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Etavopivat A

Phase 3

Sickle Cell Disease | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Jun 10, 2026

Success Probability
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Market & Valuation
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Trial Design
CONTROLLED
Total Trials1
Total Enrollment480
FDA Designations
No designations recorded
Clinical trial landscape

Etavopivat A · 1 trial · 2 indications

Phase 3 1
NCT06609226A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaSickle Cell Disease
RECRUITING480 Analytics
PHASE3RECRUITING
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Sickle Cell Disease
NCT06609226480
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Study Endpoints
Primary Endpoints
Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Measured as number of events.

Number of adverse reactions, reported for each indication and age group separately
Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Measured as number of adverse reactions.

Secondary Endpoints
Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately
Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Change in VOCs, reported for each age group separately
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
Change in hemoglobin (Hb) concentration, reported for each age group separately
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Participants greater than or equal to (≥) 12 years old with sickle cell diseaseEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with sickle cell disease transfusion dependentEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with transfusion-dependent thalassaemiaEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with non-transfusion dependent thalassaemiaEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 2 years to less than (<) 12 years old with sickle cell diseaseEXPERIMENTALParticipants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B.
Interventions
NameTypeDescription
Etavopivat ADRUGParticipants will receive an oral dose of Etavopivat A.
Etavopivat BDRUGParticipants will receive an oral dose of Etavopivat B.
Etavopivat CDRUGParticipants will receive an oral dose of Etavopivat C.
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Eligibility Criteria
Age Range2 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites105

Inclusion Criteria: * Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study. * Participant must have derived clinical benefit from treatment with etavopi...

Countries:United StatesCanadaEgyptFranceGermanyGhanaGreeceIndiaItalyKenyaLebanonNigeriaOmanSaudi ArabiaSpainTurkey (Türkiye)United Kingdom
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Competitive Landscape -Sickle Cell Disease 27 trials
CompanyTickerTrialsLead PhaseDrugs
Novo Nordisk A/S Sponsored ADR Class BNVO5PHASE3Etavopivat Low dose
Novartis AG Sponsored ADRNVS4PHASE3Crizanlizumab
Sanofi SA Sponsored ADRSNY2PHASE3PCV21, 20vPCV
Vertex Pharmaceuticals IncorporatedVRTX3PHASE3CTX001
Agios Pharmaceuticals, Inc.AGIO2PHASE2Mitapivat
Pfizer Inc.PFE1PHASE2Osivelotor
Bristol-Myers Squibb CompanyBMY1PHASE1BMS-986470, Famotidine, Pantoprazole
Fulcrum Therapeutics, Inc.FULC1PHASE2Pociredir
Beam Therapeutics, Inc.BEAM2PHASE1BEAM-101
Editas Medicine, Inc.EDIT2PHASE1EDIT-301
Disc Medicine, Inc.IRON1PHASE1DISC-3405
Illumina, Inc.ILMN1 - Undisclosed
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Recent Changes (Last 90 Days)
LOWJun 10, 2026NCT06609226lastUpdatePostDate: changed
LOWJun 10, 2026NCT06609226lastUpdatePostDate: changed
LOWMay 26, 2026NCT06609226primaryCompletionDate: changed
LOWMay 24, 2026NCT06609226studyFirstPostDate: changed