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Etavopivat

Phase 1

Healthy Volunteers | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Jun 18, 2026

Target and mechanism

Molecular targetPKLR
Target classActivator
ModalitySmall molecule
ChEMBLCHEMBL4650332

Also known as Etavopivat Tablets Low dose, Etavopivat Low dose

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment57

FDA Designations

No designations recorded

Clinical trial landscape

Etavopivat · 5 trials · 5 indications

Phase 1 5
NCT07023029A Study on the Effect of Etavopivat on Heart Rhythm in Healthy ParticipantsHealthy Volunteers
COMPLETED33 Analytics
NCT06813924A Research Study of the Effect of Etavopivat on Other Drugs in Healthy ParticipantsHealthy Volunteers Sickle Cell Disease, Thalassemia
COMPLETED37 Analytics
NCT06581627A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese ParticipantsHealthy Volunteers
COMPLETED24 Analytics
NCT06433661A Research Study of the Effect of Food on Etavopivat in Healthy ParticipantsHealthy Volunteers Sickle Cell Disease, Thalassemia
COMPLETED16 Analytics
NCT06336018A Research Study on Etavopivat in Participants With and Without Liver DiseaseLiver Diseases
COMPLETED48 Analytics
PHASE1COMPLETED
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
Healthy Volunteers
NCT0702302933
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PHASE1COMPLETED
A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants
Healthy Volunteers Sickle Cell Disease, Thalassemia
NCT0681392437
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PHASE1COMPLETED
A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants
Healthy Volunteers
NCT0658162724
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PHASE1COMPLETED
A Research Study of the Effect of Food on Etavopivat in Healthy Participants
Healthy Volunteers Sickle Cell Disease, Thalassemia
NCT0643366116
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PHASE1COMPLETED
A Research Study on Etavopivat in Participants With and Without Liver Disease
Liver Diseases
NCT0633601848
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Study Endpoints

Primary Endpoints

Part A: Number of treatment-emergent adverse events (AEs)
From dosing (Day 1) until end of study (Day 8)

Measured as count of events.

Part B: Change from baseline in Fridericia heart rate corrected QT interval (ΔQTcF) for etavopivat
From pre-dose to post etavopivat/ etavopivat placebo dose on day 1 to day 23

Measured as milliseconds.

Cmax, digoxin, SD: Maximum observed digoxin plasma concentration with and without etavopivat at steady state
Day 1 and day 3 after digoxin administration

Measured as picograms per milliliter (pg/mL).

AUC0-inf, digoxin, SD: Area under the digoxin plasma concentration-time curve from 0 hours and extrapolated to infinity with and without etavopivat at steady state
Day 1 and day 3 after digoxin administration

Measured as hours\*picograms per milliliter (h\*pg/mL).

Cmax, rosuvastatin, SD: Maximum observed rosuvastatin plasma concentration with and without etavopivat at steady state
Day 1 after rosuvastatin administration

Measured as pg/mL.

AUC0-inf, rosuvastatin, SD: Area under the rosuvastatin plasma concentration-time curve from 0 hours and extrapolated to infinity with and without etavopivat at steady state
Day 1 after rosuvastatin administration

Measured as h\*pg/mL.

Cmax, midazolam, SD: Maximum observed midazolam plasma concentration without etavopivat, with a single dose of etavopivat, and with etavopivat at steady state
Day 1 after midazolam administration

Measured as pg/mL.

AUC0-inf, midazolam, SD: Area under the midazolam plasma concentration-time curve from 0 hours and extrapolated to infinity without etavopivat, with a single dose of etavopivat, and with etavopivat at steady state
Day 1 after midazolam administration

Measured as h\*pg/mL.

Cmax, pitavastatin, SD: Maximum observed pitavastatin plasma concentration with and without etavopivat at steady state
Day 1 and day 3 after pitavastatin administration

Measured as nanograms per milliliter (ng/mL).

AUC0-inf, pitavastatin, SD: Area under the pitavastatin plasma concentration-time curve from 0 hours and extrapolated to infinity with and without etavopivat at steady state
Day 1 and day 3 after pitavastatin administration

Measured as hours\*nanograms per milliliter(h\*ng/mL).

Cmax, metformin, SD: Maximum observed metformin plasma concentration with and without etavopivat at steady state
Day 1 and day 3 after metformin administration

Measured as ng/mL.

AUC0-inf, metformin, SD: Area under the metformin plasma concentration-time curve from 0 hours and extrapolated to infinity with and without etavopivat at steady state
Day 1 and day 3 after metformin administration

Measured as h\*ng/mL.

AUC0-inf,etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in hour\*nanogram/millilitre (h\*ng/mL).

Cmax,etavopivat: Maximum observed etavopivat plasma concentration after a single dose
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in nanograms/millilitre (ng/mL).

Weight-adjusted AUC0-inf,etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose divided by body weight
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in(hour\*nanogram/millilitre)/kilograms ((h\*ng/mL)/kg).

Weight-adjusted Cmax,etavopivat: Maximum observed etavopivat plasma concentration after a single dose divided by body weight
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in (nanograms/millilitre )/kilogram ((ng/mL)/kg).

AUC0-last,etavopivat: Area under the etavopivat plasma concentration-time curve from0 hours to the time of last quantifiable concentration
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in hour\*nanogram/millilitre (h\*ng/mL).

tmax,etavopivat: Time to maximum observed etavopivat plasma concentration after a single dose
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in hour (h).

t½,etavopivat: Terminal half-life for etavopivat after a single dose
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in hour (h).

CL/F etavopivat: Apparent plasma clearance of etavopivat after a single dose
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measured in Litre/hour (L/h).

Vz/F etavopivat: Apparent volume of distribution of etavopivat after a single dose based on plasma concentration values
From 0 to 120 hours after investigational medicinal product (IMP) administration (day 6)

Measure in litre (L).

AUC0-inf, etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose
From 0 to 120 hours after IMP administration (V2/V6)

Measured as hours nanograms per milliliter (h\*ng/mL).

Cmax, etavopivat: Maximum observed etavopivat plasma concentration after a single dose
From 0 to 120 hours after IMP administration (V2/V6)

Measured as nanograms per milliliter (ng/mL).

Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose (AUC0-inf, etavopivat)
From IMP administration on day 1 to completion of the end of study visit (day 9)

Measured in hours\*nanogram per milliter (h\*ng/mL).

Maximum observed etavopivat plasma concentration after a single dose (Cmax, etavopivat)
From IMP administration on day 1 to completion of the end of study visit (day 9)

Measured in nanogram per milliter (ng/mL).

Secondary Endpoints

Part A: Number of treatment-emergent clinically significant abnormal findings in electrocardiogram (ECGs)
From dosing (Day 1) until end of study (Day 8)
Parts A and B: Cmax,etavopivat- Maximum observed etavopivat plasma concentration after a single dose
Day 1 (Part B)/ From day 1 to day 5 (Part A) after investigational medicinal product (IMP) administration
Parts A and B: AUC0-last,etavopivat- Area under the etavopivat plasma concentration-time curve from 0 hours to the time of last quantifiable concentration
Day 1 (Part B)/ From day 1 to day 5 (Part A) after IMP administration
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelFACTORIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A- EtavopivatEXPERIMENTALParticipants will receive a single dose of etavopivat.
Part A- PlaceboPLACEBO_COMPARATORParticipants will receive a single dose of placebo.
Part B- EtavopivatEXPERIMENTALParticipants will receive a single dose of etavopivat.
Part B- MoxifloxacinOTHERParticipants will receive a single dose of moxifloxacin.
Part B- PlaceboPLACEBO_COMPARATORParticipants will receive a single dose of placebo.
Period 1: digoxin+pitavastatin+metforminEXPERIMENTALParticipants will receive a single dose of oral digoxin, pitavastatin and metformin.
Period 1: midazolam+rosuvastatinEXPERIMENTALParticipants will receive a single dose of oral midazolam and rosuvastatin.
Period 2: etavopivat+midazolamEXPERIMENTALParticipants will receive a daily dose of oral etavopivat and single dose of oral midazolam.
Period 2: etavopivat+midazolam+rosuvastatinEXPERIMENTALParticipants will receive a daily dose of oral etavopivat, single dose of oral midazolam and rosuvastatin.
Period 2: etavopivat+digoxin+pitavastatin+metforminEXPERIMENTALParticipants will receive a daily dose of oral etavopivat, single dose of oral digoxin, pitavastatin and metformin.
EtavopivatEXPERIMENTALParticipants will be administered a single dose of 2 tablets of etavopivat together.
Sequence 1: Etavopivat: fed-fastedEXPERIMENTALParticipants will receive a single dose of Etavopivat in fed condition in period 1 and a single dose of Etavopivat in fasted condition in period 2.
Sequence 2: Etavopivat: fasted-fedEXPERIMENTALParticipants will receive a single dose of Etavopivat in fasted condition in period 1 and a single dose of Etavopivat in fed condition in period 2.
Mild hepatic impairment: Etavopivat dose 2EXPERIMENTALParticipants will receive a single dose of oral etavopivat.
Moderate hepatic impairment: Etavopivat dose 2EXPERIMENTALParticipants will receive a single dose of oral etavopivat.
Healthy matched controls: Etavopivat dose 2EXPERIMENTALParticipants will receive a single dose of oral etavopivat.
Severe hepatic impairment: Etavopivat dose 1EXPERIMENTALParticipants will receive a single dose of oral etavopivat.
Healthy matched controls: Etavopivat dose 1EXPERIMENTALParticipants will receive a single dose of oral etavopivat.

Interventions

NameTypeDescription
EtavopivatDRUGEtavopivat will be administered orally.
MoxifloxacinDRUGMoxifloxacin will be administered orally.
PlaceboDRUGPlacebo matching Etavopivat will be administered orally.
DigoxinDRUGParticipants will receive a single dose of digoxin orally.
PitavastatinDRUGParticipants will receive a single dose of pitavastatin orally.
MetforminDRUGParticipants will receive a single dose of metformin orally.
MidazolamDRUGParticipants will receive a single dose of midazolam orally.
RosuvastatinDRUGParticipants will receive a single dose of rosuvastatin orally.
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Eligibility Criteria

Age Range18 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Male or female. * Aged 18-55 years (both inclusive) at the time of signing informed consent. * Body mass index (BMI) between 18.0 and 32.0 kilograms per square meter (kg/m\^2) (both inclusive) at screening. * Body weight above 40.0 kg at screening. * Considered to be generally...

Countries:United StatesChina
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Recent Changes (Last 90 Days)

MEDIUMJul 19, 2026NCT06336018TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT06336018TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT06336018TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT06336018TRIAL_REMOVED: changed

Frequently asked questions about Etavopivat

What is Etavopivat used for?

Etavopivat is an investigational small molecule being developed for hematology conditions, including sickle cell disease, thalassemia, and liver diseases. It is also studied in healthy volunteers. The drug is not approved and remains in clinical development, with trials spanning adults, adolescents, and pediatric patients.

Who makes Etavopivat?

Etavopivat is being developed by Novo Nordisk A/S, a biopharmaceutical company traded on the New York Stock Exchange under the ticker NVO. The company is conducting multiple clinical trials to evaluate the drug's safety and efficacy in sickle cell disease and related conditions.

What phase is Etavopivat in?

Etavopivat is in Phase 1 through Phase 3 clinical development. The most advanced trial, NCT04624659, is a Phase 3 study in sickle cell disease that is active but not recruiting. Other trials are in Phase 2, and the drug remains investigational and not FDA approved.

What clinical trials is Etavopivat in?

Etavopivat is being studied in several trials, including NCT04624659, a Phase 3 study in sickle cell disease with 450 participants; NCT04987489, a completed Phase 2 trial in thalassemia or sickle cell disease; NCT05725902, a completed Phase 2 cerebral hemodynamic study; and NCT06198712, a recruiting Phase 2 pediatric pharmacokinetic study.

Is Etavopivat the same as Etavopivat Tablets Low dose?

Yes, Etavopivat is also known as Etavopivat Tablets Low dose and Etavopivat Low dose. These names refer to the same investigational drug, which is being developed by Novo Nordisk for sickle cell disease and other hematology conditions.

How does Etavopivat work?

Etavopivat is a small molecule, but its specific molecular target has not been disclosed in the available information. The drug is being evaluated for its effects on sickle cell disease and thalassemia, with trials measuring clinical outcomes and pharmacokinetics in affected patient populations.

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