A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
4202-HEM-301: An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)
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Clinical Trial Information
Trial Contact: Spinelli, Jennifer; Parker, Melanie; Armatti, Julie M; Doyle, Katherine M; Dubberly, Paige D
Trial Phone: 321.841.5357 ; 321-843-1036 ; 321-843-5284 ; 3218412008 ; 321-841-7561
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IRB No: 22.034.02
Protocol Abbrev: 4202-HEM-301
Principal Investigator: Shveta Gupta, MD
Phase: Drug: Phase II
Age Group: Adult;Pediatric
Secondary Protocol No: 4202-HEM-301
Treatment: Drug: Etavopivat Tablets; Drug: Placebo Tablets
Therapies Involved: Medication
ClinicalTrials.gov ID: NCT04624659
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Objective
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).
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Key Eligibility
Ages Eligible for Study: 12 Years to 65 Years (Child, Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
- Patient has a confirmed diagnosis of sickle cell disease
- At least 2 episodes of vaso-occlusive crises in the past 12 months
- Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening
- Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
- Patients on crizanlizumab or L-glutamine treatment at the time of consent must be on a stable dose for ≥ 12 months and must be ≥ 80% compliant with the planned regimen at the time of consent and meet the VOC eligibility criteria
