Evaluating Etavopivat for Sickle Cell Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to determine the effectiveness of etavopivat in reducing the frequency of vaso-occlusive crises (pain crises) in adults and adolescents with sickle cell disease. Participants will be randomly assigned to receive either etavopivat or a placebo over a period of approximately two years. The study will also assess the drug's ability to minimize organ damage, enhance exercise tolerance, and alleviate fatigue associated with the condition. The results will help establish the therapeutic potential of etavopivat for managing sickle cell disease.

Why it matters

Who can join: the full rules

Inclusion 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 2-15 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 grams per litre \[g/L\]) at screening.

Exclusion Criteria:

* 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 within the previous 12 months prior to screening (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 or moderate 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.

Where it's running

• Uni of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Univer South Alabama Ped/Onc
  Mobile, Alabama, United States Recruiting
• Phoenix Children's Hsptl
  Phoenix, Arizona, United States Recruiting
• Arkansas Children's Hospital
  Little Rock, Arkansas, United States Recruiting
• Children's Hospital Los Angeles - Endocrinology
  Los Angeles, California, United States Recruiting
• Valley Children's Hospital
  Madera, California, United States Recruiting

+125 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novo Nordisk
• Email: clinicaltrials@novonordisk.com
• Phone: (+1) 866-867-7178

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Cerebrovascular Reactivity Testing with HD-DOT in Sickle Cell Patients

  This study tests whether high-density diffuse optical tomography can measure brain blood flow and oxygen use in people with sickle cell disease, to see how these signals relate to stroke risk.

  Recruiting Washington University School of Medicine · St Louis, Missouri · NCT07776444

• Adapting and testing PEN-Plus in Zambian primary care

  Aiming to see if the PEN-Plus package can be adapted and used in two Zambian hospitals to better manage chronic diseases like heart disease, diabetes, hypertension, and sickle cell in primary care.

  Recruiting Centre for Infectious Disease Research in Zambia · Lusaka, Lusaka Province · NCT07753681

• Newborn Sickle Cell Screening in Mozambique

  We’re testing a three-part plan to help babies in Mozambique get diagnosed early for sickle cell disease and start care quickly.

  Recruiting St. Jude Children's Research Hospital · Nhamatanda, Sofala · NCT07719972

• Optimizing Hydroxyurea in Children with Sickle Cell in Africa

  This study tests whether raising hydroxyurea to a maximum tolerated dose is safe and beneficial for Ugandan children with sickle cell anemia already taking a fixed daily dose.

  Recruiting Phase 1/2 Children's Hospital Medical Center, Cincinnati · Kampala · NCT07708714

• Hematopoietic stem cell editing to treat sickle cell disease

  This study tests whether a patient’s own blood stem cells can be edited to remove the sickle cell triggering gene and then returned to the patient.

  Recruiting Phase 1 Boston Children's Hospital · Boston, Massachusetts · NCT07708350

• Virtual reality options to help with VOC pain in adults with SCD

  This study tests whether headset-based virtual reality can help reduce pain for adults with sickle cell disease during vaso-occlusive crises treated in an infusion center.

  Recruiting University of Maryland, Baltimore · Baltimore, Maryland · NCT07674277

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.