Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome

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.

Timeline

Start

2021-02-08

Primary completion

2024-11-13

Completion

2024-11-13

Outcome

Missed primary endpoint

Stopped (Business): “As per Sponsor decision, the study was terminated.”

paper The study did not meet the primary end point of CR, with a P value of .2084 for the treatment effect in the tamibarotene + AZA group. PMID 40334070 ↗

Publications

• 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.
• Stahl 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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Tamibarotene	Small molecule	6 mg	Oral
Background	Azacitidine	Other / unclassified	75 mg/m2	Intravenous

Indications