PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Conditions
Keywords
ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth, Congenital malformations, MOSAIC
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.
Interventions
DRUGMiransertib
Miransertib capsules administered orally either 1 hour before or 2 hours after a meal.
Sponsors
Merck Sharp & Dohme LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
None (Open-label)
Intervention model description
Multicenter
Eligibility
Sex/Gender
ALL
Age
2 Years to 120 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Australia, Brazil, Italy, United Kingdom, United States
Outcome results
None listed