HIBISCUS 2
Title of Protocol:
A global phase 3, randomized, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease.
Purpose/Objective of Study:
To confirm whether etavopivat works well at reducing the number of VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. This study will also evaluate how well etavopivat can reduce the damage to different organs, improve exercise tolerance and reduce fatigue in people with sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
Etavopivat is the investigational drug this clinical trial. Etavopivat may help treat sickle cell disease by acting to lower the rate of blood cell sickling.
Toxicities/Side Effects:
Possible side effects are headaches.
Time Committment: 104 weeks
FLORAL
Title of Protocol:
An open-label, multi-centre, rollover study to characterize long-term safety and efficacy of etavopivat in adults, adolescents and children who have sickle cell disease or thalassaemia and have completed a treatment period in an etavopivat study.
Purpose/Objective of Study:
This research study is looking at the long-term effects of etavopivat in people with sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
Etavopivat is the investigational drug for this clinical trial. Etavopivat may help treat sickle cell disease by acting to lower the rate of red blood cell sickling.
Toxicities/Side Effects:
Possible side effects are headaches.
Time Committment: Approximately 262 weeks
DS102A-10-RD2
Title of Protocol:
An open-label mechanistic study to assess the pharmacokinetics, pharmacodynamics, and safety of orally administered epeleuton in patients with sickle cell disease.
Purpose/Objective of Study:
This primary purpose of this study is to assess the efficacy and safety of Epeleuton in subjects with sickle cell disease.
Study Agent(s) and Mechanism of Drug Action/Device Description:
Etavopivat is the investigational drug for this clinical trial. Epeleuton may have a positive effect on the health of red blood cells and hemolytic anemia (when red blood cells are destroyed faster than they can be made), on blood vessel inflammation and organ damage.
Toxicities/Side Effects:
Possible side effects are diarrhea, bad taste in mouth, headache, nasal/sinus congestion, acne, abnormal blood and urine results, musculoskeletal pain and stiffness, throat pain, cough, abdominal pain, and respiratory infection.
Time Committment: Approximately 20 weeks