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Absk021 Hydrochloride Hydrate

ABSK021 Hydrochloride Hydrate is a promising drug being studied in clinical trials for the treatment of Tenosynovial Giant Cell Tumor (TGCT). This article explores the ongoing research to determine its safety and effectiveness in patients with this rare condition. The trials aim to compare ABSK021 with a placebo to assess its impact on tumor response, joint function, and overall quality of life for TGCT patients.

Table of Contents

What is ABSK021?

ABSK021, also known as ABSK021 HYDROCHLORIDE HYDRATE, is a new medication being studied for the treatment of Tenosynovial Giant Cell Tumor (TGCT). It is a synthetic small molecule that comes in the form of a capsule and is taken orally (by mouth)[1]. ABSK021 has been designated as an orphan drug (EU/3/23/2873), which means it is intended to treat a rare condition[1].

What is Tenosynovial Giant Cell Tumor (TGCT)?

Tenosynovial Giant Cell Tumor (TGCT) is a rare, non-cancerous tumor that affects the tissues surrounding joints, tendons, and synovium (the lining of joints). It can cause pain, swelling, and reduced range of motion in the affected joint. TGCT is typically treated with surgery, but in some cases, the tumor may not be suitable for surgical removal[1].

How ABSK021 Works

ABSK021 is believed to work by targeting a specific pathway in the body called the CSF-1/CSF-1R pathway. This pathway is thought to play a role in the growth of TGCT. By inhibiting this pathway, ABSK021 may help to slow down or stop the growth of the tumor[1].

Clinical Trial Details

A Phase 3 clinical trial is currently underway to study the effectiveness and safety of ABSK021 in patients with TGCT. This trial is:

  • Randomized: Patients are randomly assigned to receive either ABSK021 or a placebo (a pill with no active ingredient).
  • Double-blind: Neither the patients nor the doctors know who is receiving ABSK021 or the placebo.
  • Placebo-controlled: The effects of ABSK021 are compared to a placebo to determine its true impact.
  • Multicenter: The study is being conducted at multiple hospitals or clinics.

The main goal of this study is to compare the Objective Response Rate (ORR) between patients taking ABSK021 and those taking a placebo within 25 weeks of treatment. ORR measures how many patients experience a significant reduction in tumor size[1].

Eligibility Criteria

To participate in this clinical trial, patients must meet certain criteria. Some key requirements include:

  • Having a confirmed diagnosis of TGCT that cannot be treated with surgery
  • Having at least one tumor that measures 2 cm or larger
  • Having adequate organ function and bone marrow function
  • Not having received previous treatment with certain types of drugs that target the CSF-1/CSF-1R pathway
  • Not having known metastatic TGCT (TGCT that has spread to other parts of the body)

It’s important to note that these are just some of the criteria. A doctor or research team would need to assess each potential participant to determine if they are eligible for the study[1].

Potential Benefits of ABSK021

The clinical trial aims to assess several potential benefits of ABSK021, including:

  • Reduction in tumor size
  • Improvement in range of motion of the affected joint
  • Reduction in stiffness and pain
  • Improvement in overall physical functioning and quality of life
These outcomes are being measured using various scales and imaging techniques throughout the study[1].

Safety Considerations

As with any new medication, safety is a crucial aspect of the ABSK021 clinical trial. The study is designed to carefully monitor and compare the safety of ABSK021 to the placebo. Patients with certain conditions, such as impaired cardiac function, active liver disease, or a history of certain other cancers, may not be eligible to participate in the trial due to potential safety concerns[1].

It’s important to remember that ABSK021 is still an investigational drug, which means it has not yet been approved for general use. The purpose of this clinical trial is to gather more information about its effectiveness and safety in treating TGCT.

Aspect Details
Drug Name ABSK021 Hydrochloride Hydrate
Condition Studied Tenosynovial Giant Cell Tumor (TGCT)
Study Phase Phase 3
Study Design Randomized, Double-blind, Placebo-Controlled, Multicenter
Primary Objective Compare Objective Response Rate (ORR) within 25 weeks
Key Secondary Objectives Assess Range of Motion, Patient Reported Outcomes, Duration of Response, Safety
Dosage Form Oral capsule
Maximum Daily Dose 50 mg
Treatment Duration Up to 48 weeks
Key Inclusion Criteria Histologically confirmed TGCT, measurable disease ≥ 2 cm, not suitable for surgery
Key Exclusion Criteria Known allergy to drug components, previous CSF-1/CSF-1R inhibitor treatment, metastatic TGCT

FAQ

  • What is Tenosynovial Giant Cell Tumor (TGCT)? Tenosynovial Giant Cell Tumor (TGCT) is a rare, non-cancerous growth that affects the tissue lining joints and tendons. It can cause pain, swelling, and reduced range of motion in the affected joint.
  • How is ABSK021 administered in the clinical trial? ABSK021 is administered orally in capsule form. The maximum daily dose in the trial is 50 mg, with a total maximum dose of 73,000 mg over a treatment period of up to 48 weeks.
  • What are the main goals of the ABSK021 clinical trial? The main goals are to compare the Objective Response Rate (ORR) of ABSK021 versus placebo, assess its effects on Range of Motion (ROM) and patient-reported outcomes, evaluate the duration of response, and determine the safety profile of the drug in TGCT patients.
  • Who is eligible to participate in the ABSK021 clinical trial? Eligible participants include those with histologically confirmed TGCT that is not suitable for surgery, have measurable disease of at least 2 cm, and meet specific criteria for pain and stiffness. Participants must have adequate organ function and no known allergies to the drug components.
  • How is the effectiveness of ABSK021 measured in the trial? The effectiveness is primarily measured by the Objective Response Rate (ORR) at 25 weeks, assessed by a Blinded Independent Review Committee using RECIST 1.1 criteria. Secondary measures include tumor volume score, range of motion improvements, and changes in patient-reported outcomes for pain, stiffness, and quality of life.

Ongoing Clinical Trials on Absk021 Hydrochloride Hydrate

  • Study on the Safety and Effectiveness of ABSK021 for Patients with Tenosynovial Giant Cell Tumor

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Italy The Netherlands Poland Spain

Glossary

  • Tenosynovial Giant Cell Tumor (TGCT): A rare, non-cancerous tumor that affects the tissue lining joints and tendons, causing pain, swelling, and reduced joint function.
  • ABSK021 Hydrochloride Hydrate: The investigational drug being studied for the treatment of TGCT, administered orally in capsule form.
  • Objective Response Rate (ORR): A measure of how well a tumor responds to treatment, usually assessed by imaging techniques.
  • RECIST 1.1: Response Evaluation Criteria in Solid Tumors version 1.1, a standardized method to measure tumor response in cancer treatments.
  • Tumor Volume Score (TVS): A method of measuring tumor size and response to treatment based on the volume of the tumor.
  • Range of Motion (ROM): The measurement of movement around a specific joint or body part, often used to assess joint function.
  • Patient Reported Outcome (PRO): Information about a patient's health condition that comes directly from the patient, without interpretation by doctors or others.
  • Duration of Response (DOR): The length of time that a tumor continues to respond to treatment without the cancer growing or spreading.
  • Pharmacokinetic (PK) profile: The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Blinded Independent Review Committee (BIRC): A group of experts who review clinical trial data without knowing which treatment each patient received, to ensure unbiased assessment.
  • Numeric Rating Scale (NRS): A tool used to measure the intensity of symptoms like pain or stiffness on a scale, typically from 0 to 10.
  • PROMIS: Patient-Reported Outcomes Measurement Information System, a set of person-centered measures that evaluate physical, mental, and social health.
  • EuroQol visual analogue scale (VAS): A measurement tool that records an individual's health status on a vertical scale, usually from 0 (worst health) to 100 (best health).

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-absk021-for-patients-with-tenosynovial-giant-cell-tumor/