Arginine Therapy for Sickle Cell Disease Pain

Summary

The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency department visit. Funding Source - FDA OOPD.

Timeline

Start

2016-02

Primary completion

2021-02-21

Completion

2021-02-21

Outcome

Missed primary endpoint

paper This study did not meet its primary endpoint. PMID 40270092 ↗

Publications

• Background Bakshi N, Liu Z, Gillespie S, Keesari R, Leake D, Khemani K, Kumari P, Rees CA, Dampier C, Morris CR. Patient-reported outcomes in children with sickle cell disease at presentation for an acute pain episode. Blood Adv. 2023 Sep 12;7(17):5103-5107. doi: 10.1182/bloodadvances.2021006794. No abstract available.
• Results Reyes LZ, Figueroa J, Leake D, Khemani K, Kumari P, Bakshi N, Lane PA, Dampier C, Morris CR. Safety of intravenous arginine therapy in children with sickle cell disease hospitalized for vaso-occlusive pain: A randomized placebo-controlled trial in progress. Am J Hematol. 2022 Jan 1;97(1):E21-E24. doi: 10.1002/ajh.26396. Epub 2021 Nov 12. No abstract available.
• Morris CR, Hatabah D, Korman R, Gillespie S, Bakshi N, Brown LA, Harris F, Leake D, Rees CA, Khemani K, Vichinsky EP, Locke A, Wynn B, Griffiths MA, Wilkinson H, Kumari P, Sudmeier L, Shiva S, Dampier CD. Arginine Therapy for Pain in Sickle Cell Disease: A Phase-2 Randomized, Placebo-Controlled Trial. Am J Hematol. 2025 Jul;100(7):1119-1131. doi: 10.1002/ajh.27692. Epub 2025 Apr 24.
• Morris CR, Brown LAS, Reynolds M, Dampier CD, Lane PA, Watt A, Kumari P, Harris F, Manoranjithan S, Mendis RD, Figueroa J, Shiva S. Impact of arginine therapy on mitochondrial function in children with sickle cell disease during vaso-occlusive pain. Blood. 2020 Sep 17;136(12):1402-1406. doi: 10.1182/blood.2019003672.

Drugs

Indications