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)

Merck Sharp & Dohme (MSD)

Status and phase: Active, not recruiting; Phase 2

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

Treatments: Drug: Miransertib

Study type: Interventional

Funder types: Industry

Identifiers

NCT04980872

MK-7075-006 (Other Identifier)

7075-006

2021-001369-19 (EudraCT Number)

2022-500689-87-00 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: All

Ages: 2 to 120 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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60 participants in 1 patient group

Miransertib

Experimental group

Description:

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.

Treatment:

Drug: Miransertib