Table 2. Ongoing clinical trials of anti-adhesion agents and antioxidants in sickle cell disease.
| Mechanism | Drug | Sponsor | NCT number
(study acronym) |
Clinical phase/status | Intervention | Number/age | Objective |
|---|---|---|---|---|---|---|---|
| P-selectin
antagonist |
Crizanlizumab | Novartis
Pharmaceuticals |
NCT03264989
(SOLACE- adults) |
Phase II
Active, not recruiting |
IV infusion, open-label | 57/16–70 years | Pharmacokinetics,
pharmacodynamics, safety, and efficacy |
|
NCT03814746
(STAND) |
Phase III
Recruiting |
IV infusion every 2 weeks for
1 st month and then monthly for 1 year |
240/≥12 years | Compare efficacy and safety of 5
mg/kg and 7.5 mg/kg doses with placebo |
|||
|
NCT04053764
(STEADFAST) |
Phase II
Recruiting |
IV infusion every 2 weeks for
1 st month and then every 4 weeks for 51 weeks + SoC vs. SoC alone |
170/≥16 years | Evaluating effect on kidney
function (albumin-creatinine ratio, protein-creatinine ratio, estimated glomerular filtration rate) |
|||
|
NCT03938454
(SPARTAN) |
Phase II
Recruiting |
IV infusion every 2 weeks for
1 st month and then every 4 weeks x 51 weeks |
56/≥16 years | Evaluate efficacy in priapism,
uncomplicated VOC events |
|||
| NCT03474965 | Phase II
Recruiting |
IV infusion every 2 weeks for
1 st month and then every 4 weeks |
100/6 months–<18
years |
Evaluate pharmacokinetics,
pharmacodynamics, safety, and effect on VOC events |
|||
| Blockade of fcγrIII
receptors |
IVIG | Albert Einstein
College of Medicine |
NCT01757418 | Phase I–II
Recruiting |
Single dose of IVIG vs.
placebo given within 24 hours of hospitalization |
94/12–65 years | Length of VOC, total opioid
use, time to end of VOC, in vitro adhesion studies |
| Antioxidant
(increased glutathione) |
NAC | Bloodworks | NCT01800526 | Phase I/II
Enrolling by invitation |
IV or oral, NAC
Part 1: two doses of IV infusion over 8 hours 4 weeks apart or oral NAC for 4 weeks Part 2 (during VOC): IV infusion every 6 hours for 5 days |
20/≥18 years | Evaluate effect on vWF activity,
measures of hemolysis and oxidation In Part 2, evaluate efficacy during VOC |
IV, intravenous; IVIG, intravenous gammaglobulin; NAC, N-acetylcysteine; SoC, standard of care; VOC, vaso-occlusive crisis; vWF activity, von Willebrand factor activity.