Clinical trial NCT04980872: 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)
Official 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Keywords: ARQ 092
ArQule
AKT
mTOR
PIK3CA
PROS
Proteus
Overgrowth
Congenital malformations
MOSAIC
Lead sponsor: Merck Sharp & Dohme LLC
Sponsor class: INDUSTRY
First posted: 2021-07-28
Enrollment: 60
Sex: ALL
Minimum age (years): 2
Maximum age (years): 120
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Multicenter
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants experiencing a Serious Adverse Event (SAE)
timeFrame: Up to approximately 94 months
measure: Number of participants discontinuing study treatment due to an Adverse Event (AE)
timeFrame: Up to approximately 90 months
Secondary outcomes:
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital ( Site 0089)
state: Massachusetts
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
state: Ohio
city: Houston
country: United States
facility: Texas Children's Hospital ( Site 0104)
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital ( Site 0103)
state: Washington
city: Newcastle
country: Australia
facility: John Hunter Hospital ( Site 0203)
state: New South Wales
city: Bundaberg
country: Australia
facility: Bundaberg Base Hospital ( Site 0202)
state: Queensland
city: Goiânia
country: Brazil
facility: Hospital Araújo Jorge ( Site 0801)
state: Goias
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
state: Lazio
city: Rome
country: Italy
facility: Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
state: Roma
city: London
country: United Kingdom
facility: Great Ormond Street Hospital ( Site 0701)
state: London, City Of
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References: