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NDec- decitabine-tetrahydrouridine

Phase 2

Sickle Cell Disease | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Jul 22, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment96

FDA Designations

No designations recorded

Clinical trial landscape

NDec- decitabine-tetrahydrouridine · 1 trial · 1 indication

Phase 2 1
NCT05405114Research Study Investigating How Well NDec Works in People With Sickle Cell DiseaseSickle Cell Disease
COMPLETED96 Analytics
PHASE2COMPLETED
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
Sickle Cell DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Change in total haemoglobin
From baseline (week 0) to week 24

measured in g/dL

Secondary Endpoints

Cmax for decitabine from pharmacokinetic assessment
At week 24
Cmax for tetrahydrouridine from pharmacokinetic assessment
At week 24
Change in DNA methyltransferase 1 (DNMT1) activity
From baseline (week 0) to week 24
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
HU-non-eligible - NDec plus placeboEXPERIMENTALHU-non eligible patients randomised to treatment with NDec on one day and placebo on the other day
HU-non-eligible - NDec plus NDecEXPERIMENTALHU-non eligible patients randomised to treatment with NDec on both days
HU-non-eligible - Placebo plus placeboPLACEBO_COMPARATORHU-non eligible patients randomised to treatment with placebo on both days
HU-active - NDec plus placeboEXPERIMENTALHU-active patients randomised to treatment with NDec on one day and placebo on the other day
HU-active - NDec plus NDecEXPERIMENTALHU-active patients randomised to treatment with NDec on both days
HU-active - HUACTIVE_COMPARATORHU-active patients randomised to continue on open-label HU treatment

Interventions

NameTypeDescription
NDec - oral decitabine-tetrahydrouridineDRUGParticipants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight
HU - HydroxyureaDRUGParticipants will get capsules daily (oral administration) according to local labelling
PlaceboDRUGParticipants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites118

Inclusion Criteria: * Age above or equal to 18 years at the time of signing informed consent * Confirmed diagnosis of SCD (including HbSS, HbSC, HbSβ0 thalassaemia and HbSβ+ thalassaemia or other Sickle Cell disease variants) * 2-10 episodes of documented vaso-occlusive crisis (VOCs) within the las...

Countries:United StatesCanadaFranceGreeceIndiaItalyJordanLebanonOmanSouth AfricaSpainSwitzerlandTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 22, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMAug 22, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMAug 22, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMAug 22, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT05405114TRIAL_REMOVED: changed
MEDIUMJul 19, 2026NCT05405114TRIAL_REMOVED: changed

Frequently asked questions about NDec- decitabine-tetrahydrouridine

What is NDec (decitabine-tetrahydrouridine) used for?

NDec (decitabine-tetrahydrouridine) is an investigational small molecule being studied for the treatment of sickle cell disease. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities. The drug is being evaluated in adult patients with sickle cell disease.

Who is developing NDec (decitabine-tetrahydrouridine)?

NDec (decitabine-tetrahydrouridine) is being developed by Novo Nordisk A/S, a pharmaceutical company traded on the stock exchange under the ticker symbol NVO. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with sickle cell disease.

What phase is NDec (decitabine-tetrahydrouridine) in?

NDec (decitabine-tetrahydrouridine) is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities for commercial use. The drug is being studied for the treatment of sickle cell disease in adult patients.

What clinical trials is NDec (decitabine-tetrahydrouridine) in?

NDec (decitabine-tetrahydrouridine) has one completed Phase 2 clinical trial registered under the identifier NCT05405114. This study investigated how well the drug works in people with sickle cell disease and enrolled 96 participants across multiple countries, including the United States, Canada, and several European and Middle Eastern nations.

Is NDec (decitabine-tetrahydrouridine) the same as decitabine?

NDec is a combination of decitabine and tetrahydrouridine. Decitabine is a chemotherapy agent, while tetrahydrouridine is an enzyme inhibitor that may enhance the activity of decitabine. This combination is being studied specifically for sickle cell disease, whereas decitabine alone is used for other conditions.