MEN1703 (SEL24) in Participants With Acute Myeloid Leukemia

NCT03008187 | Completed Phase 1 Results FDA Drug

• 1 other identifier: CLI24-001
• Study Type: interventional
• Target: 73
• Locations: 4 countries
• Sites: 15

Brief Summary

The purpose of the clinical trial is to identify the maximum tolerated dose of MEN1703 and to further investigate its safety profile in participants with acute myeloid leukemia (AML).

Conditions

Acute Myeloid Leukemia

Keywords

AML; Relapsed/Refractory Acute Myeloid Leukemia; IDH

Outcome Measures

Primary Outcomes (2)

• Part 1 and Part 2: Number of Participants Experiencing Treatment-emergent Adverse Events

  An adverse event (AE) was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant or clinical investigation participant administered a medicinal product and which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not considered related to the investigational medicinal product. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse events module.

  Up to 21 months

• Part 1: Number of Participants Experiencing Dose-limiting Toxicity (DLT)

  AEs were graded according to the National Cancer Institute common terminology criteria for adverse events, version 4.03. The following AEs were considered as DLT unless they were clearly and incontrovertibly attributable to the underlying disease or to an extraneous cause: Grade 5 toxicity; Grade 4 neutropenia lasting ≥42 days from the start of the therapy cycle in absence of evidence of active acute myeloid leukemia (AML) (\<5% blasts); Grade 3 or 4 non-hematologic toxicity (with protocol-define exceptions). Only clinically significant abnormalities in laboratory findings, physical examination, vital signs, weight, or electrocardiogram were considered for DLT assessment.

  Day 1 through Day 21 (first treatment cycle)

Secondary Outcomes (13)

• Part 1 and Part 2: Overall Response Rate (ORR)

  Up to 32 months

• Part 1 and Part 2: Partial Remission (PR) Rate

  Up to 32 months

• Part 1 and Part 2: Duration of Response (DoR)

  Up to 32 months

• Part 1 and Part 2: Relapse Free Survival (RFS)

  Up to 32 months

• Part 1 and Part 2: Overall Survival (OS)

  Up to 32 months

Study Arms (6)

Cohort 1 (25 mg)

EXPERIMENTAL

Participants received MEN1703 (25 milligrams \[mg\]) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Cohort 2 (50 mg)

EXPERIMENTAL

Participants received MEN1703 (50 mg) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Cohort 3 (75 mg)

EXPERIMENTAL

Participants received MEN1703 (75 mg) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Cohort 4 (100 mg)

EXPERIMENTAL

Participants received MEN1703 (100 mg) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Cohort 5 (125 mg)

EXPERIMENTAL

Participants received MEN1703 (125 mg) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Cohort 6 (150 mg)

EXPERIMENTAL

Participants received MEN1703 (150 mg) orally once daily for 14 consecutive days in cycles of 21 days.

Drug: MEN1703

Interventions

MEN1703 DRUG

MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.

Also known as: SEL24-B489, SEL24

Cohort 1 (25 mg); Cohort 2 (50 mg); Cohort 3 (75 mg); Cohort 4 (100 mg); Cohort 5 (125 mg); Cohort 6 (150 mg)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants with diagnosis of AML, all comers or bearing IDH1 or IDH2 mutation (completed)
• Participant has no standard therapeutic options available and has either relapsed AML unsuitable for intensive chemotherapy, with no standard therapeutic options and/or not eligible for any approved targeted therapy or primary refractory AML unsuitable for intensive chemotherapy, with no standard therapeutic options and/or not eligible for any approved targeted therapy

You may not qualify if:

• Anti-cancer treatments (including cytotoxic chemotherapy, radiotherapy, hormonal therapy, biologic, immunotherapy or investigational drugs) received within 14 days or 5 half-lives for targeted therapies (whichever is shorter) before first dose of study drug (to be supplemented)

Sponsors & Collaborators

• Menarini Group: lead
• Medpace, Inc.: collaborator
• Theradex: collaborator

Study Sites (15)

Northside Hospital

Atlanta, Georgia, 30342, United States

Location

Cleveland Clinic, Taussig Cancer Institute

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Vanderbilt Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

Texas Oncology - Baylor Charles A. Sammons Cancer Center

Dallas, Texas, 75246, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

Location

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

Meldola, Italy

Location

Istituto Clinico Humanitas

Milan, Italy

Location

ASST Monza - Ospedale San Gerardo

Monza, Italy

Location

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi, Oddzial Hematologii z Pododdzialem Chemioterapi

Lodz, Poland

Location

Institute of Haematology and Blood Transfusion

Warsaw, Poland

Location

Institut Català d'Oncologia

Badalona, Spain

Location

Hospital 12 de Octubre

Madrid, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, Spain

Location

Related Publications (1)

• Martinelli G, Solomon SR, Mukherjee S, Santoro A, Strickland SA, Vives S, Ravandi F, Walter RB, Cook RJ, Lech-Maranda E, Calbacho M, Wierzbowska A, Marconi G, Acuna-Cruz E, Cano-Ferri I, Bertolini F, Rzymski T, Paoli A, Merlo GM, Auriol FK, Zicari S, Galleu A, Gupta I, Montesinos P. Dual FLT3/PIM inhibitor dapolsertib in acute myeloid leukemia: results from the phase 1/2 DIAMOND-01 trial. Blood Neoplasia. 2025 Oct 24;3(1):100178. doi: 10.1016/j.bneo.2025.100178. eCollection 2026 Feb.

  PMID: 41536779 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Angela Capriati, MD
• Organization: Menarini Ricerche

acapriati@menarini-ricerche.it

+39 055 56 80 9990

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2016
• First Posted: January 2, 2017
• Study Start: March 10, 2017
• Primary Completion: April 13, 2023
• Study Completion: April 13, 2023
• Last Updated: April 29, 2025
• Results First Posted: April 29, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (7); IT (3); ES (3); PL (2)