Status:
TERMINATED
L-citrulline Injection in Patients Aged 6-21 Years Old with Sickle Cell Disease Presenting with Vaso-Occlusive Crisis (VOC)
Lead Sponsor:
Asklepion Pharmaceuticals, LLC
Conditions:
Acute Vaso Occlusive Crisis (VOC)
Eligibility:
All Genders
6-21 years
Phase:
PHASE1
PHASE2
Brief Summary
The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opia...
Detailed Description
Overall Design: This is a study in children, adolescents, and young adults (6 to 21 years) with SCD presenting to an emergency department (ED) with VOC. Children below age 6 are likely to be recruite...
Eligibility Criteria
Inclusion
- Sickle cell disease (all genotypes)
- Children, adolescents and young adults between ages 6 to 21 years
- In a steady disease state and not in the midst of any acute complication other than VOC due to sickle cell disease at study entry
- For female of childbearing potential, a negative urine pregnancy test and using an adequate method of contraception, or denies sexual activity
- Subjects or parents or legal guardian of the subject who are willing and able to sign and provide consent and assent (where appropriate for the age of the child).
Exclusion
- Current pain lasting \>3 days
- \>6 hospital admissions in the prior year
- History of opioid dependence/substance abuse
- Has been on a clinical trial of a new therapy for sickle cell disease within the last 3 months
- Presence of any other complication related to sickle cell disease such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, renal dysfunction, dactylitis, acute chest syndrome and other major medical conditions or organ dysfunction
- Severe anemia (hemoglobin \<6 g/dL)
- History of red blood cell transfusion within the last 30 days
- Systemic steroid therapy within the last 48 hours
- Pregnancy or lactation (subjects must have a negative urine pregnancy test)
- Serum creatinine levels:
- Age 6 to 13 years \>0.9 mg/dL
- Age 14 to 17 years \>1.0 mg/dL
- Age \>18 years \>1.5 mg/dL
- Report of fever (\>38°C) within last 48 hours
- Presence of acute chest syndrome, sepsis, bacterial infection, hemodynamic instability
- Subjects with inability to have parental assent given (ages 6 to 17 years) or consent (ages 18 through 21 years).
- Note: Parents or legal guardians can provide consent for subjects who are unable to provide assent (eg, sleepy or preoccupied by their pain).
- History of allergic reaction to L-citrulline product
- Medications that are known to be contra-indicated with use of L-citrulline
- History of diabetes.
- Subjects with a baseline prothrombin time International Normalized ratio (INR) \>2.0.
- Received any blood products within 3 weeks of the screening visit.
- Unreliable venous access
- The PI considers that the subject will be unable to comply with the study requirements.
Key Trial Info
Start Date :
April 29 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 29 2024
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT04852172
Start Date
April 29 2021
End Date
February 29 2024
Last Update
September 19 2024
Active Locations (2)
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1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
2
The University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216