or planning to conceive during the study period.
- Currently taking or has taken any form of glutamine supplement within 30 days prior to starting L-glutamine therapy.
- Has been treated with an investigational medication/treatment within 30 days prior to starting L-glutamine therapy.
- Is currently enrolled in an investigational drug or device study and/or has participated in such a study within 30 days prior to starting L-glutamine therapy.
- Factors that would, in the judgment of the investigator, make it difficult for the patient to comply with study requirements.
- Patient is currently being treated with crizanlizumab or voxelotor.
- Exclusion Criteria for Healthy Volunteers:
- Known allergies to L-glutamine.
- Informed consent document was not completed and signed.
- Currently pregnant or lactating or planning to conceive during the study period.
- Known hematologic illness, renal or hepatic impairment.
- Received any blood products within 3 months of starting L-glutamine therapy.
Key Trial Info
Start Date :
January 4 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2021
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT04684381
Start Date
January 4 2021
End Date
June 30 2021
Last Update
August 2 2022
Active Locations (1)
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1
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229