Table 5:
Ongoing Clinical Trials of Nitric Oxide-Related Drugs and Vasodilators in Sickle Cell Disease
| Drug | Study Title | Mechanism of Action | Clinical Phase | NCT Number | Sponsor |
|---|---|---|---|---|---|
| Ambrisentan | The Role of Endothelin-1 in Sickle Cell Disease |
Vasodilator (endothelin receptor A antagonist) |
Phase 1 | NCT02712346 | Augusta University; Gilead Sciences; NHLBI |
| Citrulline | A Phase 1 Study of Continuous Intravenous L-citrulline During Sickle Cell Pain Crisis or Acute Chest Syndrome |
Increased nitric oxide synthesis |
Phase 1 | NCT02697240 | University of Mississippi Medical Center |
| Riociguat | A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases |
Vasodilator (soluble guanylate cyclase stimulator) |
Phase 2 | NCT02633397 | University of Pittsburgh |
| Sodium Nitrite | Topical Sodium Nitrite in Sickle Cell Disease and Leg Ulcers |
Vasodilator (local nitric oxide donor) |
Phase 2 | NCT02863068 | Montefiore Medical Center; Food and Drug Administration |
| Arginine | Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease (R34 pK/PD); Arginine Therapy for Sickle Cell Disease Pain |
Increased nitric oxide synthesis |
Phase 1/2; Phase 2 |
NCT02447874; NCT02536170 |
Emory University |
| Macitentan | Macitentan in Pulmonary Hypertension of Sickle Cell Disease (MENSCH) |
Vasodilator (endothelin receptor antagonist) |
Phase 1 | NCT02651272 | Boston University |
| IMR-687 | A Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia) |
Phosphodiesterase-9 inhibitor |
Phase 2 | NCT03401112 | Imara, Inc |
| Losarían | Preventing Sickle Cell Kidney Disease | Vasodilator (angiotensin receptor blocker) |
Phase 2 | NCT02373241 | University of Alabama at Birmingham |