Phase 2
N=108
Arginine Therapy for Sickle Cell Disease Pain
Sickle Cell Disease · Vaso-occlusive Pain Episode
Bottom Line
View on ClinicalTrials.gov: NCT02536170 ↗Enrolled (actual)
108
Serious AEs
17.6%
Results posted
Jun 2022
Primary outcome: Primary: Total Parenteral Opioid Use in IV Morphine Equivalents — 1.77; 1.95; 2.36 mg/Kg
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- L-arginine (Drug); L-arginine Loading Dose (Drug); Placebo (Other)
- Age
- Pediatric, Adult · 3+ yrs
- Sex
- All
- Sponsor
- Emory University
- Primary completion
- Feb 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Parenteral Opioid Use in IV Morphine Equivalents |
1.77; 1.95; 2.36 | — |
| SECONDARY Length of Hospital Stay |
71.3; 76.9; 84.9 | — |
| SECONDARY Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department |
NA; NA; NA | — |
| SECONDARY Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital |
57.3; 55.1; 69.3 | — |
| SECONDARY Change in Vaso-occlusive Pain (VOE) Scores |
8.89; 8.86; 8.86; 4.5; 4.75; 4.56 | — |
| SECONDARY Length of Emergency Department (ED) Stay |
6.29; 5.67; 4.08 | — |
| SECONDARY Rate of Emergency Department (ED) Discharge |
0; 0; 0 | — |
| SECONDARY Total Opioid Dose (ORAL + Parenteral) in mg/kg IV Morphine Equivalents |
2.11; 2.35; 2.82 | — |
| SECONDARY Total Number of Study Drug Doses |
298; 330; 329 | — |
| SECONDARY Rate of Acute Chest Syndrome |
4; 3; 1 | — |
| SECONDARY Rate of Blood Transfusion |
3; 2; 5 | — |
| SECONDARY Oxygen Saturation Level |
95.5; 98; 98.1; 98; 98.6; 98.4 | — |
| SECONDARY Oxygen Saturation Level |
95.5; 98; 98.1; 98; 98.6; 98.4 | — |
| SECONDARY Rate of Return Visits to Emergency Department (ED) Within 72 Hours |
0; 0; 0 | — |
| SECONDARY Rate of Hospital Re-admissions Within 72 Hours |
2; 2; 2 | — |
| SECONDARY Rate of Return Visits to Emergency Department (ED) Within 30 Days |
0; 1; 0 | — |
| SECONDARY Rate of Hospital Re-admissions With 30 Days |
2; 7; 4 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Established diagnosis of sickle cell disease (SCD); all genotypes
- Pain requiring medical care in an acute care setting (such as the emergency department or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, that is moderate-to-severe requiring parenteral opioids
Exclusion Criteria
- Decision to discharge home from the acute care setting
- Hemoglobin less than 5 gm/dL or immediate need for red cell transfusion anticipated within next 12 hours
- Hepatic dysfunction of SGPT greater than 3 times the upper value
- Renal dysfunction of creatinine greater than 1.0
- Mental status or neurological changes
- Acute stroke or clinical concern for stroke
- Pregnancy
- Allergy to arginine
- Two (2) or more ED visits for VOE within the last 7 days prior to CURRENT ED visit
- Hospitalization within 14 days
- Previous randomization in this arginine RCT (patient consented and screen failed before receiving study drug or placebo remains eligible for future participation).
- Use of inhaled nitric oxide, sildenafil or arginine within the last month
- PICU admission from the emergency department
- Hypotension requiring treatment with clinical intervention
- Acidosis with Co2≤ 16
- Newly started on HU for <3 months
- Not an appropriate candidate in the investigator's judgment
- Patient refusal
Data sourced from ClinicalTrials.gov (NCT02536170). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.