Table 1.
| Trial Phase(s) | Drug Compounds (and Explanation) |
|---|---|
| Phase I RCT | Metformin, Aes-103, SCD-101 (NCT02380079), and NKTT120 (NCT01783691) |
| Phase I | Ambrisentan (NCT02712346) Decitabine + tetrahydrouridine or THU (NCT01685515) Plerixafor mobilization and apheresis (NCT03226691–multicenter study) Citrulline * (NCT02314689, NCT02697240) Zileuton (NCT01136941–SAOL) Panobinostat or LBH589 (NCT01245179) INCB059872 (NCT03132324) |
| Phase I/II RCT | Voxelotor or GBT440 (NCT02850406) |
| Phase I/II | Arginine (NCT02447874–open-label randomized crossover design), rivipansel or GMI-1070 (NCT00911495–SAOL, NCT01119833–RDBPC, and NCT02187003–RDBPC), omega-3 fatty acids (NCT02947100–SAOL), N-acetylcysteine (NCT01800526–SAOL), and Simvastatin (NCT0050802–SAOL, NCT01702246–SAOL) |
| Phase II RCT | Crizanlizumab, rivipansel, intravenous Ig, or IVIG (NCT01757418–RDBPC), dalteparin, sevuparin, eptifibatide, prasugrel, haptoglobin, oral or intravenous nitrite, inhaled nitric oxide, hemopexin/haptoglobin, MP4CO, various antioxidants, canakinumab, montelukast, and simvastatin. |
| Phase II | Atorvastatin (NCT01732718), Arginine (NCT01796678–RDBPC, NCT02536170–RDBPC, and NCT00004412–open-label randomized design), Mometasone (NCT02061202), Montelukast (NCT01960413), Omega-3 fatty acids (NCT02973360–RDBPC), AMD 3100 or Mozobil (plerixafor) (NCT00075335), Riociguat (NCT02633397–RDBPC), and IW-1701 (NCT03285178–RDBPC). |
| Phase III RCT | Arginine, senicapoc, tinzaparin, ticagrelor, rivipansel (GMI-1070), crizanlizumab (NCT03814746), and antioxidants. |
| Phase III | Glutamine (NCT01179217–RDBPC), Omega-3 fatty acids (NCT02525107, NCT02604368), and N-acetylcysteine (NCT01849016–RDBPC). |
| FDA-approved | l-glutamine, hydroxyurea, crizanlizumab, or SEG101 (NCT1895361–RDBPC, NCT03264989–SAOL, and NCT03474965–SAOL). |
| Under investigation | TLR4 inhibition, DNAse-1, anakinra, and vitamin D. |
* Study drug administered during acute VOC, SAOL: single-arm open-label, RDBPC: randomized double-blind placebo-controlled.