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A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

This study is investigating a medicine called Miransertib (also known as MK-7075) for people, aged 2 years and older, who have genetic conditions causing unusual growth, specifically PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome. This research focuses on understanding how safe the medicine is and how well people tolerate it over time. It's an extension study, meaning it’s for individuals who are already taking Miransertib in other research trials, or who are approved to start treatment in these trials but haven't yet. The main goal is to gather more information on Miransertib's long-term safety and whether participants experience any side effects.

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Estado
Activo, no reclutando
Fase
FASE2
Patrocinador
Merck Sharp & Dohme LLC
Objetivo de inscripción
60
Inicio
02 Nov 2021
Estimación de finalización
07 Feb 2030

¿De qué trata este estudio?

This study is about a medicine called Miransertib (also known as MK-7075). It's being studied for people who have certain health conditions that cause parts of their body to grow unusually. These conditions are called PIK3CA-related overgrowth spectrum (often shortened to PROS) and Proteus Syndrome. Both of these conditions are related to a change in a specific gene.

The main purpose of this particular study is to check how safe Miransertib is when taken for a longer time, and if people can tolerate it well. It’s important to understand if there are any side effects and how serious they might be. This study is an 'extension' of other studies looking at Miransertib. This means it’s designed for people who are already taking the medicine in other research trials, or for those who have been approved to start taking it in those trials but haven't begun yet.

By continuing to follow these participants, researchers hope to get a clear picture of Miransertib's safety profile over a longer period. This information is very important for doctors and patients when considering treatment options for PROS and Proteus Syndrome.

Puntos clave

  • This study is for people with PROS or Proteus Syndrome, aged 2 or older.
  • It's looking at the long-term safety and tolerability of the medicine Miransertib.
  • Participants will likely already be taking Miransertib in another study.
  • Regular clinic visits and monitoring will be part of taking part.
  • The study aims to gather important information for future treatment options.

¿Quién puede ser elegible?

To join this study, you must be at least 2 years old and have a diagnosis of PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome. You'll likely be eligible if you're already taking Miransertib in a different study, or if you've been approved to start in one of those studies but haven't started yet.

There are also some important rules around contraception. Men must agree to use effective contraception or avoid sex during the study and for 3 months after their last dose. Women who could become pregnant must also use very effective contraception or avoid sex. Women who are pregnant or breastfeeding cannot take part.

However, you won't be able to join if you've had to stop taking Miransertib before because of serious side effects or if it wasn't tolerable for you. You also cannot be taking certain other medications, such as specific growth pathway inhibitors, medicines that weaken your immune system, or continuous high doses of steroids.

¿Podría este estudio ser adecuado para ti?

Responde estas preguntas rápidas para ver si puedes ser elegible. Esto es solo una guía: el equipo de investigación toma la decisión final.

  1. I am at least 2 years old.
  2. I have been diagnosed with PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome.
  3. I am currently taking Miransertib in another study OR have been approved to start it.
  4. I am not pregnant or breastfeeding.
  5. I am not currently taking certain other specific medications (like strong immune suppressants or high-dose steroids).
Responde cada pregunta para ver tu resultado.

¿En qué consiste la participación?

If you decide to take part in this study, you'll be taking Miransertib, likely continuing from a previous study. You'll have regular visits to the study clinic where the research team will monitor your health. These visits will involve check-ups, probably blood tests, and discussions about how you're feeling and any side effects you might be having. The researchers will be looking closely at how your body responds to the medicine and if you experience any unexpected issues. The total duration of your participation would depend on your original study and how long this extension study runs, but it aims to gather long-term safety data.

Riesgos y beneficios potenciales

Like all medical studies, there could be potential benefits and risks. A potential benefit could be continuing access to Miransertib, which might help manage your condition. However, there's always a risk of side effects from any medication, and these will be carefully monitored by the study team. You'll be given detailed information about known side effects before you decide to join. It's very important to remember that joining a study is always voluntary, and you have the right to withdraw at any time, for any reason, without it affecting your medical care.

Locations (11)

  • Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
    Código postal verificado
    Atlanta, Estados Unidos
  • Boston Children's Hospital ( Site 0089)
    Código postal verificado
    Boston, Estados Unidos
  • Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
    Código postal verificado
    Cincinnati, Estados Unidos
  • Texas Children's Hospital ( Site 0104)
    Código postal verificado
    Houston, Estados Unidos
  • Seattle Children's Hospital ( Site 0103)
    Código postal verificado
    Seattle, United States
  • John Hunter Hospital ( Site 0203)
    Código postal verificado
    Newcastle, Australia
  • Bundaberg Base Hospital ( Site 0202)
    Código postal verificado
    Bundaberg, Australia
  • Hospital Araújo Jorge ( Site 0801)
    Código postal verificado
    Goiânia, Brazil
  • Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
    Código postal verificado
    Rome, Italy
  • Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
    Código postal verificado
    Rome, Italy
  • Great Ormond Street Hospital ( Site 0701)
    Código postal verificado
    Londres, Reino Unido

Preguntas comunes

What is PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome?

These are rare conditions where certain parts of the body grow larger than usual due to a genetic change.

What is Miransertib?

Miransertib is a medicine being tested to see if it can help manage the unusual growth seen in PROS and Proteus Syndrome by targeting certain chemical pathways in the body.

Why is this study only for people already taking Miransertib?

This study is an 'extension' study, meaning it's designed to gather more long-term safety information from people who are already receiving the medicine in other research studies.

¿Me pagarán por participar en este estudio?

The study information doesn't mention payment, but often travel costs for study visits are covered. You should ask the study team about this.

What happens if I decide I don't want to be in the study anymore?

You are free to leave the study at any time, for any reason. Your decision will not affect your regular medical care.

Cómo obtener más información

Siempre hable con su médico de cabecera o especialista antes de decidir participar en un estudio.

Discusión

Discusión comunitaria

Impulsado por nuestro foro en community.patient.info. Por favor, sea respetuoso — esto no es un consejo médico.