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)
- Conditions
- PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
- Interventions
- Registration Number
- NCT04980872
- Lead Sponsor
- Merck Sharp & Dohme LLC
- Brief Summary
This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related overgrowth spectrum (PROS) or Proteus Syndrome (PS). This is an extension of other miransertib studies (MK-7075-002 \[NCT03094832\] or ArQule CU/EAP \[NCT03317366\]), and may also enroll participants who are approved for MK-7075-002 but have not yet started miransertib therapy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 60
- Has PROS or PS and has been screened in Study MK-7075-002 (or has been approved by the Sponsor to screen for MK-7075-002) or is currently being treated with miransertib as part of Study MK-7075-002 (NCT03094832) or ArQule's CU/EAP (NCT03317366)
- For males, agrees to be abstinent from heterosexual intercourse or use contraception unless confirmed to be azoospermic during the study period and for ≥90 days after the last dose of study intervention
- For females, is not pregnant or breastfeeding, and is either not a participant of childbearing potential (POCBP) or is a POCBP and is abstinent or uses a highly effective method of contraception
- Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib
- Has received other investigational agents, if any, that were administered between leaving Study MK-7075-02 or ArQule's CU/EAP and entering this trial
- Is receiving systemic inhibitors of the mechanistic target of rapamycin (mTOR) pathway (eg, sirolimus, everolimus)
- Is receiving immunosuppressive therapies
- Is receiving continuous high dose steroids
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Miransertib Miransertib Participants with either PROS or PS receive miransertib orally once daily between 5 and 35 mg/m\^2 based on prior approved dosing for up to 96 cycles. A cycle is 28 days long.
- Primary Outcome Measures
Name Time Method Number of participants experiencing a Serious Adverse Event (SAE) Up to approximately 94 months An SAE is defined as any untoward medical occurrence associated with the use of a drug in a participant, whether or not considered drug related. An SAE can therefore be any such event that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is considered by the investigator to be an important medical event.
Number of participants discontinuing study treatment due to an Adverse Event (AE) Up to approximately 90 months An AE is defined as any untoward medical occurrence associated with the use of a drug in a participant, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product and does not imply any judgment about causality.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (11)
Texas Children's Hospital ( Site 0104)
🇺🇸Houston, Texas, United States
Seattle Children's Hospital ( Site 0103)
🇺🇸Seattle, Washington, United States
Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
🇮🇹Rome, Roma, Italy
Boston Children's Hospital ( Site 0089)
🇺🇸Boston, Massachusetts, United States
Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
🇺🇸Atlanta, Georgia, United States
Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
🇺🇸Cincinnati, Ohio, United States
John Hunter Hospital ( Site 0203)
🇦🇺Newcastle, New South Wales, Australia
Bundaberg Base Hospital ( Site 0202)
🇦🇺Bundaberg, Queensland, Australia
Hospital Araújo Jorge ( Site 0801)
🇧🇷Goiânia, Goias, Brazil
Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
🇮🇹Roma, Lazio, Italy
Great Ormond Street Hospital ( Site 0701)
🇬🇧London, London, City Of, United Kingdom