Clinical trial NCT06609226: A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Sickle Cell Disease, Thalassemia
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2024-09-24
Enrollment: 325
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
timeFrame: Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)
measure: Number of adverse reactions, reported for each indication and age group separately
timeFrame: Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)
Secondary outcomes:
measure: Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately
timeFrame: Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier
measure: Change in VOCs, reported for each age group separately
timeFrame: Baseline (of parent study) to end of treatment at week 260, or earlier
measure: Change in hemoglobin (Hb) concentration, reported for each age group separately
timeFrame: Baseline (of parent study) to end of treatment at week 260, or earlier
measure: Annualised number of hospitalisations, reported for each age group separately
timeFrame: Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier
measure: Average length of stay of hospitalisations, reported for each age group separately
timeFrame: Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier
measure: Change in Hb concentration
timeFrame: Baseline (of parent study) to end of treatment at week 260, or earlier
measure: Number of red blood cell (RBC) units transfused, reported for each indication separately
timeFrame: Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier
measure: Change in RBC units transfused, reported for each indication separately
timeFrame: Baseline (of parent study) to end of treatment at week 260, or earlier
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Univ of Alabama Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hsptl
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles - Endocrinology
state: California
city: Oakland
country: United States
facility: UCSF Oakland Benioff ChildHosp
state: California
city: Orange
country: United States
facility: Children's Hosp Of Orange
state: California
city: Orange
country: United States
facility: University Of California Irvine
state: California
city: Farmington
country: United States
facility: University of Connecticut
state: Connecticut
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Miami
country: United States
facility: Univ of Miami/SCCC
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare Atlanta
state: Georgia
city: Augusta
country: United States
facility: Center for Blood Disorders Augusta University
state: Georgia
city: Chicago
country: United States
facility: Univer Of Illinois at Chicago
state: Illinois
city: New Orleans
country: United States
facility: Children's Hosp-New Orleans
state: Louisiana
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University-St.Louis
state: Missouri
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Brooklyn
country: United States
facility: NYC Health+Hospitals
state: New York
city: New York
country: United States
facility: Columbia University Medical Center_New York_0
state: New York
city: New York
country: United States
facility: Weill Cornell Med Coll-NYPH
state: New York
city: Durham
country: United States
facility: Duke University_Durham
state: North Carolina
city: Greenville
country: United States
facility: East Carolina Univ-Greenville
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University_Greenville
state: North Carolina
city: Winston-Salem
country: United States
facility: Atrium Health-Wake Forest Bapt
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Child's Hsp Med Ctr
state: Ohio
city: North Canton
country: United States
facility: Neuro-Behavioral Clinical Research
state: Ohio
city: Charleston
country: United States
facility: Medical University Of South Carolina_Charleston
state: South Carolina
city: Memphis
country: United States
facility: UTHSC-Memphis
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital_Houston
state: Texas
city: Houston
country: United States
facility: UT Health University of Texas
state: Texas
city: Richmond
country: United States
facility: Virginia Comm Univ Medical Ctr
state: Virginia
city: Tacoma
country: United States
facility: Mary Bridge Children's Health
state: Washington
city: Milwaukee
country: United States
facility: Versiti, CCBD_Milwaukee
state: Wisconsin
city: Vancouver
country: Canada
facility: Providence Hematolgy
state: British Columbia
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Toronto
country: Canada
facility: University Health Network - Toronto General Hospital
state: Ontario
city: Montreal
country: Canada
facility: CHU Ste-Justine
state: Quebec
city: Alexandria, Egypt
country: Egypt
facility: Alexandria University Hospital
city: Alsharkia, Egypt
country: Egypt
facility: Zagazig University Hospital
city: Cairo, Egypt
country: Egypt
facility: Cairo University
city: Cairo, Egypt
country: Egypt
facility: Abu El-Reesh El-Mounira Children University Hospital
city: Cairo
country: Egypt
facility: Faculty of Medicine Ain Shams Medical Research Institute (MASRI)
city: Créteil
country: France
facility: Ap-Hp-Hopital Henri Mondor
city: Lyon Cedex 03
country: France
facility: Hospices Civils de Lyon-Hopital Edouard Herriot
city: Paris
country: France
facility: Ap-Hp-Hopital Robert Debre
city: Paris
country: France
facility: Ap-Hp-Hopital Robert Debre
city: Berlin
country: Germany
facility: Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg, Kinder- und Jugendklinik
city: AccrA
country: Ghana
facility: Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)
city: Kintampo
country: Ghana
facility: Kintampo Health Research Centre (KHRC), Kintampo, Ghana
city: Larissa
country: Greece
facility: General Hospital Of Larissa Koutlibaneio And Triantafylleio
state: Thessaly
city: Athens
country: Greece
facility: Hippokration Hospital
city: Patra
country: Greece
facility: General University Hospital of Patras
city: Thessaloniki
country: Greece
facility: 'Ippokrateio' General Hospital of Thessaloniki
city: Raipur
country: India
facility: All India Institute of Medical Sciences (AIIMS), Raipur
state: Chhattisgarh
city: Mumbai
country: India
facility: K.J Somaiya Hospital and Research Centre
state: Maharashtra
city: Bangalore
country: India
facility: Victoria Hospital, Bangalore
city: Gujarat
country: India
facility: Nirmal Hospital Pvt. Ltd.
city: Maharashtra
country: India
facility: Suretech Hospital and Research Centre Ltd.
city: New Delhi
country: India
facility: All India Institute of Medical Sciences_Delhi
city: Orbassano
country: Italy
facility: Azienda Ospedaliera Universitaria San Luigi Gonzaga
state: Torino
city: Padova
country: Italy
facility: Azienda Ospedale Universita Padova
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo
city: Kericho
country: Kenya
facility: KEMRI-Walter-Reed Kericho
city: Kisumu
country: Kenya
facility: Kombewa Clinical Research Centre
city: Kisumu
country: Kenya
facility: Ahero Clinical Trials Unit, Kisumu, Kenya
city: Kisumu
country: Kenya
facility: KEMRI Kondele Children Hospital, Kisumu
city: Nairobi
country: Kenya
facility: Gertrude's Children's Hospital, Nairobi
city: Siaya
country: Kenya
facility: KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya
city: Hamra
country: Lebanon
facility: American University of Beirut Medical Centre
city: Hazmieh
country: Lebanon
facility: Chronic Care Center
city: Tripoli
country: Lebanon
facility: Hospital Nini
city: Ibadan
country: Nigeria
facility: University College Hospital Paediatric Haematology and Oncology Unit, Ibadan
state: Oyo State
city: Abuja
country: Nigeria
facility: University of Abuja Teaching Hospital, Gwagwalada, Abuja
city: Enugu
country: Nigeria
facility: University of Nigeria Teaching Hospital, Enugu
city: Kaduna
country: Nigeria
facility: Barau Dikko Teaching Hospital, Kaduna
city: Kano
country: Nigeria
facility: Aminu Kano Teaching Hospital (AKTH)
city: Lagos
country: Nigeria
facility: Lagos University Teaching Hospital, Lagos
city: Muscat
country: Oman
facility: Sultan Qaboos University Hospital
city: Jizan
country: Saudi Arabia
facility: Prince Mohammad Bin Naser Hospital
city: Riyadh
country: Saudi Arabia
facility: King Khalid University Hospital
city: Baracaldo
country: Spain
facility: Hospital Universitario de Cruces
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Adana
country: Turkey
facility: Baskent Universitesi Adana
city: Ankara
country: Turkey
facility: Hacettepe University Hematology
city: Mersin
country: Turkey
facility: Mersin University Medical Faculty Hospital, Hematology
city: Mersin
country: Turkey
facility: Mersin University Medical Faculty Pediatric Hematology
city: London
country: United Kingdom
facility: Kings College Hospital
city: London
country: United Kingdom
facility: Imperial College London
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary_Manchester_0
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency (dept. 2834)
role: STUDY_DIRECTOR
References: