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 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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS) Bacterial Infections Bacterial Infections and Mycoses Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Enterobacteriaceae Infections Genetic Diseases, Inborn Gram-Negative Bacterial Infections Hereditary Sensory and Autonomic Neuropathies Heredodegenerative Disorders, Nervous System Infections Nervous System Diseases Nervous System Malformations Neurodegenerative Diseases Neuromuscular Diseases Peripheral Nervous System Diseases Polyneuropathies Proteus Infections

Age range

2 year–120 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

John Hunter Hospital ( Site 0203), Newcastle, New South Wales, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Miransertib

Drug

Miransertib capsules administered orally either 1 hour before or 2 hours after a meal.

Other names: MK-7075, ARQ 092

Primary outcomes

Number of participants experiencing a Serious Adverse Event (SAE)

Time frame: 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)

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Merck Sharp & Dohme LLC

Industry

Registry information

Official study title

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

Important dates

Study start: 2021
Primary completion: 2030
Study completion: 2030
First posted: Jul 28, 2021
Registry last updated: Feb 7, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Active, Not Recruiting