Clinical trial NCT03008187: MEN1703 (SEL24) in Participants With Acute Myeloid Leukemia
Official title: A Phase I/II Study of SEL24 in Patients With Acute Myeloid Leukemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Acute Myeloid Leukemia
Keywords: AML
Relapsed/Refractory Acute Myeloid Leukemia
IDH
Lead sponsor: Menarini Group
Sponsor class: INDUSTRY
Collaborators: name: Medpace, Inc.
name: Theradex
First posted: 2017-01-02
Enrollment: 73
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Part 1 and Part 2: Number of Participants Experiencing Treatment-emergent Adverse Events
timeFrame: Up to 21 months
measure: Part 1: Number of Participants Experiencing Dose-limiting Toxicity (DLT)
timeFrame: Day 1 through Day 21 (first treatment cycle)
Secondary outcomes:
measure: Part 1 and Part 2: Overall Response Rate (ORR)
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Partial Remission (PR) Rate
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Duration of Response (DoR)
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Relapse Free Survival (RFS)
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Overall Survival (OS)
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Event Free Survival (EFS)
timeFrame: Up to 32 months
measure: Part 1 and Part 2: Transfusion Conversion Rate
timeFrame: Up to 21 months
measure: Part 1 and Part 2: Transfusion Maintenance Rate
timeFrame: Up to 21 months
measure: Part 1 and Part 2: Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) Rate
timeFrame: Up to 21 months
measure: Part 1 and Part 2: Percentage of Participants With ≥ 50% Bone Marrow Blast Reduction
timeFrame: Up to 20 months
measure: Part 1 and Part 2: Maximum Observed Concentration (Cmax) for MEN1703
timeFrame: Day 1 and Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)
measure: Part 1 and Part 2: Area Under the Concentration Versus Time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) for MEN1703
timeFrame: Day 1 of Cycle 1 (pre-dose, up to 24 hours post dose) (21 days/cycle)
measure: Part 1 and Part 2: Area Under the Concentration Versus Time Curve From Time Zero to 24 Hours (AUC0-24) for MEN1703
timeFrame: Day 14 of Cycle 1 (pre-dose, up to 120 hours post dose) (21 days/cycle)
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
city: Cleveland
country: United States
facility: Cleveland Clinic, Taussig Cancer Institute
state: Ohio
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Nashville
country: United States
facility: Vanderbilt Ingram Cancer Center
state: Tennessee
city: Dallas
country: United States
facility: Texas Oncology - Baylor Charles A. Sammons Cancer Center
state: Texas
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Seattle
country: United States
facility: Fred Hutchinson Cancer Research Center
state: Washington
city: Meldola
country: Italy
facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
city: Milano
country: Italy
facility: Istituto Clinico Humanitas
city: Monza
country: Italy
facility: ASST Monza - Ospedale San Gerardo
city: Warsaw
country: Poland
facility: Institute of Haematology and Blood Transfusion
city: Łódź
country: Poland
facility: Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi, Oddzial Hematologii z Pododdzialem Chemioterapi
city: Badalona
country: Spain
facility: Institut Català d'Oncologia
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe
Study officials:
References: