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)

Study Overview
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Brief Summary
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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.

Official Title
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A Multicenter, Open-label, Phase 2, Extension Trial to Study the Long-term Safety in Participants With PROS or Proteus Syndrome Who Are Currently Being Treated With Miransertib in Other Studies

Conditions
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PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)

Intervention / Treatment
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* Drug: Miransertib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Has PROS or PS and is currently being actively treated with miransertib as part of Study MK-7075-002 (NCT03094832) or ArQule's Compassionate Use and Extended Access Program (CU/EAP, NCT03317366) Exclusion Criteria: Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib Received other investigational agents, if any, that were administered between leaving Study MK-7075-02 or ArQule's CU/EAP and entering this trial Receives inhibitors of the mechanistic target of rapamycin (mTOR) pathway (eg, sirolimus, everolimus) Receives immunosuppressive therapies Receives continuous high dose steroids

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 120 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Multicenter
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Miransertib<br>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 48 cycles. A cycle is 28 days long. | Drug: Miransertib<br>* Miransertib capsules administered orally either 1 hour before or 2 hours after a meal.<br>* Other names: ARQ 092;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants experiencing a Serious Adverse Event (SAE) | 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. | Up to approximately 4 years | 
| Number of participants discontinuing study treatment due to an Adverse Event (AE) | 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. | Up to approximately 4 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth, Congenital malformations, MOSAIC