Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

Conditions

• Sickle Cell Disease

Eligibility Criteria

Sex

ALL

Age

7 Years - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Arginine — DRUG
  Arginine will be dispensed intravenously (in the vein) in the standard dose of arginine as 100 mg/kg three times a day for seven days or until discharge. * Loading dose: 200 mg/kg once * Continuous IV: 300 mg/kg/24 hours
• Arginine (Loading) — DRUG
  Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) at each specified group dose once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.
• Arginine (Continuous) — DRUG
  Arginine will be dispensed intravenously (in the vein) as a continuous IV infusion of 300 mg/kg/24hr

Study Details

The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

Key Dates

Start date

May 31, 2015

Status verified

Jul 2025

Primary completion

Jul 31, 2026

Completion

Jul 31, 2026

Study Design

Enrollment

21 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Experimental: Standard dose
  Subjects with sickle cell disease (SCD) and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first
• Experimental: Loading dose + standard dose
  Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first
• Experimental: Loading dose + continuous infusion
  Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive a continuous intravenous (IV) infusion of 300 mg/kg/24hr for 7 days or until discharged from the hospital, whichever occurs first
• Experimental: Non-Randomized Loading dose 500 mg/kg + standard dose
  Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 500 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.
• Experimental: Non-Randomized Loading dose 300 mg/kg + standard dose
  Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 300 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.
• Experimental: Non-Randomized Loading dose 400mg/kg + standard dose
  Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 400 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first.

Primary Outcome Measure

Pharmacokinetics of IV arginine, measured by plasma arginine concentration over time [ Time Frame: Day 1 through study completion, an average of up to 7 days ]

Central Contacts

• Reshika Mendis, MBBS
  404-785-4525
  [email protected]
• Claudia Morris, MD
  404 727-5500
  [email protected]

Locations (2)

Find similar trials in Atlanta, GA

Related Studies

• Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
  PHASE1 · Recruiting · Abdullah Kutlar · Augusta, Georgia
• ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
  Recruiting · American Thrombosis and Hemostasis Network · Phoenix, Arizona
• Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
  Enrolling By Invitation · Genetix Biotherapeutics Inc. · Birmingham, Alabama
• Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study
  PHASE4 · Recruiting · Novartis Pharmaceuticals · Birmingham, Alabama