#1 Clinical Trials Search in Europe

Miransertib

Miransertib, also known as MK-7075 or ARQ 092, is an investigational drug being studied in clinical trials for the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS). These rare genetic disorders are characterized by overgrowth of various body tissues. The clinical trials aim to evaluate the safety, tolerability, and effectiveness of miransertib in managing these conditions, offering hope for patients with limited treatment options.

Table of Contents

What is Miransertib?

Miransertib, also known as MK-7075 or ARQ 092, is an experimental drug being developed to treat rare overgrowth disorders[1][2]. It is a small molecule drug, which means it’s a tiny compound that can easily enter cells in the body. Miransertib is taken orally as a capsule, making it convenient for patients to use[1].

What Conditions Does Miransertib Treat?

Miransertib is being studied primarily for two rare conditions:

  • PIK3CA-Related Overgrowth Spectrum (PROS): This is a group of rare disorders characterized by overgrowth of various body tissues. The overgrowth can affect different parts of the body and can vary in severity[1].
  • Proteus Syndrome (PS): This is an extremely rare condition that causes overgrowth of bones, skin, and other tissues. It usually begins in infancy and becomes more noticeable as a child grows[3].

Both of these conditions are caused by genetic mutations that lead to excessive cell growth in certain parts of the body[3].

How Does Miransertib Work?

Miransertib works by targeting a specific protein in the body called AKT1. In patients with PROS and Proteus Syndrome, the AKT1 protein is overactive, which leads to excessive cell growth and tissue overgrowth[3].

Miransertib is an AKT inhibitor, meaning it blocks the activity of the AKT1 protein. By doing this, it aims to slow down or stop the overgrowth of tissues in patients with these rare disorders[4].

Current Clinical Trials

Several clinical trials are currently underway to study the effectiveness and safety of miransertib:

  • The MOSAIC study (NCT03094832) is a Phase 1/2 trial studying miransertib in patients with PROS and Proteus Syndrome[1].
  • Another study (NCT04980872) is looking at the long-term safety of miransertib in patients who have been treated with the drug in other studies[2].
  • A Phase 2 trial (NCT04316546) is specifically studying miransertib in patients with Proteus Syndrome[3].

Dosage and Administration

In the clinical trials, miransertib is typically given as follows:

  • It is taken orally once daily, either 1 hour before or 2 hours after a meal[2].
  • The dosage is often based on body surface area, starting at 15 mg/m² and potentially increasing to 25 or 35 mg/m² depending on how well the patient tolerates the drug[1].
  • Treatment is given in cycles, with each cycle typically lasting 28 days[1].

Safety and Side Effects

As with any medication, miransertib can cause side effects. The clinical trials are closely monitoring patients for any adverse events (unwanted or harmful effects). Common ways of assessing safety include:

  • Tracking the number of patients who experience adverse events[1].
  • Monitoring for serious adverse events, which are more severe or life-threatening effects[2].
  • Keeping track of how many patients need to stop treatment due to side effects[1].

The specific side effects of miransertib are still being studied. Patients in clinical trials are closely monitored with regular check-ups, blood tests, and other assessments to ensure their safety[3].

Future Prospects

Miransertib represents a promising approach to treating rare overgrowth disorders like PROS and Proteus Syndrome. If the clinical trials show positive results, it could become the first targeted treatment for these conditions[3].

Researchers are not only looking at whether miransertib can slow or stop overgrowth, but also whether it can improve patients’ quality of life, reduce pain, and improve physical functioning[3].

While the results are still pending, miransertib offers hope for patients with these rare and challenging disorders. As always, patients should discuss with their healthcare providers about the potential benefits and risks of participating in clinical trials or using experimental treatments.

Aspect Details
Drug Name Miransertib (MK-7075, ARQ 092)
Conditions Studied PIK3CA-Related Overgrowth Spectrum (PROS), Proteus Syndrome (PS)
Administration Oral, once daily
Dosage Range 5-35 mg/m² (varies by trial and patient)
Trial Phases Phase 1/2
Primary Outcomes Safety, tolerability, adverse events, treatment discontinuation
Secondary Outcomes Changes in pain, physical functioning, quality of life, lesion growth
Duration Varies by trial, up to 96 cycles (approximately 7-8 years)
Age Groups 2 years and older (varies by trial)
Key Assessments Imaging (MRI, CT, ultrasound), photography, blood tests, quality of life surveys

FAQ

  • What is Miransertib and how does it work? Miransertib is a small molecule drug that inhibits AKT, a protein involved in cell growth. In PROS and Proteus Syndrome, there are mutations that cause overactivation of the AKT/PI3K pathway, leading to excessive tissue growth. By inhibiting AKT, miransertib aims to reduce or stabilize this overgrowth.
  • What conditions are being studied in these clinical trials? The clinical trials are focusing on PIK3CA-Related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS). These are rare genetic disorders characterized by overgrowth of various body tissues, which can cause significant health problems and physical deformities.
  • How is miransertib administered in these trials? Miransertib is given as an oral medication, typically taken once daily. The dosage may vary depending on the specific trial and patient characteristics, but it's usually administered in cycles of 28 days each.
  • What are the main objectives of these clinical trials? The main objectives include assessing the safety and tolerability of miransertib, determining the appropriate dosage, and evaluating its effectiveness in reducing or stabilizing tissue overgrowth in patients with PROS and Proteus Syndrome.
  • Who can participate in these clinical trials? Eligibility varies by trial, but generally includes patients aged 2 years and older who have been diagnosed with PROS or Proteus Syndrome. Some trials may have specific criteria related to the type or extent of overgrowth.

Ongoing Clinical Trials on Miransertib

  • Long-term Safety Study of Miransertib for Patients with PIK3CA-related Overgrowth Spectrum or Proteus Syndrome

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Italy

Glossary

  • PIK3CA-Related Overgrowth Spectrum (PROS): A group of rare disorders characterized by overgrowth of various body tissues, caused by mutations in the PIK3CA gene.
  • Proteus Syndrome (PS): A rare condition characterized by overgrowth of bones, skin, and other tissues, typically caused by a mutation in the AKT1 gene.
  • AKT: A protein involved in cell signaling that plays a crucial role in regulating cell growth and survival.
  • PI3K pathway: A cellular signaling pathway involved in cell growth, proliferation, and survival, which is often overactive in certain overgrowth disorders and cancers.
  • Cerebriform Connective Tissue Nevus (CCTN): A type of skin lesion characterized by deep grooves and folds, often seen in Proteus Syndrome.
  • Adverse Event (AE): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.
  • Serious Adverse Event (SAE): An adverse event that results in death, is life-threatening, requires hospitalization, causes persistent or significant disability, or is otherwise considered medically important.
  • Pharmacokinetics (PK): The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.
  • Open-label study: A type of clinical trial where both the researchers and participants know which treatment is being administered.
  • Cycle: In these trials, a cycle typically refers to a 28-day period of treatment.

References

  1. https://clinicaltrials.gov/study/NCT03094832
  2. https://clinicaltrials.gov/study/NCT04980872
  3. https://clinicaltrials.gov/study/NCT04316546
  4. https://clinicaltrials.gov/study/NCT02594215