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)

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
• Intervention / Treatment: Drug: Miransertib

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Newcastle, New South Wales, Australia, 2305
      • John Hunter Hospital ( Site 0203)
  • Queensland
    • Bundaberg, Queensland, Australia, 4670
      • Bundaberg Base Hospital ( Site 0202)
• Brazil
  • Goias
    • Goiânia, Goias, Brazil, 74605070
      • Hospital Araújo Jorge ( Site 0801)
• Italy
  • Lazio
    • Roma, Lazio, Italy, 00168
      • Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
  • Roma
    • Rome, Roma, Italy, 00165
      • Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
• United Kingdom
  • London, City Of
    • London, London, City Of, United Kingdom, WC1N 3JH
      • Great Ormond Street Hospital ( Site 0701)
• United States
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital ( Site 0089)
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital ( Site 0104)
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital ( Site 0103)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 120 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Miransertib; 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; Miransertib capsules administered orally either 1 hour before or 2 hours after a meal.; Other Names: MK-7075; ARQ 092

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Merck Sharp & Dohme LLC
• Investigators: Study Director: Medical Director, Merck Sharp & Dohme LLC

Publications and helpful links

Helpful Links

• Merck Oncology Clinical Trials Information
• Plain Language Summary

Study record dates

Study Major Dates

• Study Start (Actual): November 2, 2021
• Primary Completion (Estimated): September 29, 2026
• Study Completion (Estimated): September 29, 2026

Study Registration Dates

• First Submitted: July 23, 2021
• First Submitted That Met QC Criteria: July 23, 2021
• First Posted (Actual): July 28, 2021

Study Record Updates

• Last Update Posted (Actual): July 3, 2023
• Last Update Submitted That Met QC Criteria: June 28, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: PIK3CA; mTOR; AKT; Congenital malformations; PROS; ArQule; Overgrowth; ARQ 092; MOSAIC; Proteus
• Additional Relevant MeSH Terms: Pathologic Processes; Infections; Neoplasms; Disease; Congenital Abnormalities; Musculoskeletal Diseases; Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Bone Diseases; Musculoskeletal Abnormalities; Abnormalities, Multiple; Enterobacteriaceae Infections; Bone Diseases, Developmental; Limb Deformities, Congenital; Hamartoma Syndrome, Multiple; Hamartoma; Neoplasms, Multiple Primary; Syndrome; Proteus Infections; Proteus Syndrome
• Other Study ID Numbers: 7075-006; MK-7075-006 (Other Identifier: Merck); 2022-500689-87-00 (Registry Identifier: EU CT); 2021-001369-19 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No