Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial.

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Date

2023-08

Authors

Rees, Chris A
Brousseau, David C
Cohen, Daniel M
Villella, Anthony
Dampier, Carlton
Brown, Kathleen
Campbell, Andrew
Chumpitazi, Corrie E
Airewele, Gladstone
Chang, Todd

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Abstract

Background

Despite substantial illness burden and healthcare utilization conferred by pain from vaso-occlusive episodes (VOE) in children with sickle cell disease (SCD), disease-modifying therapies to effectively treat SCD-VOE are lacking. The aim of the Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial is to provide definitive evidence regarding the efficacy of intravenous arginine as a treatment for acute SCD-VOE among children, adolescents, and young adults.

Methods

STArT is a double-blind, placebo-controlled, randomized, phase 3, multicenter trial of intravenous arginine therapy in 360 children, adolescents, and young adults who present with SCD-VOE. The STArT Trial is being conducted at 10 sites in the USA through the Pediatric Emergency Care Applied Research Network (PECARN). Enrollment began in 2021 and will continue for 5 years. Within 12 h of receiving their first dose of intravenous opioids, enrolled participants are randomized 1:1 to receive either (1) a one-time loading dose of L-arginine (200 mg/kg with a maximum of 20 g) administered intravenously followed by a standard dose of 100 mg/kg (maximum 10 g) three times a day or (2) a one-time placebo loading dose of normal saline followed by normal saline three times per day at equivalent volumes and duration as the study drug. Participants, research staff, and investigators are blinded to the participant's randomization. All clinical care is provided in accordance with the institution-specific standard of care for SCD-VOE based on the 2014 National Heart, Lung, and Blood Institute guidelines. The primary outcome is time to SCD-VOE pain crisis resolution, defined as the time (in hours) from study drug delivery to the last dose of parenteral opioid delivery. Secondary outcomes include total parental opioid use and patient-reported outcomes. In addition, the trial will characterize alterations in the arginine metabolome and mitochondrial function in children with SCD-VOE.

Discussion

Building on the foundation of established relationships between emergency medicine providers and hematologists in a multicenter research network to ensure adequate participant accrual, the STArT Trial will provide definitive information about the efficacy of intravenous arginine for the treatment of SCD-VOE for children.

Trial registration

The STArT Trial was registered in ClinicalTrials.gov on April 9, 2021, and enrollment began on June 21, 2021 (NCT04839354).

Type

Journal article

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Subjects

SCD Arginine Study Group and PECARN, Humans, Anemia, Sickle Cell, Arginine, Analgesics, Opioid, Adolescent, Child, Academies and Institutes, Multicenter Studies as Topic, Randomized Controlled Trials as Topic, Clinical Trials, Phase III as Topic, Young Adult, Saline Solution

Citation

Published Version (Please cite this version)

10.1186/s13063-023-07538-z

Publication Info

Rees, Chris A, David C Brousseau, Daniel M Cohen, Anthony Villella, Carlton Dampier, Kathleen Brown, Andrew Campbell, Corrie E Chumpitazi, et al. (2023). Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial. Trials, 24(1). p. 538. 10.1186/s13063-023-07538-z Retrieved from https://hdl.handle.net/10161/30146.

This is constructed from limited available data and may be imprecise. To cite this article, please review & use the official citation provided by the journal.

Scholars@Duke

Chumpitazi

Corrie Chumpitazi

Instructor Temporary In the Department of Pediatrics

I’m a pediatric emergency medicine specialist committed to providing high-quality emergency care to ill and injured children. My clinical care focuses on providing innovative pain management and procedural sedation options, while ensuring that all children have equitable access to care. I am passionate about developing effective pain management options for chronic conditions that present acutely such as sickle cell disease pain crises. I strive to harness every opportunity to teach emergent pediatric management to all levels of learners. Outside of work, I enjoy exploring all the activities the area has to offer, from lake sports to hiking, and being the biggest fan at sporting events, ballroom dance competitions, and the concerts of my husband and three children.


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