NDec for Sickle Cell Disease
(ASCENT1 Trial)
Trial Summary
What is the purpose of this trial?
This trial tests a new medicine called NDec for people with sickle cell disease. NDec combines two drugs to help manage the disease. Participants will take capsules regularly over several months to see how well the medicine works and if it is safe.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop all current medications, but if you are taking Hydroxyurea (HU), you may continue it during the study. However, you cannot participate if you have taken voxelotor, crizanlizumab, or L-glutamine within 12 weeks before the trial or plan to take them during the trial.
What data supports the effectiveness of the drug NDec for treating sickle cell disease?
Research shows that decitabine, a component of NDec, can increase fetal hemoglobin levels in patients with sickle cell anemia, which helps reduce symptoms. In a study, patients who did not respond to another treatment (hydroxyurea) saw an increase in fetal hemoglobin and overall hemoglobin levels without significant side effects.12345
What makes the drug NDec unique for treating sickle cell disease?
NDec is unique because it combines decitabine with tetrahydrouridine to improve the oral availability of decitabine, which is usually broken down quickly in the body. This combination allows for a more effective and prolonged exposure to decitabine, potentially increasing fetal hemoglobin levels, which can be beneficial for sickle cell disease.678910
Research Team
Clinical Transparency (dept. 2834)
Principal Investigator
Novo Nordisk A/S
Eligibility Criteria
Adults over 18 with sickle cell disease (SCD), having had 2-10 pain episodes in the last year, and specific blood criteria can join. Excluded are those on chronic transfusions, recent other treatments for SCD, certain blood counts outside normal ranges, pregnant or breastfeeding women, and those not using effective contraception.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive NDec, NDec and placebo, or placebo. Participants on HU may continue HU or receive NDec treatments.
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- NDec
Find a Clinic Near You
Who Is Running the Clinical Trial?
Novo Nordisk A/S
Lead Sponsor
Lars Fruergaard Jørgensen
Novo Nordisk A/S
Chief Executive Officer since 2017
MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark
Martin Holst Lange
Novo Nordisk A/S
Chief Medical Officer since 2021
MD from University of Copenhagen