Novel opioid-sparing agents targeting SCD pain
| Name . | Mechanism . | Trial phase, type, NTC# . | Primary end points . | Status . | Results and comments . |
|---|---|---|---|---|---|
| Vaporized cannabis | Activation of cannabinoid receptors | 1, RDBPC cross over, NCT01771731 | Daily pain level over 5 d | Completed | All SCD genotypes; results pending |
| Canakinumab | IL-1β mAb | 2, RDBPC, NCT02961218 | Daily pain scores | Recruiting | Age 12-20 y |
| Buprenorphine | Partial μ agonist and κ antagonist | 2, open label, NCT03492099 | Hospitalization in 72 h subsequent to full analgesia rotation to buprenorphine | Active but not yet recruiting | All SCD genotypes |
| Ketamine* | NMDA receptor antagonist | 2, open label, NCT03296345 | Treatment-associated AEs | Recruiting | All SCD genotypes; age 8-25 y |
| Randomized triple blinded, NCT03431285 | Pain scores | Recruiting | All SCD genotypes | ||
| Randomized triple blinded, NCT02573714 | Pain scores | Recruiting | All SCD genotypes; age 4-16 y | ||
| Intranasal ketamine | |||||
| 3, randomized open label, NCT02801292 | Reduction in pain scores | Not yet recruiting | All SCD genotypes; age 3-17 y | ||
| Memantine | NMDA receptor antagonist | 2, single group, NCT02615847 | AEs | Completed | Results pending |
| 2a/2b, single arm, NCT03247218 | AEs | Not yet recruiting | ≥10 y | ||
| Gabapentin* | Binding to voltage-gated calcium channels in neurons | 2, RDBPC, NCT01954927 | Percentage of patients with ≥33% pain reduction in 3 h | Recruiting | All SCD genotypes; age 1-20 y |
| Topical sodium nitrite | NO | 2, RDBPC, NCT02863068 | Tolerability | Recruiting | All SCD genotypes |
| 1, single arm open label, NCT01316796 | Safety, tolerability, dose finding | Completed | All SCD genotypes; well tolerated with positive efficacy signals |
| Name . | Mechanism . | Trial phase, type, NTC# . | Primary end points . | Status . | Results and comments . |
|---|---|---|---|---|---|
| Vaporized cannabis | Activation of cannabinoid receptors | 1, RDBPC cross over, NCT01771731 | Daily pain level over 5 d | Completed | All SCD genotypes; results pending |
| Canakinumab | IL-1β mAb | 2, RDBPC, NCT02961218 | Daily pain scores | Recruiting | Age 12-20 y |
| Buprenorphine | Partial μ agonist and κ antagonist | 2, open label, NCT03492099 | Hospitalization in 72 h subsequent to full analgesia rotation to buprenorphine | Active but not yet recruiting | All SCD genotypes |
| Ketamine* | NMDA receptor antagonist | 2, open label, NCT03296345 | Treatment-associated AEs | Recruiting | All SCD genotypes; age 8-25 y |
| Randomized triple blinded, NCT03431285 | Pain scores | Recruiting | All SCD genotypes | ||
| Randomized triple blinded, NCT02573714 | Pain scores | Recruiting | All SCD genotypes; age 4-16 y | ||
| Intranasal ketamine | |||||
| 3, randomized open label, NCT02801292 | Reduction in pain scores | Not yet recruiting | All SCD genotypes; age 3-17 y | ||
| Memantine | NMDA receptor antagonist | 2, single group, NCT02615847 | AEs | Completed | Results pending |
| 2a/2b, single arm, NCT03247218 | AEs | Not yet recruiting | ≥10 y | ||
| Gabapentin* | Binding to voltage-gated calcium channels in neurons | 2, RDBPC, NCT01954927 | Percentage of patients with ≥33% pain reduction in 3 h | Recruiting | All SCD genotypes; age 1-20 y |
| Topical sodium nitrite | NO | 2, RDBPC, NCT02863068 | Tolerability | Recruiting | All SCD genotypes |
| 1, single arm open label, NCT01316796 | Safety, tolerability, dose finding | Completed | All SCD genotypes; well tolerated with positive efficacy signals |
Unless otherwise specified, study included only adult patients with HbSS or HbSβ0.
AE, adverse event; RDBPC, randomized double-blind placebo control.
Study drug administered during acute pain event