NCT04905407 is a Phase 2 clinical trial of Tamibarotene in Acute Myeloid Leukemia, sponsored by Syros Pharmaceuticals.

Who is sponsoring NCT04905407?

NCT04905407 is sponsored by Syros Pharmaceuticals.

What drugs are being tested in NCT04905407?

Interventions in NCT04905407: Tamibarotene, Venetoclax, Azacitidine.

What condition does NCT04905407 study?

NCT04905407 studies Acute Myeloid Leukemia.

Is NCT04905407 still recruiting?

NCT04905407 is currently terminated. Last updated 24 February 2025.

Are results published for NCT04905407?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2025-02-24 · How we verify

NCT04905407

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Tamibarotene Plus Venetoclax/Azacitidine in Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

Terminated Phase 2 Results posted Last updated 24 February 2025

What this trial tests

Phase 2 trial testing Tamibarotene in Acute Myeloid Leukemia in 66 participants. Terminated before completion.

Timeline

26 August 2021

Primary endpoint
12 August 2024

12 August 2024

Quick facts

Lead sponsor	Syros Pharmaceuticals
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	66
Start date	26 August 2021
Primary completion	12 August 2024
Estimated completion	12 August 2024
Sites	28 locations across France, United States

Drugs / interventions tested

Tamibarotene — full drug profile →
Venetoclax (venetoclax) — full drug profile →
Azacitidine (azacitidine) — full drug profile →

Conditions studied

Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

Syros Pharmaceuticals — full company profile →

Who can join

18 and older, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs) Primary · Up to 3 years

An adverse event (AE) was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. TEAEs were defined as those adverse events AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study up until the last dose of study treatment plus

Group	Value	95% CI
Part 1: Tamibarotene/Venetoclax/Azacitidine	10

Part 2: Complete Remission/Complete Remission With Incomplete Hematologic Recovery (CR/CRi) Rate Primary · Up to 3 years

CR/CRi rate was estimated by the percentage of participants who achieved complete Remission (CR) and/or CR with incomplete hematologic recovery (CRi),CR/CRi (as determined by the investigator). CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.CRi was defined as absolute neutrophil count \<1,000/µL, platelets count \<100,000/µL, bone marrow blasts \<5% and all CR criteria met except either neutrophils or platelets not recovered.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	60.00	38.7 – 78.9
Part 2: Venetoclax/Azacitidine	69.23	48.2 – 85.7

Part 1: Overall Response Rate (ORR) Secondary · Up to 3 years

ORR: percentage of participants who achieved a overall response as comprised of CR(ANC ≥1,000/µL, platelets count (PC) ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods;absence of extramedullary disease),CRi (ANC \<1,000/µL, PC \<100,000/µL, bone marrow blasts \<5% and all CR criteria met except either neutrophils or platelets not recovered), CRh (ANC \>500/µL, PC \>500,000/µL, bone marrow blasts \<5% and all CR criteria met except ANC and platelets with partial recovery of peripheral counts), morphologically leukemia-free state (bone marrow blast

Group	Value	95% CI
Part 1: Tamibarotene/Venetoclax/Azacitidine	77.78	40.0 – 97.2

Part 2: Number of Participants With Treatment Emergent Adverse Events Secondary · Up to 3 years

An AE was defined as any untoward medical occurrence that developed or worsened in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. A TEAE was any untoward medical occurrence associated with use of a study drug/study participation, whether or not considered related to study drug after first dose. TEAEs were defined as those adverse events AEs with onset after the first dose of study treatment or existing events that worsened after the first dose during the study up until the last dose of study treatment plus 30 days. A summa

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	24
Part 2: Venetoclax/Azacitidine	24

Part 2: Complete Remission (CR) Rate Secondary · Up to 3 years

CR rate was estimated by the percentage of participants who achieved complete remission (as determined by the investigator). CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	48.00	27.8 – 68.7
Part 2: Venetoclax/Azacitidine	57.69	36.9 – 76.6

Part 2: Complete Remission/ Complete Remission With Partial Hematologic Recovery (CR/CRh) Rate Secondary · Up to 3 years

CR/CRh rate was estimated by the percentage of participants who achieved complete Remission (CR) and/or CR with partial hematologic recovery (CRh),CR/CRh (as determined by the investigator). CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.CRh was defined as absolute neutrophil count \>500/µL, platelets count \>500,000/µL, bone marrow blasts \<5% and all CR criteria met except ANC and platelets with partial recovery of peripheral counts.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	48.00	32.2 – 75.6
Part 2: Venetoclax/Azacitidine	57.69	42.7 – 83.6

Part 2: Duration of Complete Remission Secondary · Up to 3 years

Duration of CR was defined as duration from the date of first documented evidence of CR to the date of relapse of disease (as determined by the investigator), or death due to any cause, whichever occurred first. CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	293.0	66.0 – NA
Part 2: Venetoclax/Azacitidine	256.5	56.0 – NA

Part 2: Duration of CR/CRi Secondary · Up to 3 years

Duration of CR/CRi was defined as duration from the date of first documented evidence of CR/CRi to the date of relapse of disease (as determined by the investigator), or death due to any cause, whichever occured first.CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.CRi was defined as absolute neutrophil count \<1,000/µL, platelets count \<100,000/µL, bone marrow blasts \<5% and all CR criteria met except either neutrophils or platelets not r

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	293.0	66.0 – NA
Part 2: Venetoclax/Azacitidine	253.0	56.0 – 263.0

Part 2: Duration of CR/CRh Secondary · Up to 3 years

Duration of CR/CRh was defined as duration from the date of first documented evidence of CR/CRh to the date of relapse of disease (as determined by the investigator), or death due to any cause, whichever occured first. CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease. CRh was defined as absolute neutrophil count \>500/µL, platelets count \>500,000/µL, bone marrow blasts \<5% and all CR criteria met except ANC and platelets with partial recove

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	293.0	66.0 – NA
Part 2: Venetoclax/Azacitidine	256.5	56.0 – NA

Part 2: Time to Complete Response Secondary · Up to 3 years

Time to CR was defined as the duration from the date of Cycle 1 Day 1 visit to the date of the first documented evidence of CR as determined by the investigator. CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	21.0	16.0 – 25.0
Part 2: Venetoclax/Azacitidine	25.0	19.0 – 43.0

Part 2: Time to CR/CRi Secondary · Up to 3 years

Time to CR/CRi was defined as the duration from the date of Cycle 1 Day 1 Visit to the date of the first documented evidence of CR/CRi as determined by the investigator. CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease.CRi was defined as absolute neutrophil count \<1,000/µL, platelets count \<100,000/µL, bone marrow blasts \<5% and all CR criteria met except either neutrophils or platelets not recovered.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	21.0	16.0 – 28.0
Part 2: Venetoclax/Azacitidine	24.5	19.0 – 36.0

Part 2: Time to CR/CRh Secondary · Up to 3 years

Time to CR/CRh was defined as the duration from the date of Cycle 1 Day 1 Visit to the date of the first documented evidence of CR/CRh as determined by the investigator. CR was defined as absolute neutrophil count ≥1,000/µL, platelets count ≥100,000/µL, bone marrow blasts \<5% and absence of circulating blasts and blasts with auer rods; absence of extramedullary disease. CRh was defined as absolute neutrophil count \>500/µL, platelets count \>500,000/µL, bone marrow blasts \<5% and all CR criteria met except ANC and platelets with partial recovery of peripheral counts.

Group	Value	95% CI
Part 2: Tamibarotene/Venetoclax/Azacitidine	21.0	16.0 – 25.0
Part 2: Venetoclax/Azacitidine	25.0	19.0 – 43.0

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 3 years. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Part 1: Tamibarotene/Venetoclax/Azacitidine

Serious: 10/10 (100%)

Deaths: 7/10

Part 2: Tamibarotene/Venetoclax/Azacitidine

Serious: 15/24 (63%)

Deaths: 7/24

Part 2: Venetoclax/Azacitidine

Serious: 13/27 (48%)

Deaths: 7/27

Part 3: Tamibarotene/Venetoclax/Azacitidine

Serious: 4/5 (80%)

Deaths: 2/5

Serious adverse events (73 terms)

Reaction	System	Part 1: Tamibarotene/Venet…	Part 2: Tamibarotene/Venet…	Part 2: Venetoclax/Azaciti…	Part 3: Tamibarotene/Venet…
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—	—
Atrial fibrillation	Cardiac disorders	—	—	—	—
Asthenia	General disorders	—	—	—	—
Pyrexia	General disorders	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—	—
Muscular weakness	Musculoskeletal and connective tissue disorders	—	—	—	—
Acute kidney injury	Renal and urinary disorders	—	—	—	—
Febrile bone marrow aplasia	Blood and lymphatic system disorders	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—
Febrile bone marrow aplasia	Blood and lymphatic system disorders	—	—	—	—
Leukopenia	Blood and lymphatic system disorders	—	—	—	—
Cardiac failure	Cardiac disorders	—	—	—	—
Myocardial infarction	Cardiac disorders	—	—	—	—
Anal incontinence	Gastrointestinal disorders	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—
Upper gastrointestinal haemorrhage	Gastrointestinal disorders	—	—	—	—
Volvulus	Gastrointestinal disorders	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—
Fatigue	General disorders	—	—	—	—
General physical health deterioration	General disorders	—	—	—	—

Other adverse events (269 terms — click to expand)

Reaction	System	Part 1: Tamibarotene/Venet…	Part 2: Tamibarotene/Venet…	Part 2: Venetoclax/Azaciti…	Part 3: Tamibarotene/Venet…
Constipation	Gastrointestinal disorders	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—
Neutropenia	Blood and lymphatic system disorders	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Oedema peripheral	General disorders	—	—	—	—
Weight decreased	Investigations	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—	—
Platelet count decreased	Investigations	—	—	—	—
White blood cell count decreased	Investigations	—	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—	—
Asthenia	General disorders	—	—	—	—
Fatigue	General disorders	—	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—	—
Pyrexia	General disorders	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—
Anxiety	Psychiatric disorders	—	—	—	—
Dysgeusia	Nervous system disorders	—	—	—	—
Dysphagia	Gastrointestinal disorders	—	—	—	—
Gastrooesophageal reflux disease	Gastrointestinal disorders	—	—	—	—
Headache	Nervous system disorders	—	—	—	—
Hypocalcaemia	Metabolism and nutrition disorders	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—	—	—
Pericardial effusion	Cardiac disorders	—	—	—	—
Stomatitis	Gastrointestinal disorders	—	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—	—
Weight increased	Investigations	—	—	—	—
COVID-19	Infections and infestations	—	—	—	—
Confusional state	Psychiatric disorders	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—

Most-reported serious reactions: Febrile neutropenia, Neutropenia, Atrial fibrillation, Asthenia, Pyrexia, Pneumonia, Sepsis, Fall.

Data from ClinicalTrials.gov NCT04905407 adverse events section.

Sponsor's own description

Tamibarotene is being studied as a treatment for participants with a type of leukemia called acute myeloid leukemia, or AML for short. Tamibarotene is being studied as a treatment for participants with AML whose cancer has a specific genetic abnormality characterized by the overexpression of the retinoic acid receptor alpha (RARA) gene. This genetic profile is found in about 3 of every 10 people with AML. During the trial, tamibarotene will be given with 2 other drugs that are already used together to treat people who have AML and who cannot start treatment with standard chemotherapy.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Recent advances in targeted therapies in acute myeloid leukemia.
Bhansali RS, Pratz KW, Lai C. · · 2023 · cited 151× · PMID 36966300 · DOI 10.1186/s13045-023-01424-6
An Overview of Targeted Therapies in Acute Myeloid Leukemia.
Turkalj S, Radtke FA, Vyas P. · · 2023 · cited 29× · PMID 37304938 · DOI 10.1097/hs9.0000000000000914
The Promise of Retinoids in the Treatment of Cancer: Neither Burnt Out Nor Fading Away.
Nagai Y, Ambinder AJ. · · 2023 · cited 23× · PMID 37509198 · DOI 10.3390/cancers15143535
Venetoclax: A New Partner in the Novel Treatment Era for Acute Myeloid Leukemia and Myelodysplastic Syndrome.
El-Cheikh J, Bidaoui G, Saleh M, Moukalled N, et al · · 2023 · cited 22× · PMID 37071328 · DOI 10.1007/s44228-023-00041-x
How We Incorporate Venetoclax in Treatment Regimens for Acute Myeloid Leukemia.
Maiti A, Konopleva MY. · · 2022 · cited 19× · PMID 35072368 · DOI 10.1097/ppo.0000000000000567
Building on Foundations: Venetoclax-Based Combinations in the Treatment of Acute Myeloid Leukemia.
Oyogoa E, Traer E, Tyner J, Lachowiez C. · · 2023 · cited 16× · PMID 37509251 · DOI 10.3390/cancers15143589
The multi-CDK inhibitor dinaciclib reverses bromo- and extra-terminal domain (BET) inhibitor resistance in acute myeloid leukemia via inhibition of Wnt/β-catenin signaling.
Marr AR, Halpin M, Corbin DL, Asemelash Y, et al · · 2024 · cited 10× · PMID 38438856 · DOI 10.1186/s40164-024-00483-w
Precision and strategic targeting of novel mutation-specific vulnerabilities in acute myeloid leukemia: the semi-centennial of 7 + 3.
Patel SA. · · 2023 · cited 8× · PMID 37328939 · DOI 10.1080/10428194.2023.2224473

Verify or expand the search:

Other trials of Tamibarotene

Trials testing the same drug.

NCT06085638 — Phase I/II Study of SY-1425 (Tamibarotene) in Combination With Azacitidine and Venetoclax for Patients With Chronic Myel · Phase 1, PHASE2 · withdrawn
NCT04797780 — Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome · Phase 3 · terminated
NCT02807558 — A Biomarker-Directed Phase 2 Trial of Tamibarotene (SY-1425) in Participants With Acute Myeloid Leukemia or Myelodysplas · Phase 2 · completed

Other recruiting trials for Acute Myeloid Leukemia

Currently open trials in the same condition.

NCT07020533 — A Vaccine (CMV-MVA Triplex Vaccine) for the Enhancement of CMV-Specific Immunity and the Prevention of CMV Viremia in Pa · Phase 1 · recruiting
NCT06782542 — Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for I · Phase 2 · recruiting
NCT07177079 — High-dose Ascorbate (HDA) in Combination With Standard of Care Azacitidine and Venetoclax in Acute Myeloid Leukemia (AML · Phase 1 · recruiting
NCT07384715 — First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelod · Phase 1 · recruiting
NCT07107126 — Safety and Proof-of-Concept Study of RPT1G in Adults With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes · Phase 1 · recruiting

Other Syros Pharmaceuticals trials

Trials by the same sponsor.

NCT04797780 — Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome · Phase 3 · terminated
NCT04247126 — A Study of SY 5609, a Selective CDK7 Inhibitor, in Advanced Solid Tumors · Phase 1 · completed
NCT03134638 — A Study of SY-1365 in Adult Patients With Advanced Solid Tumors · Phase 1 · terminated
NCT02807558 — A Biomarker-Directed Phase 2 Trial of Tamibarotene (SY-1425) in Participants With Acute Myeloid Leukemia or Myelodysplas · Phase 2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04905407 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Syros Pharmaceuticals
Last refreshed: 24 February 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04905407.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing