Study Stopped
As per Sponsor decision, the study was terminated.
Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome
A Randomized, Double-blind, Placebo-controlled Phase 3 Study of Tamibarotene Plus Azacitidine Versus Placebo Plus Azacitidine in Newly Diagnosed, Adult Patients Selected for RARA-positive Higher-risk Myelodysplastic Syndrome (SELECT MDS-1)
2 other identifiers
interventional
246
13 countries
128
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Feb 2021
Typical duration for phase_3
128 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 8, 2021
CompletedFirst Submitted
Initial submission to the registry
March 11, 2021
CompletedFirst Posted
Study publicly available on registry
March 15, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 13, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
November 13, 2024
CompletedResults Posted
Study results publicly available
April 10, 2025
CompletedApril 10, 2025
April 1, 2025
3.8 years
March 11, 2021
January 2, 2025
April 7, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Complete Remission (CR)
CR was determined by the investigator per the modified International Working Group Myelodysplastic Syndrome (IWG MDS). CR was defined as participants with hemoglobin ≥11 grams/deciliter (g/dL), neutrophils ≥1.0\*10\^9/L, platelets ≥100\*10\^9/L, blasts 0%, bone marrow blasts (BMBs) ≤5% and normal maturation of all cell lines, persistent dysplasia were noted.
Up to 45 months
Secondary Outcomes (9)
Duration of Overall Response (DOR)
Up to 45 months
Number of Participants Who Achieved Transfusion Independence (TI)
Up to 45 months
Percentage of Participants Who Achieved Overall Response (OR)
Up to 45 months
Duration of Complete Response (DOCR)
Up to 45 months
Time to Complete Remission (TCR)
Up to 45 months
- +4 more secondary outcomes
Study Arms (2)
Tamibarotene + Azacitidine
EXPERIMENTALTamibarotene: 6 mg administered orally twice per day (BID) on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m\^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.
Tamibarotene Matched Placebo + Azacitidine
PLACEBO COMPARATORPlacebo: Tamibarotene-matching tablets administered orally BID on Days 8 through 28 of each 28-day treatment cycle. Azacitidine: 75 mg/m\^2 administered intravenously or subcutaneously each day on Days 1 through 7 of each 28-day treatment cycle.
Interventions
Administered as specified in the treatment arm
Administered as specified in the treatment arm
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (128)
University of Arizona
Tucson, Arizona, 85719, United States
University of Southern California
Los Angeles, California, 90033, United States
University of California, Los Angelas
Los Angeles, California, 90095, United States
Yale Cancer Center
New Haven, Connecticut, 06510, United States
Comprehensive Hematology and Oncology
St. Petersburg, Florida, 33709, United States
Moffitt Cancer Center
Tampa, Florida, 33612, United States
University of Illinois Cancer Center
Chicago, Illinois, 60612, United States
Orchard Healthcare Research Inc.
Skokie, Illinois, 60077, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Tulane Cancer Center
New Orleans, Louisiana, 70112, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
American Oncology Partners of Maryland, PA
Bethesda, Maryland, 20817, United States
Morristown Medical Center- Atlantic Hematology Oncology
Morristown, New Jersey, 02140, United States
Northwell Health
Lake Success, New York, 11042, United States
Novant Health Inc
Charlotte, North Carolina, 28204, United States
Novant Health Cancer Institute - Forsyth
Winston-Salem, North Carolina, 27103, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, 27157, United States
The Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
TriStar - Sarah Cannon BMT
Nashville, Tennessee, 37203, United States
University of Utah Huntsman Cancer Institute
Salt Lake City, Utah, 84112, United States
Swedish Cancer Institute
Seattle, Washington, 98104, United States
Medical University of Graz
Graz, 8036, Austria
Salzburg Cancer Research Institute (SCRI)
Salzburg, 5020, Austria
Klinik Hietzing
Vienna, Austria
ZNA Middelheim
Antwerp, Belgium
Institut Jules Bordet
Brussels, Belgium
Ghent University Hospital
Ghent, Belgium
Hospital De Jolimont
La Louvière, 7100, Belgium
CHU de Liège
Liège, Belgium
AZ Delta
Roeselare, 8800, Belgium
CHU-UCL Namur
Yvoir, 5530, Belgium
CancerCare Manitoba
Winnipeg, Manitoba, Canada
Juravinski Cancer Centre
Hamilton, Ontario, ON L8V 1C3, Canada
London Health Sciences Center
London, Ontario, N6A 5W9, Canada
Princess Margaret Cancer Centre - University Health Network
Toronto, Ontario, Canada
McGill University Health Centre (MUHC)
Montreal, Quebec, H4A 3J1, Canada
Tom Baker Cancer Centre
Calgary, Canada
London Health Sciences Centre
London, ON, Canada
Fakultni nemocnice Olomouc (University Hospital Olomouc)
Olomouc, Czechia
University Hospital Ostrava
Ostrava, Czechia
General University Hospital Prague
Prague, Czechia
CHU Amiens-Picardie
Amiens, 80054, France
CHU d'Angers
Angers, 49933, France
Centre Hospitalier de la Côte Basque Hématologie Clinique
Bayonne, 64109, France
Hôpital Avicenne
Bobigny, 93000, France
CHRU de BREST - Hôpital Morvan
Brest, 29200, France
CHU Clermont Ferrand / CHU Estaing
Clermont-Ferrand, France
CHU Grenoble Alpes Hôpital Nord Michallon
La Tronche, France
Hopital Mignot
Le Chesnay, 78150, France
CHU de Limoges
Limoges, 87000, France
Centre Léon Bérard
Lyon, 69373, France
CHU la Conception
Marseille, France
CHU Nantes - Hotel Dieu
Nantes, 44093, France
CHU de Nice - Hopital L'Archet
Nice, 06200, France
Hôpital Saint Louis
Paris, 75475, France
CHU Bordeaux
Pessac, 33604, France
CHU Lyon Sud
Pierre-Bénite, 69495, France
CHU Poiters
Poitiers, 86021, France
CHU Saint Etienne
Saint-Priest-en-Jarez, France
ICANS Strasbourg
Strasbourg, 67033, France
Institut Gustave Roussy
Villejuif, 94805, France
Universitätsklinikum Augsburg
Augsburg, Germany
Helios Klinikum Berlin Buch GmbH
Berlin, Germany
Universitätsklinikum Freiburg
Freiburg im Breisgau, Germany
Universitätsmedizin Göttingen
Göttingen, Germany
Universitätsklinikum Halle (Saale)
Halle, Germany
Universitätsklinikum Marburg
Marburg, Germany
Universitätsklinikum Münster
Münster, Germany
Semmelweis Egyetem
Budapest, 1083, Hungary
Debrecen University Medical and Health Science Centre
Debrecen, H-4012, Hungary
Markhot Ferenc Oktatókórház és Rendelőintézet, Belgyógyászati- és Infektológiai Centrum
Eger, Hungary
Petz Aladar County Teaching Hospital
Győr, Hungary
Josa Andras Teaching Hospital
Nyíregyháza, 4400, Hungary
Barzilai Medical Center of Ashkelon
Ashkelon, Israel
Bnai Zion Medical center
Haifa, 31048, Israel
Shaare Zedek Medical Center
Jerusalem, 9103102, Israel
Sheba Medical Center
Ramat Gan, Israel
Kaplan Medical Center
Rehovot, 7661041, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, Israel
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Bologna, 40138, Italy
ASST Spedali Civili di Brescia
Brescia, Italy
Azienda Ospedaliero-Universitaria di Ferrara
Ferrara, 44124, Italy
AOU Careggi
Florence, Italy
Ospedale Policlinico San Martino
Genova, 16132, Italy
Romagnolo Institute to Study Tumors "Dino Amadori "/ Meldona
Meldola, 47014, Italy
A.O.U. Maggiore della Carità
Novara, 28100, Italy
Ospedali Marche Nord Centro Ematologia e Trapianti
Pesaro, 61122, Italy
Ospedale S.Maria delle Croci
Ravenna, Italy
Ospedale S. Eugenio - ASL Roma 2
Roma, Italy
Fondazione Policlinico Tor Vergata
Rome, 00133, Italy
AOUI di Verona
Verona, Italy
University Clinic Gdansk
Gdansk, 80-952, Poland
Pomeranian Hospital Gdynia
Gdynia, 81-519, Poland
Wojewodzki Szpital Specjalistyczny w Legnicy
Legnica, 59-220, Poland
Instytut Hematologii i Transfuzjologii, Klinika Hematologii
Warsaw, Poland
University Clinic Warsaw
Warsaw, Poland
Alfred Sokołowski Hospital Wałbrzych
Wałbrzych, Poland
Institut Catala d'Oncologia - Hospital Duran i Reynals Location
Badalona, Spain
Hospital del Mar
Barcelona, 08003, Spain
H. Santa Creu i Sant Pau
Barcelona, 08025, Spain
Fundación Privada Instituto de Investigación Oncológica Vall d'Hebron
Barcelona, 08035, Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, 08041, Spain
Hospital del Mar Secretaria d'Hematologia Clínica
Barcelona, Spain
Hospital Universitario de Burgos
Burgos, 09006, Spain
Hospital San Pedro de Alcántara
Cáceres, Spain
MD Anderson Cancer Center
Madrid, 28033, Spain
Hospital Universitario Fundacion Jimenez Diaz
Madrid, 28040, Spain
H. Universitario La Paz
Madrid, 28046, Spain
Hospital Universitario de La Princesa
Madrid, Spain
Hospital General Universitario J.M. Morales Meseguer
Murcia, Spain
Hospital Universitario Central de Asturia
Oviedo, 33011, Spain
Hospital Son Llatzer
Palma de Mallorca, 07198, Spain
Clinica Universidad Navarra
Pamplona, 31008, Spain
Hospital Universitario de Salamanca
Salamanca, 37007, Spain
Hospital Universitario Donostia
San Sebastián, Spain
Hospital Universitario Marqués de Valdecilla
Santander, 39008, Spain
Hospital Universitario Virgen Del Rocio
Seville, 41013, Spain
Hospital Universitario y Politécnico La Fe
Valencia, 46026, Spain
Hospital Clinico Universitario de Valencia
Valencia, Spain
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 Kingdom
Related Publications (2)
DeZern AE, Thepot S, de Botton S, Patriarca A, Deeren D, Torregrossa-Diaz JM, Marconi G, Bernal T, Bergua Burgues J, Xicoy B, Jonasova A, Zeidan AM, Dimicoli-Salazar S, Simand C, Valcarcel D, Diez Campelo M, Chai-Ho W, Saini L, Garnier A, Geissler K, Ofran Y, Nagy Z, Krishnamurthy P, Lubbert M, Basak G, Carraway HE, Sallman DA, Borate U, Santini V, Campbell V, Fenaux P, Braun T, Lanza F, Zaucha JM, Roth DA, Paul S, Roy P, Kelly MJ, Volkert A, Chisholm J, Malak TA, Klimek VM, Cluzeau T. Pivotal results of SELECT-MDS-1 phase 3 study of tamibarotene with azacitidine in newly diagnosed higher-risk MDS. Blood Adv. 2025 Aug 26;9(16):4090-4099. doi: 10.1182/bloodadvances.2025016229.
PMID: 40334070DERIVEDStahl M, DeZern AE. Is there a path forward for immunotherapy in patients with myelodysplastic syndromes? Lancet Haematol. 2024 Jan;11(1):e5-e7. doi: 10.1016/S2352-3026(23)00343-5. Epub 2023 Dec 5. No abstract available.
PMID: 38065202DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Limitations and Caveats
As per Sponsor decision, the study was terminated. Note that the study was terminated prior to the data from the questionnaires were collected, analyzed, summarized, or made available by the study sponsor.
Results Point of Contact
- Title
- Tiffany Crowell
- Organization
- Syros Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Medical Director
Syros Pharmaceuticals Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 11, 2021
First Posted
March 15, 2021
Study Start
February 8, 2021
Primary Completion
November 13, 2024
Study Completion
November 13, 2024
Last Updated
April 10, 2025
Results First Posted
April 10, 2025
Record last verified: 2025-04
Data Sharing
- IPD Sharing
- Will not share