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NCT06609226 | NOT YET RECRUITING | Sickle Cell Disease, Thalassemia

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Sponsor:

Novo Nordisk A/S

Brief Summary:

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Condition or disease

Sickle Cell Disease, Thalassemia

Intervention/treatment

Etavopivat A

Etavopivat B

Phase

PHASE3

Study Type : INTERVENTIONAL
Estimated Enrollment : 325 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study
Actual Study Start Date : 2024-12-09
Estimated Primary Completion Date : 2029-11-30
Estimated Study Completion Date : 2029-11-30

Information not available for Arms and Intervention/treatment

Ages Eligible for Study:
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • * Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study.
  • * Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
  • * Any participant with dose reduction or temporary discontinuation will need to be rechallenged before transferring.
  • * Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they:
  • * Have been on a stable dose during participation in the parent study (i.e., no changes to the dose except for changes to weight or age reasons).
  • * Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
Exclusion Criteria
  • * Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
  • * Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
  • * Participants on permanent dose reduction or temporary treatment discontinuation.
  • * Use of any of the following within the timeframes prior to the transfer visit as stated:
  • * Use of voxelotor within participation of the parent study or anticipated need for this agent during this study.
  • * Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study.
  • * Use of erythropoietin or other haematopoietic growth factor treatment within the parent study or anticipated need for such agents during this study.
  • * Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study.
  • * Current participation in a study that is not a designated parent study, or planned participation in any other clinical trial, for the duration of FLORAL.
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Location Details

NCT06609226

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, alabama

Univ of Alabama Birmingham

Birmingham, alabama, United States, 35233

Not yet recruiting

United States, Arizona

Phoenix Children's Hsptl

Phoenix, arizona, United States, 85016

Not yet recruiting

United States, California

University Of California Irvine

Irvine, California, United States, 92697

Not yet recruiting

United States, California

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States, 90027

Not yet recruiting

United States, California

UCSF Oakland Benioff ChildHosp

Oakland, California, United States, 94609

Not yet recruiting

United States, California

Children's Hosp Of Orange

Orange, California, United States, 92868

Not yet recruiting

United States, Connecticut

University of Connecticut

Farmington, Connecticut, United States, 06030

Not yet recruiting

United States, District of Columbia

Children's National Medical Center

Washington, District of Columbia, United States, 20010

Not yet recruiting

United States, Florida

Univ Miami-Miller School Med

Miami, florida, United States, 33136

Not yet recruiting

United States, Georgia

Emory University School of Medicine

Atlanta, Georgia, United States, 30303

Not yet recruiting

United States, Georgia

Children's Healthcare Atlanta

Atlanta, Georgia, United States, 30342

Not yet recruiting

United States, Louisiana

Children's Hosp-New Orleans

New Orleans, Louisiana, United States, 70118

Not yet recruiting

United States, Massachusetts

Boston Medical Center

Boston, Massachusetts, United States, 02118

Not yet recruiting

United States, New York

Jacobi Medical Center

Bronx, New York, United States, 10461

Not yet recruiting

United States, New York

Montefiore Medical Center

Bronx, New York, United States, 10467

Not yet recruiting

United States, New York

NYC Health+Hospitals

Brooklyn, New York, United States, 11203

Not yet recruiting

United States, New York

Columbia University Medical Center_New York_0

New York, New York, United States, 10032

Not yet recruiting

United States, New York

Weill Cornell With Coll-NYMPH

New York, New York, United States, 10065

Not yet recruiting

United States, North Carolina

Duke University_Durham

Durham, North Carolina, United States, 27705

Not yet recruiting

United States, North Carolina

East Carolina University_Greenville

Greenville, North Carolina, United States, 27834

Not yet recruiting

United States, North Carolina

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina, United States, 27157

Not yet recruiting

United States, Ohio

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio, United States, 45229

Not yet recruiting

United States, Ohio

Neuro-Behavioral Clinical Research

North Canton, Ohio, United States, 44720

Not yet recruiting

United States, South Carolina

Medical University Of South Carolina_Charleston

Charleston, South Carolina, United States, 29425

Not yet recruiting

United States, South Carolina

East Carolina University_Greenville_0

Greenville, South Carolina, United States, 27858

Not yet recruiting

United States, Tennessee

UTHSC-Memphis

Memphis, Tennessee, United States, 38104

Not yet recruiting

United States, Texas

UT Health University of Texas

Houston, Texas, United States, 77030

Not yet recruiting

United States, Virginia

Virginia Comm Univ Medical Ctr

Richmond, Virginia, United States, 23298

Not yet recruiting

United States, Washington

Mary Bridge Children's Health

tacoma, Washington, United States, 98405

Not yet recruiting

Canada, British Columbia

Providence Hematolgy

Vancouver, British Columbia, Canada, V6E 1M7

Not yet recruiting

Canada, Ontario

The Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8

Not yet recruiting

Canada, Ontario

University Health Network - Toronto General Hospital

Toronto, Ontario, Canada, M5G 2C4

Not yet recruiting

Canada, Quebec

CHU Ste-Justine

Montreal, Quebec, Canada, H3T 1C5

Not yet recruiting

France,

Ap-Hp-Hospital Henri Mondor

Creteil, France, 94000

Not yet recruiting

France,

Hospices Civils de Lyon-Hospital Edouard Herriot

Lyon Cedex 03, France, 69437

Not yet recruiting

France,

aap-hap-hopital robert debre

Paris, France, 75019

Not yet recruiting

Germany,

Charité Campus Virchow Klinikum - Clinic for pediatrics with a focus on oncology and hematology

Berlin, Germany, 13353

Not yet recruiting

Germany,

Freiburg University Hospital, children's and youth clinic

Freiburg, Germany, 79106

Not yet recruiting

Greece, Thessaly

General Hospital of Larissa Koutlivanio And Triantafyllio

Larissa, Thessaly, Greece, 412 21

Not yet recruiting

Greece,

Hippokration Hospital

Athens, Greece, 11527

Not yet recruiting

Greece,

General University Hospital of Patras

Patra, Greece, 26504

Not yet recruiting

Greece,

'Hippocrateo' General Hospital of Thessaloniki

Thessaloniki, Greece, 54642

Not yet recruiting

Italy, Torino

San Luigi Gonzaga University Hospital

Orbassano, Torino, Italy, 10043

Not yet recruiting

Italy,

Padua University Hospital Company

Padova, Italy, 35128

Not yet recruiting

Italy,

IRCCS Policlinico San Matteo Foundation

Pavia, Italy, 27100

Not yet recruiting

Lebanon,

American University of Beirut Medical Centre

Hammer, Lebanon,

Not yet recruiting

Lebanon,

Chronic Care Center

Hazmieh, Lebanon, 21211

Not yet recruiting

Lebanon,

Hospital What

Tripoli, Lebanon, 1434

Not yet recruiting

Oman,

Sultan Qaboos University Hospital

Muscat, Oman, 123

Not yet recruiting

Saudi Arabia,

Prince Mohammad Bin Nasser Hospital

Jizan, Saudi Arabia, 82943

Not yet recruiting

Saudi Arabia,

King Khalid University Hospital

Riyadh, Saudi Arabia, 12372

Not yet recruiting

Spain,

Cruces University Hospital

Barakaldo, Spain, 48903

Not yet recruiting

Spain,

Hebron Valley Hospital

Barcelona, Spain, 08035

Not yet recruiting

Spain,

La Paz University Hospital

Madrid, Spain, 28046

Not yet recruiting

Turkey,

Baskent University Adana

Adana, Turkey, 01250

Not yet recruiting

Turkey,

Hacettepe University Hematology

Ankara, Turkey, 06230

Not yet recruiting

Turkey,

Mersin University Medical Faculty Hospital, Hematology

Myrtle, Turkey, 33110

Not yet recruiting

Turkey,

Mersin University Medical Faculty Pediatric Hematology

Myrtle, Turkey, 33110