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Patidegib Hydrochloride Isopropanolate

Patidegib Hydrochloride Isopropanolate, formulated as a 2% gel, is being studied in clinical trials for its potential to reduce the burden of basal cell carcinomas (BCCs) in adults with Gorlin syndrome. This rare genetic condition predisposes individuals to develop multiple BCCs throughout their lifetime. The ongoing phase 3 clinical trial aims to evaluate the efficacy and safety of Patidegib Gel when applied twice daily to the face of patients with Gorlin syndrome over a 12-month period.

Table of Contents

What is Patidegib?

Patidegib is a medication being studied for the treatment of basal cell carcinomas (BCCs) in patients with Gorlin syndrome. It is formulated as a gel containing 2% of the active ingredient, patidegib hydrochloride isopropanolate. This gel is designed to be applied directly to the skin (topically) on the face[1].

Patidegib is also known by its alternative name, IPI-926 hydrochloride isopropanolate[1]. It belongs to a class of drugs called Hedgehog inhibitors, which work by blocking a specific cellular pathway involved in the development of basal cell carcinomas.

Target Condition: Gorlin Syndrome and Basal Cell Carcinomas

Gorlin syndrome, also known as Gorlin-Goltz syndrome or basal cell nevus syndrome, is a rare genetic condition characterized by the development of multiple basal cell carcinomas (BCCs) on the skin[1]. People with this syndrome have a mutation in the PTCH1 gene, which makes them prone to developing these skin cancers, especially on the face[1].

Basal cell carcinomas are a type of skin cancer that develops in the basal cells, which are found in the lower part of the epidermis (the outermost layer of skin). While BCCs are usually slow-growing and rarely spread to other parts of the body, they can cause significant local damage and disfigurement if left untreated[1].

How Patidegib Works

Patidegib is a Hedgehog pathway inhibitor. The Hedgehog signaling pathway is a complex system of proteins that play a crucial role in cell growth and differentiation. In Gorlin syndrome, this pathway is overactive due to the PTCH1 gene mutation, leading to the formation of multiple BCCs[1].

By inhibiting the Hedgehog pathway, Patidegib aims to reduce the formation of new BCCs and potentially shrink existing ones. The gel formulation allows for direct application to affected areas on the face, potentially minimizing systemic side effects compared to oral medications[1].

Clinical Trial Details

A Phase 3 clinical trial is currently underway to evaluate the efficacy and safety of Patidegib Gel 2% in patients with Gorlin syndrome[1]. Here are some key details about the trial:

  • It is a multicenter, randomized, double-blind, vehicle-controlled study[1].
  • Participants are randomly assigned to either Patidegib Gel 2% or a vehicle (placebo) gel[1].
  • The gel is to be applied twice daily to the face for up to 12 months[1].
  • The main goal is to assess how well Patidegib Gel 2% reduces the number of new BCCs compared to the vehicle gel[1].

Eligibility Criteria

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

  • Age 18 years or older[1]
  • Confirmed PTCH1 gene mutation[1]
  • At least 10 BCCs on the face at the start of the study[1]
  • Willingness to avoid other topical medications on the face during the study[1]

Some exclusion criteria include:

  • Recent use of treatments that might interfere with the study[1]
  • Known hypersensitivity to any ingredients in the gel[1]
  • Uncontrolled systemic disease[1]
  • Treatment for invasive cancer within the past 5 years (with some exceptions)[1]

Potential Benefits

If proven effective, Patidegib Gel 2% could offer several benefits for patients with Gorlin syndrome:

  • Reduction in the number of new BCCs developing on the face[1]
  • Potential shrinkage or resolution of existing BCCs[1]
  • Improvement in quality of life related to skin cancer[1]
  • A non-invasive treatment option that could reduce the need for surgical interventions[1]

Safety and Side Effects

The clinical trial is designed to assess the safety and tolerability of Patidegib Gel 2% when applied twice daily for 12 months[1]. Potential side effects and safety concerns will be closely monitored throughout the study. These may include:

  • Local skin reactions at the application site[1]
  • Any adverse events or serious adverse events occurring during the trial[1]
  • Changes in dermal safety and local tolerability as assessed by the investigator[1]

It’s important to note that as this is an investigational treatment, not all potential side effects may be known at this time. Patients participating in the trial will be closely monitored for any unexpected reactions or safety concerns[1].

Aspect Details
Drug Name Patidegib Hydrochloride Isopropanolate (2% Gel)
Study Type Phase 3, Multicenter, Randomized, Double-blind, Vehicle-controlled
Target Condition Basal Cell Carcinomas in Gorlin Syndrome
Main Objective Assess efficacy in reducing new BCCs after 12 months
Key Eligibility Criteria Age ≥18, confirmed PTCH1 mutation, ≥10 facial BCCs
Treatment Duration Up to 12 months
Application Method Twice daily to the face
Primary Endpoint Number of new BCCs at Month 12
Key Secondary Endpoints New surgically eligible BCCs, change in aBCCdex score, quality of life, safety and tolerability

FAQ

  • What is Gorlin syndrome? Gorlin syndrome, also known as Gorlin-Goltz syndrome or basal cell nevus syndrome, is a rare genetic condition that predisposes individuals to develop multiple basal cell carcinomas (BCCs) throughout their lifetime. It is caused by a mutation in the PTCH1 gene.
  • How is Patidegib Gel being tested in the clinical trial? Patidegib Gel 2% is being tested in a phase 3 clinical trial where it is applied twice daily to the face of adults with Gorlin syndrome. The study is randomized, double-blind, and vehicle-controlled, meaning some participants will receive the active gel while others will receive a placebo gel.
  • What are the main goals of the clinical trial? The main goals of the trial are to assess the efficacy of Patidegib Gel in reducing the number of new BCCs after 12 months of treatment, evaluate its safety and tolerability, and measure its impact on the quality of life of patients with Gorlin syndrome.
  • Who is eligible to participate in this clinical trial? Eligible participants must be at least 18 years old, have a confirmed PTCH1 mutation, and have at least 10 BCCs on the face at the start of the trial. They must also be willing to abstain from using other topical medications on their facial skin during the study period.
  • How long does the clinical trial last? The clinical trial consists of a screening period, followed by a treatment period of up to 12 months. After the last application of the study medicine, there is a remote safety follow-up visit 30 days later.

Ongoing Clinical Trials on Patidegib Hydrochloride Isopropanolate

  • Study on Patidegib Gel for Reducing Basal Cell Carcinomas in Adults with Gorlin Syndrome

    Not recruiting

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

Glossary

  • Basal Cell Carcinoma (BCC): A type of skin cancer that begins in the basal cells, which are responsible for producing new skin cells. BCCs are the most common type of skin cancer and are usually slow-growing and rarely spread to other parts of the body.
  • Gorlin Syndrome: A rare genetic condition, also known as Gorlin-Goltz syndrome or basal cell nevus syndrome, characterized by the development of multiple basal cell carcinomas and other developmental abnormalities. It is caused by a mutation in the PTCH1 gene.
  • PTCH1 Mutation: A genetic alteration in the PTCH1 gene, which is responsible for producing a protein that helps regulate cell growth and division. Mutations in this gene are associated with Gorlin syndrome.
  • Hedgehog Inhibitor: A class of drugs that block the Hedgehog signaling pathway, which is involved in cell growth and division. These inhibitors are used to treat certain types of cancer, including basal cell carcinomas.
  • Randomized, Double-blind, Vehicle-controlled Study: A type of clinical trial where participants are randomly assigned to either the treatment group or control group. Neither the participants nor the researchers know who is receiving the active treatment or placebo (vehicle). This design helps reduce bias in the study results.
  • Adverse Event (AE): Any unfavorable or unintended sign, symptom, or disease that occurs during a clinical trial, whether or not it is related to the treatment being studied.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect.
  • Surgically Eligible BCC (SEB): A basal cell carcinoma that meets the criteria for surgical removal, based on its size, location, or other characteristics.
  • Advanced Basal Cell Carcinoma Index (aBCCdex): A scale used to measure the symptoms and severity of basal cell carcinomas in clinical trials.
  • Skin Cancer Index: A quality of life questionnaire specifically designed to assess the impact of skin cancer on a patient's well-being and daily life.

References

  1. http://clinicaltrials.eu/trial/study-on-patidegib-gel-for-reducing-basal-cell-carcinomas-in-adults-with-gorlin-syndrome/