A Research Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Description

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

Conditions

Sickle Cell Disease

Study Overview

Study Details

Study overview

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

A Global Phase 3, Randomised, Double-blind and Placebo-controlled Study Evaluating the Efficacy and Safety of Etavopivat in Adolescents and Adults With Sickle Cell Disease

A Research Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Birmingham

Uni of Alabama at Birmingham, Birmingham, Alabama, United States, 35233

Mobile

Univer South Alabama Ped/Onc, Mobile, Alabama, United States, 36604

Phoenix

Phoenix Children's Hsptl, Phoenix, Arizona, United States, 85016

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202

Los Angeles

Children's Hospital Los Angeles - Endocrinology, Los Angeles, California, United States, 90027

Los Angeles

UCLA Health, Los Angeles, California, United States, 90095

Madera

Valley Children's Hospital, Madera, California, United States, 93636

Orange

University Of California Irvine, Orange, California, United States, 92868

Palo Alto

Stanford University_Palo Alto, Palo Alto, California, United States, 94304

Torrance

Harbor-UCLA Medical Center, Torrance, California, United States, 90502

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Male or female.
  • * Age 12 years or above at the time of signing the informed consent.
  • * Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing or screening test results from central laboratory. Molecular genotyping is not required. SCD genotype may be determined from the results of haemoglobin (Hb) electrophoresis, high-performance liquid chromatography (HPLC) or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening.
  • * Have 1-15 episodes of documented vaso occlusive crises (VOC) within the 12 months prior to screening. Documentation must exist in the participant's medical record prior to randomisation. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
  • * Hb greater than or equal to (≥) 5.0 and less than or equal to (≤) 10.0 g/dL (greater than or equal to (≥) 50 and less than or equal to (≤) 100 g/L) at screening.
  • * More than 15 VOCs within the past 12 months prior to screening documented in the participant's medical record. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
  • * Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study.
  • * Use of a selectin antagonist (e.g., crizanlizumab, monoclonal antibody or small molecule) within 28 days or 5 half-lives (whichever is longer) prior to starting study treatment or anticipated need for such agents during the study.
  • * Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or greater than or equal to 6 transfusion events in the previous 12 months (i.e., an average of 1 transfusion event every 60 days).
  • * Participants who have received an RBC transfusion for any reason within 60 days of the screening period or 60 days of the randomisation day are only eligible if HbA (adult haemoglobin) less than 10% by Hb electrophoresis is documented prior to starting study treatment.
  • * Receiving or use of concomitant medications that are strong inducers of CYP3A4 (cytochrome p450 3a4) within 2 weeks of starting study treatment or anticipated need for such agents during the study.
  • * Use of erythropoietin or other haematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study.
  • * Receipt of prior cellular-based therapy (e.g., haematopoietic cell transplant, gene modification therapy).
  • * Hepatic dysfunction characterized by:
  • * Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN) or
  • * Direct bilirubin greater than 3.0 × ULN.
  • * Participants who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended.
  • * Severe renal dysfunction (estimated glomerular filtration rate \[eGFR\] at screening, calculated by the central laboratory greater than 30 mL/min/1.73 m\^ 2) or on chronic dialysis.
  • * Travelled distance on standardized 6MWT below 100m at screening.

Ages Eligible for Study

12 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novo Nordisk A/S,

Clinical Transparency (dept. 2834), STUDY_DIRECTOR, Novo Nordisk A/S

Study Record Dates

2028-09-22