Not Yet RecruitingPhase 3

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

United States120 participantsStarted 2025-07-25

Plain-language 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 study will include about 120 participants between the ages of 6 and 21 who come to the emergency department (ED) with a VOC. A VOC is a painful episode that happens with no clear cause and no signs of infection or major problems with organs like the liver or kidneys. Before joining the study, patients and their families may be asked to learn about it and give permission (called consent or assent) while at a regular clinic visit. If that hasn't happened yet, the consent/assent process will happen at the emergency department when the patient comes in for care. If the patient meets all the study requirements, they can join the treatment part of the study. Participants will be randomly assigned (like flipping a coin) to receive either: L-citrulline, the study drug, or A placebo, which looks the same but has no active ingredients. Everyone has an equal chance of getting either one. The study drug is given through an IV. It starts with one larger dose, followed by a steady infusion for up to 12 hours. All patients in the study will still receive the usual pain treatment (called standard of care), which may include opioids. However, some patients may need fewer opioids if the study treatment helps with their pain. If any medicines are not allowed during the study, the doctor will explain this during the consent process. Patients can go home once: Their pain is controlled with oral (by mouth) pain medicine, They're eating and drinking well, and They've been given a personal pain management plan to use at home. After leaving the hospital, the study team will follow up with patients by phone about 2 days later (within a 12-hour window), again around Day 7, and again around Day 30 to check how they're doing.

Who can participate

Age range6 Years – 21 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.,…

What they're measuring

Change in pain score from baseline to discharge

Timeframe: From enrollment to hospital discharge (typically within 12 to 72 hours), up to max of 30 day study duration

Time to pain crisis resolution

Timeframe: From start of study drug infusion to discontinuation of IV opioids (within the index hospitalization), up to max of 30 day study duration

Trial details

NCT IDNCT06989567

SponsorAsklepion Pharmaceuticals, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-06

Contact for this trial

Gurdyal Kalsi, MD, MFPM (Hon)

+1 410-736-3750 gurdyal.kalsi@asklepionpharm.com

View on ClinicalTrials.gov More Sickle Cell Disease trials