L-Citrulline for Sickle Cell Disease
Trial Summary
What is the purpose of this trial?
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.
Research Team
Suvankar Majumdar, MD
Principal Investigator
Center for Cancer and Blood Disorders, Children's National Hospital
Eligibility Criteria
This trial is for children and young adults aged 6-21 with Sickle Cell Disease experiencing a pain episode known as vaso-occlusive crisis. They must be able to give consent or have a guardian who can, and should not be taking any medications that are disallowed in the study.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either L-citrulline or placebo intravenously, with a bolus dose followed by a continuous infusion for up to 12 hours
Follow-up
Participants are monitored for safety and effectiveness after treatment, with follow-up calls around Day 2, Day 7, and Day 30
Treatment Details
Interventions
- L-citrulline
Find a Clinic Near You
Who Is Running the Clinical Trial?
Asklepion Pharmaceuticals, LLC
Lead Sponsor