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)

NCT04980872 | Active Not Recruiting Phase 2 FDA Drug

• 4 other identifiers: 7075-006MK-7075-0062022-500689-87-002021-001369-19
• Study Type: interventional
• Target: 60
• Locations: 5 countries
• Sites: 11

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.

Conditions

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

Keywords

ARQ 092; ArQule; AKT; mTOR; PIK3CA; PROS; Proteus; Overgrowth; Congenital malformations; MOSAIC

Outcome Measures

Primary Outcomes (2)

• 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 94 months

• 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 90 months

Study Arms (1)

Miransertib

EXPERIMENTAL

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.

Drug: Miransertib

Interventions

Miransertib DRUG

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

Also known as: MK-7075, ARQ 092

Miransertib

Eligibility Criteria

• Age: 2 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: lead

Study Sites (11)

Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)

Atlanta, Georgia, 30322, United States

Location

Boston Children's Hospital ( Site 0089)

Boston, Massachusetts, 02115, United States

Location

Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)

Cincinnati, Ohio, 45229, United States

Location

Texas Children's Hospital ( Site 0104)

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital ( Site 0103)

Seattle, Washington, 98105, United States

Location

John Hunter Hospital ( Site 0203)

Newcastle, New South Wales, 2305, Australia

Location

Bundaberg Base Hospital ( Site 0202)

Bundaberg, Queensland, 4670, Australia

Location

Hospital Araújo Jorge ( Site 0801)

Goiânia, Goiás, 74605070, Brazil

Location

Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)

Rome, Lazio, 00168, Italy

Location

Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)

Rome, Roma, 00165, Italy

Location

Great Ormond Street Hospital ( Site 0701)

London, London, City of, WC1N 3JH, United Kingdom

Location

Related Links

• Merck Oncology Clinical Trials Information
• Plain Language Summary

MeSH Terms

Conditions

Hereditary Sensory and Autonomic Neuropathies; Proteus Infections; Congenital Abnormalities

Interventions

Miransertib

Condition Hierarchy (Ancestors)

Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Enterobacteriaceae Infections; Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Study Officials

• Medical Director

  Merck Sharp & Dohme LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: None (Open-label)
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multicenter

Study Record Dates

• First Submitted: July 23, 2021
• First Posted: July 28, 2021
• Study Start: November 2, 2021
• Primary Completion (Estimated): February 7, 2030
• Study Completion (Estimated): February 7, 2030
• Last Updated: February 7, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will share

Locations

AU (2); IT (2); BR (1); US (5); GB (1)