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N-Ethyl-2-[4-[7-[[4-(Ethylsulfonylamino)Cyclohexyl]Methyl]-2,7-Diazaspiro[3.5]Nonan-2-Yl]Pyrimidin-5-Yl]Oxy-5-Fluoro-N-Propan-2-Ylbenzamide

This article discusses the ongoing clinical trials of SNDX-5613, a promising drug being studied for patients with relapsed or refractory acute leukemias. The trials aim to evaluate the safety, tolerability, and effectiveness of SNDX-5613 in patients whose leukemia has not responded to previous treatments or has come back after initial therapy. The study focuses on specific genetic mutations associated with these challenging forms of leukemia.

Table of Contents

Introduction

SNDX-5613 is an investigational drug being studied for the treatment of relapsed or refractory acute leukemias. This article will provide an overview of the drug, its potential uses, and the ongoing clinical trials investigating its safety and effectiveness.[1]

What is SNDX-5613?

SNDX-5613 is a medication developed by Syndax Pharmaceuticals, Inc. It is also known by its chemical name: N-ethyl-2-[4-[7-[[4-(ethylsulfonylamino)cyclohexyl]methyl]-2,7-diazaspiro[3.5]nonan-2-yl]pyrimidin-5-yl]oxy-5-fluoro-N-propan-2-ylbenzamide. The drug is available in capsule form and is taken orally.[1]

Target Conditions

SNDX-5613 is being studied for the treatment of relapsed or refractory acute leukemias. This includes:[1]

  • Acute Myeloid Leukemia (AML)
  • Acute Lymphoblastic Leukemia (ALL)
  • Mixed Phenotype Acute Leukemia (MPAL)

The drug is particularly focused on leukemias with specific genetic characteristics, such as:

  • KMT2A (MLL) gene rearrangements: These are genetic changes that can occur in leukemia cells and are associated with a poor prognosis.
  • NPM1 mutations: Mutations in the Nucleophosmin 1 gene, which are common in some types of AML.
  • NUP98 rearrangements: Another type of genetic change found in some leukemias.

How SNDX-5613 Works

While the exact mechanism of action is not fully described in the provided information, SNDX-5613 is likely designed to target specific molecular pathways involved in the growth and survival of leukemia cells, particularly those with the genetic changes mentioned above. By interfering with these pathways, the drug aims to stop the growth of cancer cells and potentially eliminate them.[1]

Clinical Trial Overview

SNDX-5613 is currently being studied in a Phase 1/2 clinical trial. The trial has two main parts:[1]

  1. Phase 1: This phase aims to:
    • Determine the safety and tolerability of SNDX-5613
    • Find the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D)
    • Study how the drug is processed by the body (pharmacokinetics)
  2. Phase 2: This phase focuses on:
    • Evaluating short- and long-term safety
    • Assessing how well the drug works in treating leukemia, including measures like complete remission rate and overall response rate

Eligibility Criteria

The trial has specific criteria for who can participate. Some key points include:[1]

  • Patients must be at least 6 months old
  • Have relapsed or refractory acute leukemia
  • Have specific genetic changes in their leukemia cells (KMT2A rearrangement, NPM1 mutation, or NUP98 rearrangement)
  • Must have completed previous treatments and recovered from side effects

There are also several exclusion criteria, such as having certain other medical conditions or taking specific medications that might interfere with the study.

Potential Benefits

While it’s important to note that SNDX-5613 is still experimental, the study aims to assess several potential benefits:[1]

  • Achieving complete remission or partial remission of leukemia
  • Becoming independent of blood transfusions
  • Improved overall survival
  • Longer event-free survival (time without cancer progression or other significant events)

Safety Considerations

As with any experimental treatment, there are potential risks and side effects. The study is carefully monitoring for:[1]

  • Treatment-emergent adverse events (side effects that occur during treatment)
  • Changes in laboratory test results
  • Effects on heart rhythm (monitored by ECG)
  • Changes in vital signs and overall health status

Patients in the study will be closely monitored for any side effects or safety concerns.

Aspect Details
Drug Name SNDX-5613
Study Type Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion
Target Condition Relapsed/Refractory Acute Leukemias (AML, ALL, MPAL)
Genetic Focus KMT2A rearrangements, NPM1 mutations
Primary Objectives Safety, tolerability, maximum tolerated dose, recommended Phase 2 dose
Secondary Objectives Pharmacokinetics, response rates, survival outcomes
Key Eligibility Age ≥6 months, R/R acute leukemia, specific genetic mutations
Main Endpoints CR+CRh rate, adverse events, laboratory abnormalities

FAQ

  • What is SNDX-5613? SNDX-5613 is an investigational drug being studied for the treatment of relapsed or refractory acute leukemias. It is specifically designed to target leukemias with certain genetic mutations, such as KMT2A rearrangements or NPM1 mutations.
  • What types of leukemia are being studied in these trials? The trials are focusing on relapsed or refractory acute leukemias, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and mixed phenotype acute leukemia (MPAL). These are forms of leukemia that have either not responded to previous treatments or have come back after initial therapy.
  • What are the main goals of the clinical trials? The main goals of the trials are to determine the safety and tolerability of SNDX-5613, find the best dose for future studies, and evaluate how effective the drug is in treating these difficult forms of leukemia. The studies will also look at how the body processes the drug and how long the effects of the treatment last.
  • Who can participate in these clinical trials? Participants must be at least 6 months old and have relapsed or refractory acute leukemia. They must meet specific criteria regarding their disease status, previous treatments, and overall health. Some arms of the study have additional requirements, such as having certain genetic mutations or being able to take specific antifungal medications.
  • How is the effectiveness of SNDX-5613 being measured? The effectiveness of SNDX-5613 is being measured in several ways, including the rate of complete remission, how long patients remain free from needing blood transfusions, overall response rate, and how long patients survive without their disease getting worse. The study will also track overall survival rates.

Ongoing Clinical Trials on N-Ethyl-2-[4-[7-[[4-(Ethylsulfonylamino)Cyclohexyl]Methyl]-2,7-Diazaspiro[3.5]Nonan-2-Yl]Pyrimidin-5-Yl]Oxy-5-Fluoro-N-Propan-2-Ylbenzamide

  • Study on the Safety and Tolerability of SNDX-5613 for Patients with Relapsed or Refractory Acute Leukemias

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Lithuania The Netherlands Spain

Glossary

  • Acute Leukemia: A fast-growing cancer of the blood and bone marrow that affects the production of normal blood cells.
  • Relapsed/Refractory (R/R): Refers to cancer that has either returned after a period of improvement (relapsed) or has not responded to previous treatments (refractory).
  • KMT2A Rearrangement: A genetic change in the KMT2A gene that is associated with some types of leukemia and can affect how the disease responds to treatment.
  • NPM1 Mutation: A change in the NPM1 gene that is commonly found in some types of acute myeloid leukemia.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including how it's absorbed, distributed, metabolized, and eliminated.
  • Complete Remission (CR): A state where there is no evidence of disease and blood cell counts have returned to normal levels.
  • Maximum Tolerated Dose (MTD): The highest dose of a drug that can be given without causing unacceptable side effects.
  • Dose-Limiting Toxicity (DLT): Side effects that are severe enough to prevent increasing the dose of a drug in a clinical trial.
  • Overall Response Rate (ORR): The percentage of patients whose cancer shrinks or disappears after treatment.
  • Event-Free Survival (EFS): The length of time after treatment that a patient remains free of certain complications or events related to their disease.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-sndx-5613-for-patients-with-relapsed-or-refractory-acute-leukemias/