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

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What is this study about?

This clinical trial is focused on studying the long-term safety of a treatment for two rare conditions: PIK3CA-related overgrowth spectrum (PROS) and Proteus Syndrome. These conditions are characterized by abnormal growth of body tissues. The treatment being studied is a medication called miransertib, which is taken orally in the form of a hard capsule. Miransertib is a type of drug known as a selective allosteric AKT inhibitor, which means it targets specific pathways in the body that are involved in cell growth and survival.

The purpose of this study is to evaluate how safe and tolerable miransertib is when used on its own over a long period. Participants in this study are those who are already receiving miransertib in other studies. The study will monitor participants to see if they experience any serious side effects or if they need to stop taking the medication due to any adverse effects. The study is designed to provide valuable information about the safety of miransertib for individuals with PROS or Proteus Syndrome.

Participants will continue their treatment with miransertib and will be regularly assessed by the study team. The study aims to ensure that the medication is safe for long-term use and to gather data on any potential side effects. This information will help in understanding the long-term impact of miransertib on individuals with these rare conditions.

1 joining the study

Upon joining the study, you will be informed about the purpose and procedures of the trial. This study is designed to evaluate the long-term safety of miransertib for individuals with PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome (PS).

2 initial assessment

An initial assessment will be conducted to confirm your eligibility. This includes verifying that you have been previously treated with miransertib in another study and meet the age criteria of 2 to 120 years.

3 medication administration

You will receive miransertib in the form of a hard capsule for oral use. The dosage and frequency will be determined by the study team based on your specific needs and previous treatment history.

4 regular monitoring

Throughout the study, regular monitoring will be conducted to assess your health and any potential side effects. This includes tracking any serious adverse events (SAEs) or discontinuation due to adverse events (AEs).

5 follow-up visits

You will be required to attend follow-up visits as scheduled by the study team. These visits are essential to ensure your safety and the effectiveness of the treatment.

6 end of study

The study is expected to conclude by December 15, 2025. At the end of the study, a final assessment will be conducted to evaluate your overall health and the outcomes of the treatment.

Who Can Join the Study?

The patient must have a condition called PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome (PS).
The patient must have been screened in a specific study (MK-7075-002) or be approved by the sponsor to screen for this study, or currently be treated with a medication called miransertib as part of certain studies.
The patient can be male or female and must be between 2 and 120 years old.
If the patient is male, he must agree to either not have heterosexual intercourse or use contraception, unless it is confirmed that he cannot produce sperm, during the study and for at least 90 days after the last dose of the study medication.
If the patient is female, she must not be pregnant or breastfeeding. If she can have children, she must agree to either not have sexual intercourse or use a highly effective method of contraception.

Who Cannot Join the Study?

Patients with any other serious medical condition that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients who have a known allergy to the study medication or its ingredients.
Patients who are unable to comply with the study procedures.
Patients with a history of drug or alcohol abuse within the past year.
Patients with certain heart conditions that could be affected by the study medication.
Patients who are taking medications that might interact with the study drug.
Patients with a history of certain types of cancer.
Patients with uncontrolled diabetes or high blood pressure.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country	Status
Ospedale Pediatrico Bambino Gesu’	Rome	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	01.06.2022

Trial locations

Investigated drugs:

Miransertib

Miransertib is a medication being studied for its safety and how well people can tolerate it when used alone. It is being tested in people who have conditions called PROS or Proteus Syndrome. These are rare disorders that can cause overgrowth of body tissues. The goal of using miransertib is to see if it can help manage these conditions safely over a long period. Participants in this trial are already taking miransertib in other studies, and this trial aims to continue monitoring its effects on them.

Investigated diseases:

PIK3CA-related overgrowth spectrum (PROS) – This is a group of disorders caused by mutations in the PIK3CA gene, leading to abnormal cell growth. It results in overgrowth of various tissues in the body, which can include skin, bones, fat, and blood vessels. The overgrowth can be asymmetric, meaning it affects one side of the body more than the other. Symptoms can vary widely depending on which tissues are affected. The condition can lead to complications such as pain, mobility issues, and functional impairments. The progression of the disease is typically slow and can continue throughout life.

Proteus Syndrome – This is a rare condition characterized by overgrowth of bones, skin, and other tissues. The overgrowth is often asymmetric and can affect any part of the body. It is caused by a mutation in the AKT1 gene, which occurs after conception and is not inherited. The syndrome can lead to a variety of symptoms, including skin lesions, skeletal abnormalities, and vascular malformations. The progression of Proteus Syndrome is unpredictable and varies greatly among individuals. Over time, the overgrowth can result in significant physical deformities and functional challenges.

Trial ID:

2022-500689-87-00

Protocol code:

MK-7075-006

Trial Phase:

Therapeutic exploratory (Phase II)

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