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. Author manuscript; available in PMC: 2019 May 14.
Published in final edited form as: Expert Opin Orphan Drugs. 2018 May 14;6(5):329–343. doi: 10.1080/21678707.2018.1471983

Table 1:

Ongoing Clinical Trials of Anti-Sickling Agents in Sickle Cell Disease

Drug Study Title (Sponsor) Mechanism of Action Clinical Phase NCT Number Sponsor
Sanguinate A Study of SANGUINATE for the
Treatment of Vaso-occlusive Crisis (VOC)
in Adult Sickle Cell Disease Patients		 Oxygen transfer agent Phase 2 NCT02672540 Prolong
Pharmaceuticals
Voxelotor Study to Evaluate the Effect of GBT440
Administered Orally to Patients With
Sickle Cell Disease (GBT_HOPE)
(GBT_HOPE)		 Increase hemoglobin
oxygen affinity (left shift
of oxyhemoglobin
dissociation curve)		 NCT03036813 Global Blood
Therapeutics
Panobinostat Study of Panobinostat (LBH589) in
Patients With Sickle Cell Disease
(LBH589)		 Increase hemoglobin F
(histone deacetylase
inhibitor)		 Phase 1 NCT01245179 Novartis
INCB059872 A Study to Evaluate Safety,
Pharmacokinetic, and Biological Activity of
INCB059872 in Subjects With Sickle Cell
Disease		 Increase hemoglobin F
by inhibition of lysine-
specific demethylase 1
(LSD1)		 Phase 1 NCT03132324 Incyte
Corporation
Decitabine and
Tetrahydrouridine		 Study of Decitabine and Tetrahydrouridine
(THU) in Patients With Sickle Cell Disease		 Increase hemoglobin F
(hypomethylating agent)		 Phase 1 NCT01685515 The Cleveland
Clinic
Metformin Fetal Hemoglobin Induction Treatment
Metformin (FITMet)		 Increase hemoglobin F Phase 1 NCT02981329 Baylor College
of Medicine