RECRUITINGPhase 2INTERVENTIONAL

Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease

A Phase 2 Randomized Double-blind, Placebo-controlled Clinical Trial of Intravenous Citrulline for Vaso-occlusive Pain Episode in Hospitalized Patients With Sickle Cell Disease (CONQUER SCD Pain Trial)

About This Trial

The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline. The main questions it aims to answer are: * Does intravenous citrulline decrease the duration of sickle cell pain during hospitalization * What medical problems do participants have when taking intravenous citrulline? Researchers will compare intravenous citrulline to a placebo (a look-alike substance that contains no drug) to see if intravenous citrulline works to treat acute pain. Participants will: * Receive baseline tests and intravenous citrulline for 16 hours during the hospital stay * After hospital discharge, visit the clinic in about 30 days for checkup and tests

Who May Be Eligible (Plain English)

Who May Qualify: - Sickle cell disease (all genotypes) - Patients with sickle cell disease ages 4 to 21 years old - Presence of sickle cell vaso-occlusive pain episode requiring hospitalization and parenteral opioid therapy - Able to randomize to study drug/placebo within 12 hours of first dose of parenteral opioid in the Emergency Department Who Should NOT Join This Trial: - Current pain lasting \>3 days. - \>9 hospitalizations in the prior year - Presence of any other complication related to sickle cell disease requiring the hospitalization such as splenic sequestration, hepatic sequestration, stroke, transient ischemic attack, etc. - History of opioid use disorder, chronic pain or medical regimen requiring daily opioid use. - Severe anemia (hemoglobin \<6g/dL) - Pregnant (as confirmed by a positive urine pregnancy test) or lactating female. - Alanine/aspartate transferase \>2x upper limit of normal laboratory range for age. - Subject has the following serum creatinine: - Age 4 to 13 years \> 0.9 mg/dL - Age 14 to 17 years 1.0 mg/dL - Age ≥18 years \>1.5mg/dL - Presence of acute chest syndrome, sepsis, bacterial infection, hemodynamic instability - Use of L-glutamine - History of allergic reaction to L-citrulline products Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Sickle cell disease (all genotypes) * Patients with sickle cell disease ages 4 to 21 years old * Presence of sickle cell vaso-occlusive pain episode requiring hospitalization and parenteral opioid therapy * Able to randomize to study drug/placebo within 12 hours of first dose of parenteral opioid in the Emergency Department Exclusion Criteria: * Current pain lasting \>3 days. * \>9 hospitalizations in the prior year * Presence of any other complication related to sickle cell disease requiring the hospitalization such as splenic sequestration, hepatic sequestration, stroke, transient ischemic attack, etc. * History of opioid use disorder, chronic pain or medical regimen requiring daily opioid use. * Severe anemia (hemoglobin \<6g/dL) * Pregnant (as confirmed by a positive urine pregnancy test) or lactating female. * Alanine/aspartate transferase \>2x upper limit of normal laboratory range for age. * Subject has the following serum creatinine: * Age 4 to 13 years \> 0.9 mg/dL * Age 14 to 17 years 1.0 mg/dL * Age ≥18 years \>1.5mg/dL * Presence of acute chest syndrome, sepsis, bacterial infection, hemodynamic instability * Use of L-glutamine * History of allergic reaction to L-citrulline products

Treatments Being Tested

DRUG

L-citrulline

Intravenous L-citrulline (50 mg/kg + 9mg/kg/hr.) for 16 hours

OTHER

Placebo

Isotonic normal saline

DRUG

L-citrulline

Intravenous L-citrulline (25 mg/kg + 9mg/kg/hr.) for 16 hours

Locations (1)

Children's National Hospital

Washington D.C., District of Columbia, United States