Clinical trial NCT04624659: A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
Official title: An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients With Sickle Cell Disease (HIBISCUS)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Sickle Cell Disease
Keywords: Sickle Cell Disease
Sickle Cell
Anemia
Sickle Cell Anemia
Hemolytic
Hemoglobin
Vaso-occlusive Crisis
Sickle Cell Crisis
Congenital Anemia
Hemolytic Anemia
Hematologic Disease
Hemoglobinopathies
Genetic Disease
Inborn Disease
Sickle Cell Trait
Pyruvate Kinase
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
Collaborators: name: Novo Nordisk A/S
First posted: 2020-11-12
Enrollment: 450
Sex: ALL
Minimum age (years): 12
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).

Detailed description: Etavopivat is designed to activate PKR and thereby modulate RBC metabolism by impacting two critical pathways in RBCs. The etavopivat clinical development program will investigate whether decreasing 2,3-DPG may help oxygen bind to hemoglobin (i.e. increasing oxygen affinity), and thereby increase ATP and impact RBC function. This study is a randomized, placebo-controlled, double-blind, multicenter Phase 2/3 study of patients age 12 to 65 years (inclusive), with sickle cell disease. There is one planned interim analyses in this study design. Initially, patients will be randomized at 1:1:1 to one of two dose levels of etavopivat or placebo. At the first interim analysis, one of the two etavopivat dose levels will be selected for the Phase 3 portion of the study, in which patients will be randomized at 1:1 to the selected etavopivat dose or placebo. Efficacy on hemoglobin will be evaluated at conclusion of the double-blind treatment period. Following completion of 52 weeks of double-blind treatment, patients may enter a 112-week etavopivat open-label extension period.

Eligibility criteria: Key Inclusion Criteria:

* Provision of consent
* Patient has a confirmed diagnosis of sickle cell disease
* At least 2 episodes of vaso-occlusive crises in the past 12 months
* Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening
* Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
* Patients on crizanlizumab or L-glutamine treatment at the time of consent must be on a stable dose for ≥ 12 months and must be ≥ 80% compliant with the planned regimen at the time of consent and meet the VOC eligibility criteria
* Female patients of childbearing potential must use highly effective methods of contraception, male patients are willing to use barrier methods of contraception

Key Exclusion Criteria:

* More than 15 vaso-occlusive crises within the past 12 months
* Female who is breastfeeding or pregnant
* Hepatic dysfunction characterized by:

  * Alanine aminotransferase (ALT) \> 4.0 × upper limit of normal (ULN)
  * Direct bilirubin \> 3.0 × ULN
* Known HIV positivity
* Active hepatitis B or hepatitis C infection
* Severe renal dysfunction or on chronic dialysis
* History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:

  * Unstable angina pectoris or myocardial infarction or elective coronary intervention
  * Congestive heart failure requiring hospitalization
  * Uncontrolled clinically significant arrhythmias
  * Symptomatic pulmonary hypertension
* History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
* History of deep venous thrombosis requiring systemic anti-coagulation therapy for ≥ 6 weeks, occurring within 6 months prior to Day 1 of study treatment.

Prior/Concomitant Therapy

* Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
* Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
* Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
* Use of an experimental selectin antagonist (eg, monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study
* Use of erythropoietin or other hematopoietic 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 (eg, hematopoietic cell transplant, gene modification therapy)

Primary outcomes:
measure: Hemoglobin response rate
timeFrame: 24 Weeks
measure: Annualized vaso-occlusive crisis
timeFrame: 52 Weeks
Secondary outcomes:
measure: Hemoglobin
timeFrame: 52 Weeks
measure: Absolute reticulocyte count
timeFrame: 24 Weeks
measure: Indirect bilirubin
timeFrame: 24 Weeks
measure: Lactate dehydrogenase
timeFrame: 24 Weeks
measure: Vaso-occlusive crisis
timeFrame: 52 Weeks
measure: Fatigue
timeFrame: 52 Weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Univ of Alabama Birmingham
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama at Birmingham (UAB)
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Phoenix
country: United States
facility: Phoenix Children's Hsptl
state: Arizona
city: Little Rock
country: United States
facility: [Legal] Woodland International Research Group, LLC
state: Arkansas
city: Little Rock
country: United States
facility: Woodland International Research Group
state: Arkansas
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Long Beach
country: United States
facility: [Legal] Collaborative Neuroscience Network, LLC
state: California
city: Long Beach
country: United States
facility: Collaborative Neuroscience Research, LLC.
state: California
city: Oakland
country: United States
facility: Pacific Research Partners, LLC
state: California
city: Oakland
country: United States
facility: Pacific Research Partners
state: California
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Oakland
country: United States
facility: UCSF Oakland Benioff ChildHosp
state: California
city: Orange
country: United States
facility: University Of California Irvine
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center - UC Davis Comprehensive Cancer Center - Hemotology/Oncology Clinic
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: Farmington
country: United States
facility: University of Connecticut (UCONN) Health
state: Connecticut
city: Farmington
country: United States
facility: University of Connecticut
state: Connecticut
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Washington
country: United States
facility: Children's National Health Center
state: District of Columbia
city: Washington
country: United States
facility: Howard University
state: District of Columbia
city: Altamonte Springs
country: United States
facility: Cornerstone Research Institute
state: Florida
city: Gainesville
country: United States
facility: University of Florida Hematology
state: Florida
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Hollywood
country: United States
facility: Office of Gershwin T. Blyden, MD
state: Florida
city: Hollywood
country: United States
facility: Foundation for Sickle Cell Disease Research
state: Florida
city: Miami
country: United States
facility: University of Miami - Miller School of Medicine
state: Florida
city: Miami
country: United States
facility: University Of Miami
state: Florida
city: Miami
country: United States
facility: Advanced Pharma CR LLC.
state: Florida
city: Miami
country: United States
facility: Advanced Pharma CR
state: Florida
city: Orlando
country: United States
facility: Arnold Palmer Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Arnold Palmer Hospital for Children - Haley Center for Children's Cancer and Blood Disorders
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Atlanta
country: United States
facility: Sonar Clinical Research
state: Georgia
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Atlanta
country: United States
facility: Sonar Clinical Research
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Pediatric Research Center
state: Georgia
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta Investigational Medication Pharmacy
state: Georgia
city: Augusta
country: United States
facility: Augusta University Center for Blood Disorders.
state: Georgia
city: Augusta
country: United States
facility: Center for Blood Disorders Augusta University
state: Georgia
city: Decatur
country: United States
facility: iResearch Atlanta, LLC
state: Georgia
city: Chicago
country: United States
facility: University of Illinois at Chicago Sickle Cell Center
state: Illinois
city: Chicago
country: United States
facility: University of Illinois at Chicago
state: Illinois
city: New Orleans
country: United States
facility: Children's Hosp-New Orleans
state: Louisiana
city: New Orleans
country: United States
facility: Children's Hospital New Orleans
state: Louisiana
city: Baltimore
country: United States
facility: University of Maryland School of Medicine
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital_Boston
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Barbara Ann Karmanos Cancer Institute
state: Michigan
city: Detroit
country: United States
facility: Detroit Medical Center (DMC) - Children's Hospital of Michigan (CHM)
state: Michigan
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Saint Louis
country: United States
facility: Washington University School of Medicine Barnes - Jewish Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine_St. Louis
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Washington
country: United States
facility: Children's National Health Hospital
state: New Jersey
city: Bronx
country: United States
facility: Jacobi Medical Center
state: New York
city: Bronx
country: United States
facility: Children's Hospital at Montefiore
state: New York
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Brooklyn
country: United States
facility: Kings County Hospital
state: New York
city: Brooklyn
country: United States
facility: NYC Health+Hospitals
state: New York
city: Jamaica
country: United States
facility: Queens Hospital Center
state: New York
city: Jamaica
country: United States
facility: Queens Hospital Center
state: New York
city: New Hyde Park
country: United States
facility: North Shore Long Island Jewish Medical Center
state: New York
city: New Hyde Park
country: United States
facility: Long Island Jewish Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center_New York
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: New York
country: United States
facility: Weill Cornell Med Coll-NYPH
state: New York
city: Chapel Hill
country: United States
facility: [Legal] The University of North Carolina at Chapel Hill
state: North Carolina
city: Chapel Hill
country: United States
facility: UNC School of Medicine
state: North Carolina
city: Charlotte
country: United States
facility: Levine Cancer Institute
state: North Carolina
city: Durham
country: United States
facility: Duke University - Sickle Cell Center
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University (ECU) Physicians
state: North Carolina
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Health
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati Cancer Center
state: Ohio
city: Cincinnati
country: United States
facility: University of Cincinnati
state: Ohio
city: Cincinnati
country: United States
facility: [Legal] Children's Hospital Medical Center dba Cincinnati Children's
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Medical Center
state: Ohio
city: North Canton
country: United States
facility: Neuro-Behavioral Clinical Research
state: Ohio
city: Oklahoma City
country: United States
facility: Jimmy Everest Cancer Hematology & Oncology
state: Oklahoma
city: Tulsa
country: United States
facility: Lynn Institute of Tulsa
state: Oklahoma
city: Philadelphia
country: United States
facility: Children's Hosptl Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia (CHOP)
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Charleston
country: United States
facility: Medical University of South Carolina (MUSC)
state: South Carolina
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Greenville
country: United States
facility: Prisma Health-Upstate
state: South Carolina
city: Greenville
country: United States
facility: Prisma Health
state: South Carolina
city: Memphis
country: United States
facility: Methodist University Hospital
state: Tennessee
city: Memphis
country: United States
facility: [Legal] St. Jude Children's Research Hospital
state: Tennessee
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital (SJCRH)
state: Tennessee
city: Nashville
country: United States
facility: Methodist University Hospital
state: Tennessee
city: Houston
country: United States
facility: [Legal] Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Houston
country: United States
facility: UT Health University of Texas
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Tacoma
country: United States
facility: Mary Bridge Children's Health Center
state: Washington
city: Tacoma
country: United States
facility: Mary Bridge Children's Health
state: Washington
city: Milwaukee
country: United States
facility: Blood Center of Wisconsin (BCW)
state: Wisconsin
city: Milwaukee
country: United States
facility: Versiti Blood Center of Wisconsin
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 Sainte-Justine Mother and Child University Hospital
state: Quebec
city: Montréal
country: Canada
facility: Universite de Montreal - Centre Hospitalier Universitaire (CHU) Sainte-Justine
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Toronto
country: Canada
facility: University Health Network Toronto General Hospital
city: Vancouver
country: Canada
facility: Providence Hematology
city: Alexandria
country: Egypt
facility: [Legal] Alexandria Clinical Research LLC
city: Alexandria
country: Egypt
facility: [Legal] Alexandria Clinical Research LLC
city: Alexandria
country: Egypt
facility: Alexandria Clinical Research Center
city: Alexandria
country: Egypt
facility: Alexandria Faculty of Medicine - Clinical Research Center
city: Cairo
country: Egypt
facility: Ain Shams University Hospital
city: Cairo
country: Egypt
facility: Cairo University
city: Cairo
country: Egypt
facility: Al Kasr AL Aini Hospital
city: Cairo
country: Egypt
facility: Ain Shams University Hospital
city: Cairo
country: Egypt
facility: Al Kasr AL Aini Hospital
city: Cairo
country: Egypt
facility: Cairo University
city: Zagazig
country: Egypt
facility: Sydnawy University Hospital
city: Zagazig
country: Egypt
facility: Sydnawy University Hospital
city: Montpellier
country: France
facility: CHU Montpellier, Hôpital Saint-Eloi
state: Montpellier Cedex 5
city: Creteil
country: France
facility: Ap-Hp-Hopital Henri Mondor
city: Créteil
country: France
facility: Hôpital Henri Mondor
city: Lyon
country: France
facility: Hospices Civils de Lyon-Hopital Edouard Herriot
city: Lyon
country: France
facility: Hôpital Edouard HERRIOT
city: Montpellier Cedex 5
country: France
facility: Centre Hospitalier Universitaire de Montpellier-Hopital Saint-Eloi
city: Mulhouse
country: France
facility: GHR Mulhouse Sud-Alsace
city: Mulhouse
country: France
facility: Hôpital Emile Muller
city: Paris
country: France
facility: Ap-Hp-Hopital Robert Debre
city: Paris
country: France
facility: CHU Paris - Hôpital Robert Debré
city: Saint-Denis
country: France
facility: Saint-Denis Hospital Center
city: Berlin
country: Germany
facility: Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie
city: Berlin
country: Germany
facility: Charite Berlin - University Medicine
city: Frankfurt am Main
country: Germany
facility: Universitätsklinikum Frankfurt
city: Frankfurt
country: Germany
facility: Univertatsklinikum Frankfurt
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg-Zentrum für Kinder- und Jugendmedizin
city: Freiburg
country: Germany
facility: Universitätsklinikum Freiburg
city: Hamburg
country: Germany
facility: University Medical Center Hamburg-Eppendorf
city: Hamburg
country: Germany
facility: UKE Hamburg
city: Heidelberg
country: Germany
facility: Uniklinik Heidelberg - Klinik für Pädiatrische Onkologie, Hämatologie, Immunologie und Pneumologie
city: Heidelberg
country: Germany
facility: University Hospital of Heidelberg
city: Regensburg
country: Germany
facility: Universitätsklinikum Regensburg
city: Regensburg
country: Germany
facility: Universitatsklinikum Regensburg
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: Patras
country: Greece
facility: General University Hospital of Patras
state: Larissa
city: Rio
country: Greece
facility: General University Hospital of Patras
state: Patras
city: Athens
country: Greece
facility: Gen Hosp Athens Ippokrateio,B' Uni Clinic Internal Medicine
city: Athens
country: Greece
facility: Hippocratio General Hospital
city: Larissa
country: Greece
facility: General Hospital Of Larissa Koutlibaneio And Triantafylleio
city: Larissa
country: Greece
facility: General University Hospital of Larissa
city: Thessaloniki
country: Greece
facility: 'Ippokrateio' General Hospital of Thessaloniki
city: Thessaloniki
country: Greece
facility: General Hospital of Thessaloniki "Hippokration"
city: Mumbai
country: India
facility: K.J Somaiya Hospital and Research Centre
state: Maharashtra
city: Bangalore
country: India
facility: Victoria Hospital, Bangalore
city: Cutack
country: India
facility: Srirama Chandra Bhanj Medical College & Hospital (S.C.B Medical College)
city: Hyderabad
country: India
facility: Yashoda Hospital
city: New Delhi
country: India
facility: All India Institute of Medical Sciences (AIIMS)
city: Pavia
country: Italy
facility: Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo
state: PV
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico "G. Rodolico - San Marco"
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico G. Rodolico - San Marco
city: Orbassano
country: Italy
facility: Azienda Ospedaliera Universitaria San Luigi Gonzaga
city: Padova
country: Italy
facility: Azienda Ospedale Universita Padova
city: Padova
country: Italy
facility: Azienda Ospedale Università Padova
city: Palermo
country: Italy
facility: ARNAS Ospedali Civico Di Cristina Benfratelli
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Ges
city: Rome
country: Italy
facility: IRCCS Ospedale Pediatrico Bambino Gesu
city: Kisumu
country: Kenya
facility: Kombewa Clinical Research Centre
city: Kisumu
country: Kenya
facility: KEMRI Kondele Children Hospital, Kisumu
city: Kisumu
country: Kenya
facility: KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya
city: Nairobi
country: Kenya
facility: Gertrude's Children's Hospital, Nairobi
city: Beirut
country: Lebanon
facility: American University of Beirut Medical center
city: Beirut
country: Lebanon
facility: American University of Beirut Medical Center
city: Tripoli
country: Lebanon
facility: Hospital Nini
city: Tripoli
country: Lebanon
facility: Nini Hospital
city: Abuja
country: Nigeria
facility: University of Abuja Teaching Hospital, Gwagwalada, Abuja
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: Lagos
country: Nigeria
facility: University of Nigeria Teaching Hospital, Enugu
city: Muscat
country: Oman
facility: Sultan Qaboos University Hospital
city: Damman
country: Saudi Arabia
facility: King Fahad Specialist Hospital-Dammam
city: Jazan
country: Saudi Arabia
facility: Prince Mohammad Bin Naser Hospital
city: Riyadh
country: Saudi Arabia
facility: King Khaled University Hospital,King Saud Univ. Med. City
city: Badalona
country: Spain
facility: Hospital Germans Trias i Pujol
city: Baracaldo
country: Spain
facility: Hospital Universitario de Cruces
city: Barakaldo
country: Spain
facility: Hospital De Cruces
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron de Barcelona
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu
city: La Coruna
country: Spain
facility: Complejo Hospitalario Universitario A Coruña
city: Madrid
country: Spain
facility: Hospital Gregorio Marañón
city: Madrid
country: Spain
facility: Hospital Ramón y Cajal
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Madrid
country: Spain
facility: Hospital Clínico San Carlos
city: Madrid
country: Spain
facility: Hospital Universitario La Paz
city: Madrid
country: Spain
facility: Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío
city: Adana
country: Turkey
facility: Cukurova Universitesi
city: Adana
country: Turkey
facility: Baskent Universitesi Adana
city: Adana
country: Turkey
facility: Adana City Training and Research Hospital
city: Ankara
country: Turkey
facility: Hacettepe Universitesi
city: Izmir
country: Turkey
facility: Ege University Medical Faculty
city: Mersin
country: Turkey
facility: Mersin University Medical Faculty Hospital, Hematology
city: Mersin
country: Turkey
facility: VM Medical Park Mersin Hospital
city: Leicester
country: United Kingdom
facility: Leicester Royal Infirmary
city: London
country: United Kingdom
facility: University Hospital Lewisham, Lewisham and Greenwich NHS Trust
city: London
country: United Kingdom
facility: Guy's and St Thomas' NHS Foundation Trust
city: London
country: United Kingdom
facility: Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
city: London
country: United Kingdom
facility: King's College Hospital
city: London
country: United Kingdom
facility: Kings College Hospital
city: London
country: United Kingdom
facility: Hammersmith Hospital - Imperial College Healthcare NHS Trust
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust - Hammersmith Hospital
city: London
country: United Kingdom
facility: Imperial College Healthcare NHS Trust - St Mary's Hospital
city: Oxford
country: United Kingdom
facility: Churchill Hospital
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Trust - Churchill Hospital - Cancer and Haematology Centre
city: Sheffield
country: United Kingdom
facility: The Royal Hallamshire Hospital, Sheffield Teaching Hospital NHS Foundation Trust
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:
citation: Forsyth S, Schroeder P, Geib J, Vrishabhendra L, Konstantinidis DG, LaSalvia K, Ribadeneira MD, Wu E, Kelly P, Kalfa TA. Safety, Pharmacokinetics, and Pharmacodynamics of Etavopivat (FT-4202), an Allosteric Activator of Pyruvate Kinase-R, in Healthy Adults: A Randomized, Placebo-Controlled, Double-Blind, First-in-Human Phase 1 Trial. Clin Pharmacol Drug Dev. 2022 May;11(5):654-665. doi: 10.1002/cpdd.1058. Epub 2022 Jan 12.