MEN1703 (SEL24) in Participants With Acute Myeloid Leukemia (Diamond-01)

April 10, 2025 updated by: Menarini Group

A Phase I/II Study of SEL24 in Patients With Acute Myeloid Leukemia

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Phase I/II, open-label, multi-center, dose escalation study to estimate the maximum tolerated dose of MEN1703 in participants with acute myeloid leukemia.

The clinical trial will investigate the safety profile and anti-leukemic activity of MEN1703 in participants with AML and that have no standard therapeutic options available.

The clinical trial encompasses 2 parts:

  • Part 1: Ascending dose levels - the main purpose of this part of the clinical trial is to determine the highest dose of MEN1703 considered to be well tolerated.
  • Part 2: Expansion cohort - the main purpose of this part of the clinical trial is to assess the safety and anti-leukemia activity of MEN1703 given at the highest tolerated dose in participant with relapsed/refractory acute myeloid leukemia, either all comers as well as harboring isocitrate dehydrogenase (IDH1/IDH2) mutations.

Participants participating to the clinical trial will take the study drug as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
73

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Meldola, Italy
        • Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
      • Milano, Italy
        • Istituto Clinico Humanitas
      • Monza, Italy
        • ASST Monza - Ospedale San Gerardo
  • Poland
      • Warsaw, Poland
        • Institute of Haematology and Blood Transfusion
      • Łódź, Poland
        • Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi, Oddzial Hematologii z Pododdzialem Chemioterapi
  • Spain
      • Badalona, Spain
        • Institut Catala d'Oncologia
      • Madrid, Spain
        • Hospital 12 de Octubre
      • Valencia, Spain
        • Hospital Universitari i Politecnic La Fe
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Northside Hospital
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic, Taussig Cancer Institute
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt Ingram Cancer Center
    • Texas
      • Dallas, Texas, United States, 75246
        • Texas Oncology - Baylor Charles A. Sammons Cancer Center
      • Houston, Texas, United States, 77030
        • MD Anderson Cancer Center
    • Washington
      • Seattle, Washington, United States, 98109
        • Fred Hutchinson Cancer Research Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Cohort 1 (25 mg)
Participants received MEN1703 (25 milligrams [mg]) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24
Experimental: Cohort 2 (50 mg)
Participants received MEN1703 (50 mg) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24
Experimental: Cohort 3 (75 mg)
Participants received MEN1703 (75 mg) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24
Experimental: Cohort 4 (100 mg)
Participants received MEN1703 (100 mg) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24
Experimental: Cohort 5 (125 mg)
Participants received MEN1703 (125 mg) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24
Experimental: Cohort 6 (150 mg)
Participants received MEN1703 (150 mg) orally once daily for 14 consecutive days in cycles of 21 days.
Drug: MEN1703
MEN1703 given as oral capsules once daily for 14 consecutive days over a 21-day treatment cycle.
Other Names:
  • SEL24-B489
  • SEL24

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Part 1 and Part 2: Number of Participants Experiencing Treatment-emergent Adverse Events
Time Frame: Up to 21 months
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)
Time Frame: Day 1 through Day 21 (first treatment cycle)
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Part 1 and Part 2: Overall Response Rate (ORR)
Time Frame: Up to 32 months
ORR was defined as the percentage of participants who had a complete remission (CR), complete remission with incomplete hematologic recovery (CRi), complete remission with partial hematologic recovery (CRh), or morphologic leukemia-free state (MLFS) response to therapy.
Up to 32 months
Part 1 and Part 2: Partial Remission (PR) Rate
Time Frame: Up to 32 months
PR rate was defined as the percentage of participants who had a partial remission response to therapy.
Up to 32 months
Part 1 and Part 2: Duration of Response (DoR)
Time Frame: Up to 32 months
DoR was defined as the time from the date of first CR, CRi, CRh, CR without minimal residual disease (CRMRD-), MLFS or PR until the date of documented relapse of any type, progressive disease or death due to disease progression for participants who achieve CR, CRi, CRh, CRMRD-, MLFS or PR. Results are reported in days.
Up to 32 months
Part 1 and Part 2: Relapse Free Survival (RFS)
Time Frame: Up to 32 months
RFS was defined as the time from the date of first CR, CRi, CRh, or CRMRD- until the date of documented relapse or death from any cause. Results are reported in days.
Up to 32 months
Part 1 and Part 2: Overall Survival (OS)
Time Frame: Up to 32 months
OS was defined as the number of days between the first study drug administration and death from any cause. Results are reported in days.
Up to 32 months
Part 1 and Part 2: Event Free Survival (EFS)
Time Frame: Up to 32 months
EFS was defined as the time from the date of first study drug intake until the date of documented relapse, treatment failure, or death from any cause. Results are reported in days.
Up to 32 months
Part 1 and Part 2: Transfusion Conversion Rate
Time Frame: Up to 21 months
Transfusion conversion rate was defined as the percentage of participants who were transfusion dependent at baseline but became transfusion independent post-baseline. Participants were classified as baseline transfusion independent if there were no red blood cells (RBC) or platelet transfusions at baseline; otherwise, the participant was considered as baseline transfusion dependent. Participants were classified post-baseline transfusion independent in the event of 56 consecutive days without any RBC or platelet transfusion post-baseline; otherwise, the participant was considered post-baseline transfusion dependent.
Up to 21 months
Part 1 and Part 2: Transfusion Maintenance Rate
Time Frame: Up to 21 months
Transfusion maintenance rate was defined as the percentage of participants who were transfusion independent at baseline and still maintained to be transfusion independent post-baseline. Participants were classified as baseline transfusion independent if there were no RBC or platelet transfusions at baseline; otherwise, the participant was considered as baseline transfusion dependent. Participants were classified post-baseline transfusion independent in the event of 56 consecutive days without any RBC or platelet transfusion post-baseline; otherwise, the participant was considered post-baseline transfusion dependent.
Up to 21 months
Part 1 and Part 2: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) Rate
Time Frame: Up to 21 months
Allogeneic HSCT rate was defined as the percentage of participants undergoing allogeneic stem cell transplant during the study period of each participant.
Up to 21 months
Part 1 and Part 2: Percentage of Participants With ≥ 50% Bone Marrow Blast Reduction
Time Frame: Up to 20 months
Bone marrow aspirates/biopsies were taken at designated timepoints for evaluation of leukemic blast proportion in the bone marrow. A reduction in bone marrow blast proportion indicates increased anti-leukemic activity of the study drug.
Up to 20 months
Part 1 and Part 2: Maximum Observed Concentration (Cmax) for MEN1703
Time Frame: Day 1 and Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)
Nominal blood samples were taken at designated timepoints for evaluation of concentration levels of MEN1703 in plasma. Results are reported as nanograms/milliliter (ng/mL). Standard error not reported, arithmetic coefficient of variation (CV%) reported instead.
Day 1 and Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)
Part 1 and Part 2: Area Under the Concentration Versus Time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) for MEN1703
Time Frame: Day 1 of Cycle 1 (pre-dose, up to 24 hours post dose) (21 days/cycle)
Nominal blood samples were taken at designated timepoints for evaluation of concentration levels of MEN1703 in plasma. AUClast was calculated by the linear trapezoidal rule. Results are reported in hour times nanograms/milliliter (h*ng/mL). Standard error not reported, arithmetic CV% reported instead.
Day 1 of Cycle 1 (pre-dose, up to 24 hours post dose) (21 days/cycle)
Part 1 and Part 2: Area Under the Concentration Versus Time Curve From Time Zero to 24 Hours (AUC0-24) for MEN1703
Time Frame: Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)
Nominal blood samples were taken at designated timepoints for evaluation of concentration levels of MEN1703 in plasma. AUC0-24 was calculated by the linear trapezoidal rule. Results are reported in h*ng/mL. Standard error not reported, arithmetic CV% reported instead.
Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Menarini Group

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Medpace, Inc.
Theradex

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Farhad Ravandi, MD, Department of Leukemia, MDACC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 10, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 13, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 13, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 10, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 30, 2016

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 2, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 29, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 10, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CLI24-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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