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.
Överblick
Vad handlar denna studie om?
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.
Viktiga punkter
- 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.
Vem kan vara berättigad?
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.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- I am at least 2 years old.
- I have been diagnosed with PIK3CA-related overgrowth spectrum (PROS) or Proteus Syndrome.
- I am currently taking Miransertib in another study OR have been approved to start it.
- Jag är inte gravid eller ammar.
- I am not currently taking certain other specific medications (like strong immune suppressants or high-dose steroids).
Vad innebär deltagande?
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.
Potentiella risker och fördelar
Platser (11)
- Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)Verifierat postnummerAtlanta, USA
- Boston Children's Hospital ( Site 0089)Verifierat postnummerBoston, USA
- Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)Verifierat postnummerCincinnati, USA
- Texas Children's Hospital ( Site 0104)Verifierat postnummerHouston, USA
- Seattle Children's Hospital ( Site 0103)Verifierat postnummerSeattle, USA
- John Hunter Hospital ( Site 0203)Verifierat postnummerNewcastle, Australien
- Bundaberg Base Hospital ( Site 0202)Verifierat postnummerBundaberg, Australia
- Hospital Araújo Jorge ( Site 0801)Verifierat postnummerGoiânia, Brazil
- Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)Verifierat postnummerRome, Italy
- Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)Verifierat postnummerRome, Italy
- Great Ormond Street Hospital ( Site 0701)Verifierat postnummerLondon, Storbritannien
Vanliga frågor
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.
Will I get paid to be in this study?
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.
Hur du får reda på mer
Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.
Diskussion
Gemenskapsdiskussion
Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.