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.

Eligibility
Potential participants must meet the following eligibility criteria

• Age: 18-65 years old
• Diagnosis: HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants
• Patient must have had 2–15 episodes of documented pain crisis within the 12 months
• Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL
• For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
• For participants taking crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent, they must have been on a stable dose for ≥ 12 months at the time of consent (i.e., no changes to the dose except for changes to weight or for safety reasons)

Toxicities/Side Effects
Possible side effects are headaches

Time Commitment
104 weeks

Website link of sponsor
https://clinicaltrials.gov/study/NCT04624659

Research Team
PI: Julie Kanter, MD

FLORAL

Title of Protocol:

An open-label, multi-centre, rollover study to characterise 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 study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

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. 

Key Eligibility

Potential participants must meet the following eligibility criteria

• Participants must have ongoing participation in HIBISCUS or HIBISCUS 2 and have completed at least a treatment period.
• Participants must have gained clinical benefit from treatment with etavopivat, as determined by the investigator.
• Any participant with dose reduction or temporary discontinuation will need to be challenged before transferring.
• Participants on hydroxurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligibile if they:
  • Have been on a stable dose during participation in the parent study (i.e., no changes to the dose except for changes to weight or age reasons).
  • Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.

Toxicities/Side Effects
Possible side effects are headaches

Time Commitment
264 weeks

Website link of sponsor
NA

Research Team
PI: Julie Kanter, MD
Clinical Research Administrative Manager: Emily Warner
Site Coordinator: Ramona Colvin

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