Status:
NOT_YET_RECRUITING
A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis
Lead Sponsor:
Asklepion Pharmaceuticals, LLC
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
6-21 years
Phase:
PHASE3
Brief Summary
This study is being done to learn more about a possible new treatment for pain episodes (called vaso-occlusive crises or VOCs) in children, teens, and young adults with sickle cell disease (SCD). The ...
Detailed Description
What is Sickle Cell Disease (SCD)? Sickle cell disease is a genetic blood condition that affects red blood cells. People with SCD have a different kind of hemoglobin (called hemoglobin S) that can mak...
Eligibility Criteria
Inclusion
- SCD (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 SCD at study entry
- Baseline NRS score \>5 or Faces Pain Scale score ≥6
- For females of childbearing potential, a negative urine pregnancy test and using an adequate method of contraception including abstinence
- Patients or parents or legal guardian of the patient who are willing and able to sign and provide consent and assent (where appropriate for the age of the child)
- Patients willing to begin study treatment within 120 minutes after providing informed consent/assent and soon after the first/initial dose, but before any subsequent doses of IV opioid or ketamine
Exclusion
- Current pain lasting \>3 days
- History of 9 hospital admissions in the prior year
- Participation in a clinical trial of a new therapy for SCD within the last 1 month
- Presence of any other complication related to SCD such as splenic sequestration, hepatic sequestration, stroke, avascular necrosis of the hip/shoulder, acute priapism, acute renal dysfunction, acute chest syndrome and other major medical conditions or organ dysfunction
- Hypotension requiring clinical intervention; hemodynamic instability; septic shock
- Severe anemia (hemoglobin \<6 g/dL)
- Systemic steroid therapy within the last 24 hours
- Use of inhaled NO or medications that are known to cause hypotension (e.g., nitrates, sildenafil, tadalafil, vardenafil, osildenafil, or arginine) within the last 30 days
- Serum creatinine levels:
- Age 6-13 years: \>0.9 mg/dL
- Age 14-17 years: \>1.0 mg/dL
- Age ≥18 years: \>1.5 mg/dL
- Report of fever (\>38°C) within the last 24 hours
- Presence of acute chest syndrome, sepsis, known bacterial infection, or hemodynamic instability
- Acute mental status or neurological changes
- Acute stroke or clinical concern for stroke
- Patients with inability to have assent given (ages 6 to 17 years) or consent (ages 18 through 21 years).
- Note: Parents or legal guardians can provide consent for patients who are unable to provide assent (e.g., sleepy or preoccupied by their pain).
- History of allergic reaction to L-citrulline product or dextrose
- Unreliable venous access
- The PI considers that the patient will be unable to comply with the study requirements
- Patients pre-planned for admission on arrival in ED
Key Trial Info
Start Date :
July 25 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2029
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT06989567
Start Date
July 25 2025
End Date
June 1 2029
Last Update
May 29 2025
Active Locations (6)
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1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
2
University of Miami
Coral Gables, Florida, United States, 33146
3
Emory University
Atlanta, Georgia, United States, 30322
4
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287