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12 Trials

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Trial + Data / Events	Status	Ph	N	Region	Interventions	Sponsor	Conditions	Primary compl	Study compl
Brown, Cynthia J
ELEVAATE, NCT05856331: Study of INBRX-101 Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema	Recruiting	2	90	Europe, US, RoW	INBRX-101, Zemaira	Inhibrx Biosciences, Inc	Alpha 1-Antitrypsin Deficiency, Emphysema	05/25	06/25
NCT04798014: HERO-2: Home-Reported Outcomes With CFTR Modulator Therapy	Completed	N/A	860	US		Indiana University, Folia Health, Cystic Fibrosis Foundation	Cystic Fibrosis	08/23	08/23
HOPeCF, NCT06296394: Health Outcomes of Parents With Cystic Fibrosis-Aim 2	Recruiting	N/A	146	US	Parenthood	University of Pittsburgh, Cystic Fibrosis Foundation, National Heart, Lung, and Blood Institute (NHLBI)	Cystic Fibrosis, Parenthood Status	02/30	02/31
Wozniak, Linda
HIBISCUS, NCT04624659: A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease	Recruiting	2/3	344	Europe, Canada, US, RoW	Etavopivat Tablets Low dose, FT-4202, Etavopivat Tablets High dose, Placebo Tablets, placebo, Etavopivat Tablets	Forma Therapeutics, Inc., Novo Nordisk A/S	Sickle Cell Disease	12/25	12/26
Bergmann, Shayla
HOPE Kids 2, NCT04218084 / 2017-000903-26: Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease	Active, not recruiting	3	236	Europe, US, RoW	Voxelotor, Placebo	Pfizer	Sickle Cell Disease	08/23	01/25
NCT02932618 / 2016-001477-33: A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)	Recruiting	3	34	Europe, US, RoW	von Willebrand factor (Recombinant), VONVENDI, rVWF, BAX111, BAX 111, Antihemophilic Factor (Recombinant), ADVATE, Recombinant Factor VIII, rFVIII	Baxalta now part of Shire, Takeda Development Center Americas, Inc.	Von Willebrand Disease	01/25	01/25
SPARTAN, NCT03938454: A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism	Completed	2	36	US	crizanlizumab, SEG101	Novartis Pharmaceuticals	Priapism	03/23	11/23
NCT04817670 / 2020-005072-34: Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease	Completed	2	25	Europe, US, RoW	VIT-2763 120 mg, Vamifeport, VIT-2763 360 mg, VIT-2763 240 mg, Placebo BID, Placebo TID	Vifor (International) Inc., Fortrea	Sickle Cell Disease	03/24	03/24
SOLACE-Kids, NCT03474965 / 2017-001747-12: Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients	Recruiting	2	119	Europe, Canada, US, RoW	Crizanlizumab, SEG101	Novartis Pharmaceuticals	Sickle Cell Disease (SCD)	01/25	01/25
NCT04724421: Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options	Available	N/A		US, RoW	Voxelotor, GBT440	Pfizer, Pfizer	Sickle Cell Disease
PROSPECT, NCT04930445: Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD	Recruiting	N/A	1000	US	Oxbryta® (voxelotor) 500mg Tablets, Voxelotor, Oxbryta®	Pfizer, Pfizer	Sickle Cell Disease	01/30	01/30
Milks, Kreighton M
SOLACE-Kids, NCT03474965 / 2017-001747-12: Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients	Recruiting	2	119	Europe, Canada, US, RoW	Crizanlizumab, SEG101	Novartis Pharmaceuticals	Sickle Cell Disease (SCD)	01/25	01/25