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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04797780
Other study ID # SY-1425-301
Secondary ID 2020-004528-40
Status Recruiting
Phase Phase 3
First received
Last updated
Start date February 8, 2021
Est. completion date February 8, 2029

Study information

Verified date March 2024
Source Syros Pharmaceuticals
Contact Clinical Trial Manager
Phone 617-674-9069
Email SELECTMDS1@syros.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study compares the efficacy of Tamibarotene in combination with azacitidine to azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha (RARA) positive, and newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS), and who have not received treatment for this diagnosis. The primary goal of the study is to compare the complete remission rate between the two treatment arms.


Description:

A subset of participants have MDS characterized by an overexpression of the RARA gene. A blood test will be used to identify participants with RARA-positive MDS. Assessment of the RARA biomarker for study eligibility will be done by collection of blood samples from potential study participants at the pre-screening visit and testing at a central laboratory. Participants who meet eligibility requirements will be randomized 2:1 to receive either Tamibarotene plus azacitidine or placebo plus azacitidine.


Recruitment information / eligibility

Status Recruiting
Enrollment 550
Est. completion date February 8, 2029
Est. primary completion date November 15, 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Key Inclusion Criteria: - Participants must be RARA-positive based on the investigational assay. - Participants must be newly diagnosed with HR-MDS as follows: - Diagnosis of MDS according to the World Health Organization (WHO) classification and classified by the Revised International Prognostic Scoring System (IPSS R) risk category as very high, high, or intermediate risk. - Participants must have Eastern Cooperative Oncology Group (ECOG) Performance Status of =2. Key Exclusion Criteria: - Participants are suitable for and agree to undergo allogeneic hematopoietic stem cell transplant (HSCT) at the time of screening. - Participants who need treatment prior to stem cell transplant can receive treatment on this study and stop the study treatment when they are ready to proceed to transplant. - Participants who received prior treatment for MDS with any hypomethylating agent, lenalidomide, chemotherapy or allogeneic HSCT.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Tamibarotene
Administered as specified in the treatment arm
Placebo
Administered as specified in the treatment arm
Azacitidine
Administered as specified in the treatment arm

Locations

Country Name City State
Austria Medical University of Graz Graz
Austria Salzburg Cancer Research Institute (SCRI) Salzburg
Austria Klinik Hietzing Vienna
Belgium ZNA Middelheim Antwerp
Belgium Institut Jules Bordet Brussels
Belgium Ghent University Hospital Gent
Belgium Hospital De Jolimont La Louvière
Belgium CHU de Liège Liège
Belgium AZ Delta Roeselare
Belgium CHU-UCL Namur Yvoir
Canada Tom Baker Cancer Centre Calgary
Canada Juravinski Cancer Centre Hamilton Ontario
Canada London Health Sciences Center London Ontario
Canada London Health Sciences Centre London
Canada McGill University Health Centre (MUHC) Montréal Quebec
Canada Princess Margaret Cancer Centre - University Health Network Toronto Ontario
Canada CancerCare Manitoba Winnipeg Manitoba
Czechia Fakultni nemocnice Olomouc (University Hospital Olomouc) Olomouc
Czechia University Hospital Ostrava Ostrava
Czechia Nemocnice Pelhrimov Pelhrimov
Czechia General University Hospital Prague Prague
France CHU Amiens-Picardie Amiens
France CHU d'Angers Angers
France Centre Hospitalier de la Côte Basque Hématologie Clinique Bayonne
France Hôpital Avicenne Bobigny
France CHRU de BREST - Hôpital Morvan Brest
France CHU Clermont Ferrand / CHU Estaing Clermont-Ferrand
France CHU Grenoble Alpes Hôpital Nord Michallon La Tronche
France Hopital Mignot Le Chesnay
France CHU de Limoges Limoges
France Centre Léon Bérard Lyon
France CHU la Conception Marseille
France CHU Nantes - Hotel Dieu Nantes
France CHU de Nice - Hopital L'Archet Nice
France Hôpital Saint Louis Paris
France Centre Hospitalier de Perpignan Perpignan
France CHU Bordeaux Pessac
France CHU Lyon Sud Pierre-Bénite
France CHU Poiters Poitiers
France CHU Saint Etienne Saint-Priest-en-Jarez
France ICANS Strasbourg Strasbourg
France Institut Gustave Roussy Villejuif
Germany Universitätsklinikum Augsburg Augsburg
Germany Helios Klinikum Berlin Buch GmbH Berlin
Germany Universitätsklinikum Freiburg Freiburg
Germany Universitätsmedizin Göttingen Göttingen
Germany Universitätsklinikum Halle (Saale) Halle
Germany Universitätsklinikum Marburg Marburg
Germany Universitätsklinikum Münster Münster
Hungary Semmelweis Egyetem Budapest
Hungary Debrecen University Medical and Health Science Centre Debrecen
Hungary Markhot Ferenc Oktatókórház és Rendelointézet, Belgyógyászati- és Infektológiai Centrum Eger
Hungary Petz Aladar County Teaching Hospital Gyor
Hungary Josa Andras Teaching Hospital Nyíregyháza
Israel Barzilai Medical Center of Ashkelon Ashkelon
Israel Bnai Zion Medical center Haifa
Israel Carmel Medical Center Haifa
Israel Shaare Zedek Medical Center Jerusalem
Israel Sheba Medical Center Ramat Gan
Israel Kaplan Medical Center Rehovot
Israel Tel Aviv Sourasky Medical Center Tel Aviv
Italy IRCCS Azienda Ospedaliero-Universitaria di Bologna Bologna
Italy ASST Spedali Civili di Brescia Brescia
Italy Azienda Ospedaliero-Universitaria di Ferrara Ferrara
Italy AOU Careggi Firenze
Italy Ospedale Policlinico San Martino Genova
Italy Romagnolo Institute to Study Tumors "Dino Amadori "/ Meldona Meldola
Italy A.O.U. Maggiore della Carità Novara
Italy Ospedali Marche Nord Centro Ematologia e Trapianti Pesaro
Italy Ospedale S.Maria delle Croci Ravenna
Italy Ospedale Infermi di Rimini Rimini
Italy Ospedale S. Eugenio - ASL Roma 2 Roma
Italy Fondazione Policlinico Tor Vergata Rome
Italy AOUI di Verona Verona
Poland University Clinic Gdansk Gdansk
Poland Pomeranian Hospital Gdynia Gdynia
Poland Wojewodzki Szpital Specjalistyczny w Legnicy Legnica
Poland Alfred Sokolowski Hospital Walbrzych Walbrzych
Poland Instytut Hematologii i Transfuzjologii, Klinika Hematologii Warszawa
Poland University Clinic Warsaw Warszawa
Spain Institut Catala d'Oncologia - Hospital Duran i Reynals Location Badalona
Spain Fundación Privada Instituto de Investigación Oncológica Vall d'Hebron Barcelona
Spain H. Santa Creu i Sant Pau Barcelona
Spain Hospital de la Santa Creu i Sant Pau Barcelona
Spain Hospital del Mar Barcelona
Spain Hospital del Mar Secretaria d'Hematologia Clínica Barcelona
Spain Hospital Universitario de Burgos Burgos
Spain Hospital San Pedro de Alcántara Cáceres
Spain H. Universitario La Paz Madrid
Spain Hospital Universitario de La Princesa Madrid
Spain Hospital Universitario Fundacion Jimenez Diaz Madrid
Spain MD Anderson Cancer Center Madrid
Spain Hospital General Universitario J.M. Morales Meseguer Murcia
Spain Hospital Universitario Central de Asturia Oviedo
Spain Hospital Son Llatzer Palma De Mallorca
Spain Clinica Universidad Navarra Pamplona
Spain Hospital Universitario de Salamanca Salamanca
Spain Hospital Universitario Donostia San Sebastián
Spain Hospital Universitario Marqués de Valdecilla Santander
Spain Hospital Universitario Virgen Del Rocio Seville
Spain Hospital Clinico Universitario de Valencia Valencia
Spain Hospital Universitario y Politécnico La Fe Valencia
United Kingdom Castle Hill Hospital, Cottingham Cottingham
United Kingdom Ninewells Hospital Dundee
United Kingdom Western General Hospital in NHS Lothian Edinburgh Scotland Edinburgh
United Kingdom Clatterbridge Cancer Centre NHS Foundation Trust Liverpool
United Kingdom King's College Hospital NHS Foundation Trust London
United Kingdom The Royal Marsden Foundation Trust London
United Kingdom University Hospital Southampton NHS Foundation Trust Southampton
United Kingdom Sandwell and West Birmingham Hospital NHS Trust West Bromwich
United States Johns Hopkins University Baltimore Maryland
United States American Oncology Partners of Maryland, PA Bethesda Maryland
United States SCL Health Research Institute, Inc. (St. Vincent Frontier Cancer Center) Billings Montana
United States Montefiore Medical Center & Albert Einstein College of Medicine Bronx New York
United States Novant Health Inc Charlotte North Carolina
United States University of Illinois Cancer Center Chicago Illinois
United States Cleveland Clinic Cleveland Ohio
United States The Ohio State University Comprehensive Cancer Center Columbus Ohio
United States University of Iowa Iowa City Iowa
United States UC Irvine Irvine California
United States Cancer specialists of North Florida Jacksonville Florida
United States Northwell Health Lake Success New York
United States University of California, Los Angelas Los Angeles California
United States University of Southern California Los Angeles California
United States Morristown Medical Center- Atlantic Hematology Oncology Morristown New Jersey
United States TriStar - Sarah Cannon BMT Nashville Tennessee
United States Yale Cancer Center New Haven Connecticut
United States Tulane Cancer Center New Orleans Louisiana
United States Oregon Health and Science University Portland Oregon
United States Comprehensive Hematology and Oncology Saint Petersburg Florida
United States University of Utah Huntsman Cancer Institute Salt Lake City Utah
United States Swedish Cancer Institute Seattle Washington
United States Orchard Healthcare Research Inc. Skokie Illinois
United States Moffitt Cancer Center Tampa Florida
United States University of Arizona Tucson Arizona
United States Novant Health Cancer Institute - Forsyth Winston-Salem North Carolina
United States Wake Forest Baptist Health Winston-Salem North Carolina

Sponsors (1)

Lead Sponsor Collaborator
Syros Pharmaceuticals

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Canada,  Czechia,  France,  Germany,  Hungary,  Israel,  Italy,  Poland,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportion of Participants with Complete Remission Up to 5 Years
Secondary Duration of Overall Survival Up to 5 Years
Secondary Proportion of Participants Who Achieve Transfusion Independence Up to 5 Years
Secondary Proportion of Participants Who Achieve Objective Response Up to 5 Years
Secondary Duration of Complete Response Up to 5 Years
Secondary Duration of Overall Response Up to 5 Years
Secondary Time to Complete Remission Up to 5 Years
Secondary Time to Initial Response Up to 5 Years
Secondary Duration of Event Free Survival Up to 5 Years
Secondary Change in Health-Related Quality of Life (HRQOL) as assessed by the European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire 30 Scale (EORTC QLQ-30) Up to 5 Years
Secondary Change in HRQOL as assessed by the European Quality of Life 5 dimensions Scale (EuroQoL-5D) Up to 5 Years
Secondary Proportion of participants with Adverse Events and Serious Adverse Events Up to 5 Years
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