Authorised Phase II and Phase III (Integrated) sickle cell disease (SCD)

This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients, age 12 to 65 years, with sickle cell disease (SCD).

EU CTIS ID: 2024-511535-97-00

What this study is testing

To assess the efficacy of Etavopivat in adolescents and adults with SCD as compared to placebo as measured by improvement in hemoglobin (Hb) To assess the efficacy of Etavopivat as compared to placebo on the annualized vaso-occlusive crisis (VOC) rate

Phase II and Phase III (Integrated)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1) Provision of consent
2) Patient has a confirmed diagnosis of sickle cell disease
3) 2-15 episodes of documented vaso-occlusive crises in the past 12 months
4) Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening
5) Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
6) Female patients of childbearing potential must use acceptable methods of contraception; male patients are willing to use acceptable methods of contraception

You likely can't join if

Medical Conditions 1) More than 15 vaso-occlusive crises within the past 12 months prior to screening
Prior/Concomitant Therapy 1) Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
2) Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
3) Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
4) Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study
5) Uso de eritropoyetina u otro tratamiento con factor de crecimiento hematopoyético dentro de los 28 días posteriores al inicio del tratamiento del estudio o la necesidad anticipada de dichos agentes durante el estudio.

See the full eligibility criteria

Who can join

1) Provision of consent
2) Patient has a confirmed diagnosis of sickle cell disease
3) 2-15 episodes of documented vaso-occlusive crises in the past 12 months
4) Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening
5) Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
6) Female patients of childbearing potential must use acceptable methods of contraception; male patients are willing to use acceptable methods of contraception
7)Patients on crizanlizumab or L-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they: • 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) • Have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent • Meet the VOC eligibility requirement in Inclusion Criterion 4.

What rules you out

Medical Conditions 1) More than 15 vaso-occlusive crises within the past 12 months prior to screening
Prior/Concomitant Therapy 1) Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
2) Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
3) Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
4) Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study
5) Uso de eritropoyetina u otro tratamiento con factor de crecimiento hematopoyético dentro de los 28 días posteriores al inicio del tratamiento del estudio o la necesidad anticipada de dichos agentes durante el estudio.
6) Receipt of prior cellular-based therapy (e.g., hematopoietic cell transplant, gene modification therapy)
2) Female who is breast feeding or pregnant
3) Hepatic dysfunction characterized by: - Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN) OR - Direct bilirubin > 3.0 × ULN
4) Known HIV positive
5) Active hepatitis B or hepatitis C infection
6) Severe renal dysfunction or on chronic dialysis
7) History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following: - Unstable angina pectoris or myocardial infarction or elective coronary intervention - Congestive heart failure requiring hospitalization - Uncontrolled clinically significant arrhythmias - Symptomatic pulmonary hypertension
8) History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
9) History of deep venous thrombosis requiring systemic anticoagulation therapy for ≥ 6 weeks, occurring within 6 months prior to Day 1 of study treatment. Note: patients on ≥ 6 months of chronic or prophylactic anti-coagulation therapy are allowed on study.

The study team makes the final eligibility decision.

Where it's taking place

Nigeria
Lebanon
India
United States
Egypt
Ghana
Turkey
United Kingdom
Oman
Canada
Kenya
Saudi Arabia

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Nigeria; Lebanon; India; United States; Egypt; Ghana and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.