Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
Purpose
The trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) is designed to further knowledge on efficacy and safety of the therapy.
Condition
- Sickle Cell Disease
Eligibility
- Eligible Ages
- Between 3 Years and 21 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Age 3-21 years of age, inclusive; AND 2. Established diagnosis of sickle cell disease (any genotype); AND 3. Pain requiring medical care in an acute care setting (ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, treated with parenteral opioids.
Exclusion Criteria
- Responds to 2 doses of IV opioids sufficiently for outpatient management 2. Greater than 12 hours from first dose of intravenous opioids to treat current pain in acute care setting 3. Hemoglobin less than 5 gm/dL or emergent need for red blood cell transfusion for hemodynamically unstable patient; OR 4. Ketamine use in the emergency department for treatment of VOE; OR 5. Glutamine within 30 days; OR 6. New SCD drug use < 3 months (e.g. Hydroxyurea, voxelotor, crizanlizumab, etc) OR 7. Acute mental status or neurological changes; OR 8. Acute stroke or clinical concern for stroke; OR 9. Three or more ED visits for sickle cell related pain receiving parenteral opioids in previous 7 days (not including current ED visit); OR 10. Hospital discharge within previous 7 days; OR 11. Hypotension requiring clinical intervention; hemodynamic instability; septic shock; OR 12. Previous randomization in this arginine phase 3 RCT; OR 13. Use of inhaled nitric oxide, sildenafil or arginine within the last month; OR 14. Non-English or non-Spanish speaking; OR 15. pregnancy; OR 16. Allergy to arginine; OR 17. PI/clinical team concerns for compliance/issues that may adversely impact study participation/outcome; OR 18. Adults 18 years or older who lack medical decision-making capacity to consent
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Arginine Hydrochloride |
Arginine is a nutritional supplement in parenteral form |
|
|
Placebo Comparator Placebo |
Normal saline |
|
Recruiting Locations
Children's National and nearby locations
Washington, District of Columbia 20010
More Details
- NCT ID
- NCT04839354
- Status
- Recruiting
- Sponsor
- Claudia R. Morris
Detailed Description
The trial is designed as a double-blind, placebo controlled, randomized, phase 3, multi-center trial of IV arginine therapy in children with Vaso-occlusive painful episodes (VOE) in sickle cell disease (SCD) to further knowledge on efficacy and safety of the therapy. The exploratory objective is to more fully characterize the arginine metabolome in children with SCD during VOE, and evaluate the effects of arginine therapy on global arginine bioavailability and mitochondrial function together with important clinical outcomes (time to VOE resolution, pain scores, total parenteral opioid use, Patient-Reported Outcomes (PROs), and hospital length of stay in children with SCD and VOE.