RECRUITING

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

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Conditions

Sickle Cell Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2025-02-17
Study Completion:2028-09-22
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06612268

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.

Ages Eligible for Study:12 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female.
  2. * Age 12 years or above at the time of signing the informed consent.
  3. * 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.
  4. * 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.
  5. * 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.
  1. * 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.
  2. * Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study.
  3. * 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.
  4. * 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).
  5. * 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.
  6. * 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.
  7. * 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.
  8. * Receipt of prior cellular-based therapy (e.g., haematopoietic cell transplant, gene modification therapy).
  9. * Hepatic dysfunction characterized by:
  10. * Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN) or
  11. * Direct bilirubin greater than 3.0 × ULN.
  12. * 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.
  13. * 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.
  14. * Travelled distance on standardized 6MWT below 100m at screening.

Contacts and Locations

Study Contact

Novo Nordisk
CONTACT
(+1) 866-867-7178
clinicaltrials@novonordisk.com

Principal Investigator

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

Study Locations (Sites)

Uni of Alabama at Birmingham
Birmingham, Alabama, 35233
United States
Univer South Alabama Ped/Onc
Mobile, Alabama, 36604
United States
Phoenix Children's Hsptl
Phoenix, Arizona, 85016
United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
United States
UCLA Health
Los Angeles, California, 90095
United States
Valley Children's Hospital
Madera, California, 93636
United States
University Of California Irvine
Orange, California, 92868
United States
Stanford University_Palo Alto
Palo Alto, California, 94304
United States
Harbor-UCLA Medical Center
Torrance, California, 90502
United States
Clinical and Transl Res Center
Aurora, Colorado, 80045
United States
Nemours/AI duPont Hosp-Chld
Wilmington, Delaware, 19803
United States
Childrens National Medical Ctr
Washington, District of Columbia, 20010
United States
MedStar Hlth Res Institute
Washington, District of Columbia, 20010
United States
Memorial Healthcare
Hollywood, Florida, 33021
United States
Children's Healthcare Atlanta
Atlanta, Georgia, 30329
United States
Childrens Hospital of Chicago
Chicago, Illinois, 60611
United States
Univer Of Illinois at Chicago
Chicago, Illinois, 60612
United States
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Boston Medical Center
Boston, Massachusetts, 02118
United States
Henry Ford Hospital_Detroit
Detroit, Michigan, 48202
United States
University of Minnesota
Minneapolis, Minnesota, 55455
United States
Washington University-St.Louis
Saint Louis, Missouri, 63110
United States
Children's Nebraska
Omaha, Nebraska, 68114
United States
Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135
United States
Newark Beth Israel Medical Center
Newark, New Jersey, 07112-2027
United States
Jacobi Medical Center
Bronx, New York, 10461
United States
Montefiore Medical Ctr
Bronx, New York, 10467
United States
NYC Health+Hospitals
Brooklyn, New York, 11203
United States
Interfaith Medical Center
Brooklyn, New York, 11238
United States
Northwell Health
Mount Kisco, New York, 10549
United States
Cohen Children's Medical Ctr
Queens, New York, 11040
United States
Duke University Medical Center
Durham, North Carolina, 27705
United States
Duke Comprehen Sickle Cell
Durham, North Carolina, 27710
United States
East Carolina University_Greenville_0
Greenville, North Carolina, 27834
United States
Atrium Health-Wake Forest Bapt
Winston-Salem, North Carolina, 27157
United States
Univ Hosp Cleveland Med Ctr
Cleveland, Ohio, 44106
United States
Ohio State Univ Wexner Med Ctr
Columbus, Ohio, 43210
United States
Univ of OK Health Sciences Ctr
Oklahoma City, Oklahoma, 73104
United States
Children's Hosptl Philadelphia
Philadelphia, Pennsylvania, 19104
United States
St Christopher Hosp for Child
Philadelphia, Pennsylvania, 19134
United States
Methodist University Hospital
Memphis, Tennessee, 38104
United States
St. Jude Children's Res Hosp
Memphis, Tennessee, 38105
United States
UT Health University of Texas
Houston, Texas, 77030
United States
Inova Health System
Fairfax, Virginia, 22031
United States
Children's Hsptl Of The Kings
Norfolk, Virginia, 23507
United States
Virginia Comm Univ Medical Ctr
Richmond, Virginia, 23298
United States
Mary Bridge Children's Health
Tacoma, Washington, 98405
United States
Mary Bridge Children's Health
Tacoma, Washington, 98405
United States

Collaborators and Investigators

Sponsor: Novo Nordisk A/S

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-17
Study Completion Date2028-09-22

Study Record Updates

Study Start Date2025-02-17
Study Completion Date2028-09-22

Terms related to this study

Additional Relevant MeSH Terms

  • Sickle Cell Disease