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

Launched by ASKLEPION PHARMACEUTICALS, LLC · May 16, 2025

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment for children and young adults aged 6 to 21 who have sickle cell disease (SCD) and are experiencing painful episodes called vaso-occlusive crises (VOCs). The goal is to see if a medication called L-citrulline, given through an IV, can help reduce pain during these emergencies. About 120 participants will be enrolled in the study, and they will be randomly assigned to receive either L-citrulline or a placebo (which looks the same but has no active ingredients). Importantly, all participants will still receive their usual pain management care in the hospital.

To be eligible for this study, participants must have sickle cell disease and be experiencing a significant amount of pain. They should not have any other serious health issues or complications from sickle cell disease at the time of joining. After receiving treatment, patients can go home once their pain is under control and they have a plan for managing pain at home. The study team will follow up with participants a few days and weeks after treatment to see how they are doing. If you're considering this trial for yourself or a loved one, the medical team will provide detailed information and answer any questions during the consent process.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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