Study on the Effects of Etavopivat for Patients Aged 12-65 with Sickle Cell Disease

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What is this study about?

This clinical trial is focused on studying sickle cell disease, a condition that affects the red blood cells, causing them to become misshapen and break down. The study is testing a new treatment called Etavopivat, which is a type of medication known as a pyruvate kinase activator. This medication is being tested in two different doses, 100 mg and 200 mg, and will be compared to a placebo. The purpose of the study is to see if Etavopivat can help improve hemoglobin levels and reduce the number of painful episodes known as vaso-occlusive crises, which are common in people with sickle cell disease.

Participants in the study will be randomly assigned to receive either Etavopivat or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are not biased. The study will take place over a period of time, with regular check-ins to monitor the participants’ health and any changes in their condition. The main goal is to assess the effectiveness of Etavopivat in improving health outcomes for those with sickle cell disease.

Throughout the study, participants will have their hemoglobin levels checked and will be monitored for any changes in their condition. The study will also look at other health markers related to sickle cell disease, such as the number of red blood cells and certain proteins in the blood. By the end of the study, researchers hope to determine whether Etavopivat is a safe and effective treatment option for people living with sickle cell disease.

1 joining the study

Upon joining the study, consent is required. A confirmed diagnosis of sickle cell disease is necessary.

Eligibility includes having 2-15 episodes of vaso-occlusive crises in the past year and specific hemoglobin levels during screening.

2 initial assessment

An initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current treatments.

Patients on hydroxyurea must have a stable dose for at least 90 days before starting the study treatment.

3 treatment phase

The treatment involves taking etavopivat or a placebo orally. Dosages include 100 mg or 200 mg tablets.

The treatment is administered over a 52-week period, with regular monitoring of hemoglobin levels and vaso-occlusive crisis rates.

4 monitoring and evaluation

Primary evaluations occur at Week 24 and Week 52, focusing on hemoglobin response and annualized vaso-occlusive crisis rates.

Secondary evaluations include changes in clinical laboratory measurements and patient-reported fatigue levels.

5 completion of study

The study is estimated to conclude by February 2027. Final assessments will be conducted to evaluate the overall efficacy of the treatment.

Who Can Join the Study?

Provide consent to participate in the study.
Have a confirmed diagnosis of sickle cell disease (SCD).
Have experienced 2 to 15 episodes of documented vaso-occlusive crises in the past 12 months. A vaso-occlusive crisis is a painful episode that occurs when blood flow is blocked by sickle-shaped cells.
Have a hemoglobin level between 5.5 and 10.5 g/dL during screening. Hemoglobin is a protein in red blood cells that carries oxygen.
If taking hydroxyurea, must have been on a stable dose for at least 90 days before starting the study treatment. Hydroxyurea is a medication used to reduce complications of sickle cell disease.
Female patients who can have children must use acceptable methods of contraception. Male patients must be willing to use acceptable methods of contraception.
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 at least 12 months, have been at least 80% compliant with the planned regimen during the 12 months prior to consent, and meet the vaso-occlusive crisis eligibility requirement.

Who Cannot Join the Study?

Individuals who do not have sickle cell disease (SCD) cannot participate. SCD is a group of inherited red blood cell disorders.
Participants must be within the specified age range for the study.
Both males and females are eligible, but certain conditions may exclude participation.
Individuals who are part of a vulnerable population may be excluded. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.
Any other specific medical conditions or treatments that might interfere with the study’s objectives could lead to exclusion.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Clinico San Carlos	Madrid	Spain
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Hospital Edouard Herriot	Lyon	France
Ippokratio General Hospital Of Thessaloniki	Thessaloniki	Greece
General Hospital Of Larissa Koutlibaneio And Triantafylleio	Larissa	Greece
Azienda Ospedaliera di Padova	Padua	Italy
Hippokration Hospital	Athens	Greece
Virgen del Rocío University Hospital	Sevilla	Spain
Azienda Ospedaliero-Universitaria San Luigi Gonzaga	Orbassano	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Hospital Universitario De Cruces	Barakaldo	Spain
Robert Debre University Hospital	Paris	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
General University Hospital Of Patras	Patras	Greece
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Hopital Beaujon	Clichy	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Fncnmlnkk Pnxs Li Igsckjidkixeg Bfjqeaxxz Dem Hkbsctjn Uxplqyuupkemd Lu Ppr	Madrid	Spain
Hnddzacq Vqsv drgrmici	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	29.01.2021
Germany	Not recruiting	29.01.2021
Greece	Not recruiting	29.01.2021
Italy	Not recruiting	29.01.2021
Spain	Not recruiting	29.01.2021

Trial locations

Investigated drugs:

Etavopivat

Etavopivat is a medication being studied for its potential benefits in patients with Sickle Cell Disease. It works by activating an enzyme called pyruvate kinase, which may help improve the levels of hemoglobin in the blood. This could lead to better oxygen transport in the body and potentially reduce the frequency of painful episodes known as vaso-occlusive crises, which are common in people with Sickle Cell Disease.

Investigated diseases:

Sickle cell disease

Sickle Cell Disease – Sickle Cell Disease is a genetic disorder that affects the shape and function of red blood cells. Normally, red blood cells are round and flexible, allowing them to move easily through blood vessels. In Sickle Cell Disease, the red blood cells become rigid and shaped like a crescent or sickle. These sickle-shaped cells can get stuck in small blood vessels, which can slow or block blood flow and oxygen to parts of the body. This can lead to episodes of pain, known as vaso-occlusive crises, and other complications. Over time, the disease can cause damage to organs and tissues due to reduced blood flow.

Trial ID:

2024-511535-97-00

Protocol code:

4202-HEM-301

NCT ID:

NCT04624659

Trial Phase:

Therapeutic use (Phase IV)

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Study on the Effects of Etavopivat for Patients Aged 12-65 with Sickle Cell Disease

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