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Sickle Cell DiseasePhase 3HIBISCUSNCT04624659

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

A Phase 3 study testing Etavopivat Tablets Low dose, Etavopivat Tablets High dose and Etavopivat Tablets in people with Sickle Cell Disease, run by Forma Therapeutics, Inc. at 142 sites in 17 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 12 to 65 years
Conditions the study is for
Sickle Cell Disease
What is being tested?
Etavopivat Tablets Low dose, Etavopivat Tablets High dose and Etavopivat Tablets
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
450 participants
Where
142 sitesin 17 countries
Study dates
29 Jan 2021to 19 Mar 2027
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 22Show fewer
  • 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)

Trial locations and contacts

142 sites in 17 countries.

142 sites in 17 countries

United States63 sites

  • Cornerstone Research Institute

    Altamonte Springs, Florida, 32701

  • Emory University School of Medicine

    Atlanta, Georgia, 30303

  • Sonar Clinical Research

    Atlanta, Georgia, 30315

  • Children's Healthcare of Atlanta Investigational Medication Pharmacy

    Atlanta, Georgia, 30342

  • Center for Blood Disorders Augusta University

    Augusta, Georgia, 30912

  • University Of Maryland School of Medicine

    Baltimore, Maryland, 21201

  • Univ of Alabama Birmingham

    Birmingham, Alabama, 35233

  • Massachusetts General Hospital_Boston

    Boston, Massachusetts, 02114

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • NYC Health+Hospitals

    Brooklyn, New York, 11203

  • [Legal] The University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27514

  • Medical University of South Carolina

    Charleston, South Carolina, 29425

  • Levine Cancer Institute

    Charlotte, North Carolina, 28204

  • University of Illinois at Chicago

    Chicago, Illinois, 60612

  • University of Cincinnati

    Cincinnati, Ohio, 45219

  • [Legal] Children's Hospital Medical Center dba Cincinnati Children's

    Cincinnati, Ohio, 45229

  • The Ohio State University Medical Center

    Columbus, Ohio, 43203

  • iResearch Atlanta, LLC

    Decatur, Georgia, 30030

  • [Legal] Dr. Vince Clinical Research, LLC and Dr. Vince Clinical Research, P.A.

    Detroit, Michigan, 48201

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • University of Connecticut

    Farmington, Connecticut, 06030

  • University of Florida

    Gainesville, Florida, 32610

  • East Carolina University

    Greenville, North Carolina, 27858

  • Prisma Health-Upstate

    Greenville, South Carolina, 29605

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida, 33023

  • [Legal] Baylor College of Medicine

    Houston, Texas, 77030

  • UT Health University of Texas

    Houston, Texas, 77030

  • Queens Hospital Center

    Jamaica, New York, 11432-1121

  • [Legal] Woodland International Research Group, LLC

    Little Rock, Arkansas, 72211

  • [Legal] Collaborative Neuroscience Network, LLC

    Long Beach, California, 90806

  • Methodist University Hospital

    Memphis, Tennessee, 38104

  • [Legal] St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105

  • University Of Miami

    Miami, Florida, 33136

  • Advanced Pharma CR

    Miami, Florida, 33147

  • Versiti Blood Center of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Yale University

    New Haven, Connecticut, 06519

  • North Shore Long Island Jewish Medical Center

    New Hyde Park, New York, 11040

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118

  • Columbia University Medical Center_New York

    New York, New York, 10032

  • Weill Cornell Med Coll-NYPH

    New York, New York, 10065

  • Neuro-Behavioral Clinical Research

    North Canton, Ohio, 44720

  • Pacific Research Partners

    Oakland, California, 94607

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609

  • Jimmy Everest Cancer Hematology & Oncology

    Oklahoma City, Oklahoma, 73104

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68198-2168

  • University Of California Irvine

    Orange, California, 92868

  • Arnold Palmer Children's Hospital

    Orlando, Florida, 32806

  • Children's Hosptl Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Thomas Jefferson University Hospital

    Philadelphia, Pennsylvania, 19107

  • Phoenix Children's Hsptl

    Phoenix, Arizona, 85016

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213-4306

  • Rhode Island Hospital

    Providence, Rhode Island, 02903

  • Virginia Commonwealth University

    Richmond, Virginia, 23298

  • UC Davis Medical Center

    Sacramento, California, 95817

  • Washington University School of Medicine_St. Louis

    St Louis, Missouri, 63110

  • Mary Bridge Children's Health

    Tacoma, Washington, 98405

  • Jacobi Medical Center

    The Bronx, New York, 10461

  • Montefiore Medical Center

    The Bronx, New York, 10467

  • Lynn Institute of Tulsa

    Tulsa, Oklahoma, 74135

  • Children's National Health Hospital

    Washington D.C., District of Columbia, 20010

  • Howard University

    Washington D.C., District of Columbia, 20060

  • Wake Forest Baptist Health

    Winston-Salem, North Carolina, 27157

Other trials in United States

Spain10 sites

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, 15006

  • Hospital Germans Trias i Pujol

    Badalona, 8916

  • Hospital Universitario de Cruces

    Barakaldo, 48903

  • Hospital Vall d'Hebron

    Barcelona, 08035

  • Hospital Sant Joan de Déu

    Barcelona, 8950

  • Hospital Gregorio Marañón

    Madrid, 28009

  • Hospital Ramón y Cajal

    Madrid, 28034

  • Hospital Clínico San Carlos

    Madrid, 28040

  • Hospital Universitario La Paz

    Madrid, 28046

  • Hospital Universitario Virgen del Rocío

    Seville, 41013

Other trials in Spain

Turkey (Türkiye)7 sites

  • Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji

    Adana, 1130

  • Başkent Üniversitesi Adana Dr. Turgut Noyan Uygulama ve Araştırma Merkezi

    Adana, 1240

  • Adana City Training and Research Hospital

    Adana, 4522

  • Hacettepe Üniversitesi Hastanesi- Hematoloji

    Ankara, 6230

  • Ege Üniversitesi Tip Fakultesi Hastanesi - Nefroloji

    Izmir, 35040

  • Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

    Mersin, 33110

  • VM Medical Park Mersin Hastanesi- Kardiyoloji

    Mersin, 33200

Other trials in Turkey (Türkiye)

United Kingdom7 sites

  • Leicester Royal Infirmary - Haemostasis & Thrombosis Unit

    Leicester, LE1 5WW

  • University Hospital Lewisham, Lewisham and Greenwich NHS Trust

    London, SE13 6LH

  • Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital

    London, SE18 3RA

  • Kings College Hospital - Haematology

    London, SE5 9RS

  • Hammersmith Hospital - London

    London, W2 1NY

  • Churchill Hospital

    Oxford, OX3 7LE

  • Royal Hallamshire Hospital

    Sheffield, S11 8RN

Other trials in United Kingdom

Egypt6 sites

  • [Legal] Alexandria Clinical Research LLC

    Alexandria, 0

  • [Legal] Alexandria Clinical Research LLC

    Alexandria, 21131

  • Cairo University

    Cairo, 111111

  • Ain Shams University Hospital

    Cairo, 1181

  • Al Kasr AL Aini Hospital

    Cairo, 11956

  • Sydnawy University Hospital

    Zagazig, 44519

Other trials in Egypt

France6 sites

  • Ap-Hp-Hopital Henri Mondor

    Créteil, 94010

  • Hospices Civils de Lyon-Hopital Edouard Herriot

    Lyon, 69437

  • Centre Hospitalier Universitaire de Montpellier-Hopital Saint-Eloi

    Montpellier, 34295

  • GHR Mulhouse Sud-Alsace

    Mulhouse, 68100

  • Ap-Hp-Hopital Robert Debre

    Paris, 75019

  • Saint-Denis Hospital Center

    Saint-Denis, 93200

Other trials in France

Germany6 sites

  • Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie

    Berlin, 13353

  • Universitätsklinikum Frankfurt

    Frankfurt am Main, 60596

  • Universitätsklinikum Freiburg-Zentrum für Kinder- und Jugendmedizin

    Freiburg im Breisgau, 79106

  • Universitätsklinikum Hamburg-Eppendorf - University Medical Center Hamburg-Eppendorf

    Hamburg, 20246

  • Uniklinik Heidelberg - Klinik für Pädiatrische Onkologie, Hämatologie, Immunologie und Pneumologie

    Heidelberg, 69120

  • Universitätsklinikum Regensburg

    Regensburg, 93053

Other trials in Germany

Italy6 sites

  • Azienda Ospedaliero Universitaria Policlinico G. Rodolico - San Marco

    Catania, 95123

  • Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche

    Orbassano, 10043

  • Azienda Ospedale Universita Padova

    Padova, 35128

  • ARNAS Ospedali Civico Di Cristina Benfratelli - Presidio Ospedaliero "Civio e Benfratelli"

    Palermo, 90127

  • Centro per lo Studio e la Cura delle Amiloidosi Sistemiche Fondazione IRCCS Policlinico San Matteo

    Pavia, PV, 27100

  • Ospedale Pediatrico Bambino Ges

    Roma, 00165

Other trials in Italy

India5 sites

  • Victoria Hospital, Bangalore

    Bangalore, 560002

  • Srirama Chandra Bhanj Medical College & Hospital (S.C.B Medical College)

    Cutack, 753007

  • Yashoda Hospital

    Hyderabad, Telangana, 500084

  • K.J Somaiya Hospital and Research Centre

    Mumbai, Maharashtra, 400022

  • All India Institute of Medical Sciences (AIIMS)

    New Delhi, 110029

Other trials in India

Nigeria5 sites

  • University of Abuja Teaching Hospital, Gwagwalada, Abuja

    Abuja, 228

  • Barau Dikko Teaching Hospital, Kaduna

    Kaduna, 800212

  • Aminu Kano Teaching Hospital (AKTH)

    Kano, 3452

  • Lagos University Teaching Hospital, Lagos

    Lagos, 102215

  • University of Nigeria Teaching Hospital (UNTH)

    Lagos, 102215

Other trials in Nigeria

Canada4 sites

  • CHU Sainte-Justine Mother and Child University Hospital

    Montreal, Quebec, H3T 1C5

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4

  • Providence Hematolgy

    Vancouver, British Columbia, V6E 1M7

Other trials in Canada

Greece4 sites

  • Gen Hosp Athens Ippokrateio,B' Uni Clinic Internal Medicine

    Athens, 115 27

  • General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit

    Larissa, 41221

  • General University Hospital of Patras

    Pátrai, Larissa, 265 04

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642

Other trials in Greece

Kenya4 sites

  • Kombewa Clinical Research Centre

    Kisumu, 1932

  • KEMRI Kondele Children Hospital, Kisumu

    Kisumu, 40100

  • KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

    Kisumu, 40600

  • Gertrude's Children's Hospital

    Nairobi, 100

Other trials in Kenya

Lebanon3 sites

  • Master Centre for Lebanon

    Amman, 11844

  • American University of Beirut Medical center

    Beirut, 11-0236

  • Hospital Nini

    Tripoli, 113-6044

Other trials in Lebanon

Saudi Arabia3 sites

  • King Fahad Specialist Hospital-Dammam

    Dammam, 32253

  • Prince Mohammad Bin Naser Hospital

    Jizan, 82943

  • King Khaled University Hospital,King Saud Univ. Med. City

    Riyadh, 12372 6864

Other trials in Saudi Arabia

Ghana2 sites

  • Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)

    Accra, 0

  • Kintampo Health Research Centre (KHRC)

    Kintampo, Bono East, 27WF+98M

Other trials in Ghana

Oman1 site

  • Sultan Qaboos University Hospital

    Muscat, Sultanet of Oman/Muscat/Al Khoud, 123

Other trials in Oman

About this study

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)

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).

Read the 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.

Being tested
Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Placebo Tablets, Etavopivat Tablets
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
Medical subject headings
Anemia, Sickle Cell, Anemia, Hemolysis, Vaso-Occlusive Crises, Anemia, Hemolytic, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Etavopivat Tablets Low doseDrug

    Also called FT-4202

    200 mg once daily

  • Etavopivat Tablets High doseDrug

    Also called FT-4202

    400 mg once daily

  • Placebo TabletsDrug

    Also called placebo

    Placebo once daily

  • Etavopivat TabletsDrug

    Also called FT-4202

    Selected dose once daily

Study groups in NCT04624659
Study groupRoleWhat they receive
Double blind etavopivat Low DoseExperimentalDouble blind etavopivat Low Dose
Receives: Drug: Etavopivat Tablets Low dose
Double blind etavopivat High DoseExperimentalDouble blind etavopivat High Dose
Receives: Drug: Etavopivat Tablets High dose
Double blind placeboExperimentalDouble blind placebo
Receives: Drug: Placebo Tablets
Open label etavopivatExperimentalOpen label etavopivat
Receives: Drug: Etavopivat Tablets

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hemoglobin response rate

    Hemoglobin response rate at Week 24 (increase of > 1 g/dL [> 10 g/L] from baseline) during the blinded treatment period

    Measured: 24 Weeks

  2. Annualized vaso-occlusive crisis

    Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review

    Measured: 52 Weeks

Secondary outcomes (6)

  1. Hemoglobin

    Change from baseline in hemoglobin at Week 52 during the blinded treatment period

    Measured: 52 Weeks

  2. Absolute reticulocyte count

    Change in absolute reticulocyte count from baseline at Week 24 during the blinded treatment period

    Measured: 24 Weeks

  3. Indirect bilirubin

    Change in unconjugated bilirubin from baseline at Week 24 during the blinded treatment period

    Measured: 24 Weeks

  4. Lactate dehydrogenase

    Change in lactate dehydrogenase from baseline at Week 24 during the blinded treatment period

    Measured: 24 Weeks

  5. Vaso-occlusive crisis

    Time to first vaso-occlusive crisis during the blinded treatment period

    Measured: 52 Weeks

  6. Fatigue

    Change in Patient-Reported Outcome Measurement Information System (PROMIS) Fatigue Scale from baseline in adult patients at Week 52 during the blinded treatment period

    Measured: 52 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Nov 2020
  2. Study start29 Jan 2021
  3. Primary completion6 Feb 2026
  4. Study completion19 Mar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
450 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04624659
Sponsor's protocol ID
4202-HEM-301
Acronym
HIBISCUS
Status last verified
Mar 2026
First submitted
5 Nov 2020
Last updated
6 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.

    PubMed 37254256

  • 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.

    PubMed 35019238

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Etavopivat Tablets Low dose, Etavopivat Tablets High dose and Etavopivat Tablets in people with Sickle Cell Disease, run by Forma Therapeutics, Inc. at 142 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 142 sites in United States, Spain, Turkey (Türkiye), United Kingdom, Egypt and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hemoglobin response rate (time frame: 24 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04624659, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Apr 2026.

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