4470: NDec in patients with sickle cell disease
Research type
Research Study
Full title
A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease
IRAS ID
293212
Contact name
Moji Awogbade
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2020-003485-39
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 8 months, 1 days
Research summary
The primary purpose of this trial is to investigate two dosing regimens of oral decitabinetetrahydrouridine (NDec) in terms of treatment-related effects on total haemoglobin and HbF (foetal haemoglobin) as well as clinical efficacy and safety parameters compared with placebo in patients with sickle cell disease (SCD) who are not receiving hydroxyurea (HU) treatment at screening (HU-non-active patients). An active comparator HU treatment arm is included to allow exploratory comparisons between NDec and HU in patients receiving HU treatment at screening (HU-active patients). This is a multicentre, randomised, parallel-group, multiple-dose trial to assess the safety and efficacy of NDec in adult patients with SCD. There are two trial blocks: the HU-non-active block into which patients that are not receiving HU treatment at screening will be randomised to NDec once weekly, NDec twice weekly on two consecutive days or placebo, and the HU-active block into which patients that are on active HU-treatment at screening will be randomised to NDec once weekly, NDec twice weekly or continued HU treatment (open-label). Patients will be stratified by sex and historical VOC (vaso-occlusive crisis) rate (2−5 and 6−10 VOCs in the past 12 months). The trial consists of a screening phase, two treatment phases (main and extension) and a follow-up evaluation phase, giving a total of 56 weeks. In addition, there is a 4-week wash-out phase between randomisation and first dose for HU-active patients randomised to NDec once weekly or twice weekly on two consecutive days.
REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
21/EE/0059
Date of REC Opinion
4 May 2021
REC opinion
Further Information Favourable Opinion