Back to resultsA 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)
Primary Purpose
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Miransertib
Sponsored by
About this trial
This is an interventional treatment trial for PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS) focused on measuring ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth, Congenital malformations, MOSAIC
Eligibility Criteria
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
Sites / Locations
- Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
- Boston Children's Hospital ( Site 0089)
- Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
- Texas Children's Hospital ( Site 0104)
- Seattle Children's Hospital ( Site 0103)
- John Hunter Hospital ( Site 0203)
- Bundaberg Base Hospital ( Site 0202)
- Hospital Araújo Jorge ( Site 0801)
- Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
- Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
- Great Ormond Street Hospital ( Site 0701)
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Miransertib
Arm 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 48 cycles. A cycle is 28 days long.
Outcomes
Primary Outcome Measures
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.
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.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04980872
Brief Title
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
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 2, 2021 (Actual)
Primary Completion Date
September 29, 2026 (Anticipated)
Study Completion Date
September 29, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Merck Sharp & Dohme LLC
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Keywords
ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth, Congenital malformations, MOSAIC
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Multicenter
Masking
None (Open Label)
Masking Description
None (Open-label)
Allocation
N/A
Enrollment
60 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Miransertib
Arm Type
Experimental
Arm 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 48 cycles. A cycle is 28 days long.
Intervention Type
Drug
Intervention Name(s)
Miransertib
Other Intervention Name(s)
MK-7075, ARQ 092
Intervention Description
Miransertib capsules administered orally either 1 hour before or 2 hours after a meal.
Primary Outcome Measure Information:
Title
Number of participants experiencing a Serious Adverse Event (SAE)
Description
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.
Time Frame
Up to approximately 4 years
Title
Number of participants discontinuing study treatment due to an Adverse Event (AE)
Description
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.
Time Frame
Up to approximately 4 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
120 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Merck Sharp & Dohme LLC
Official's Role
Study Director
Facility Information:
Facility Name
Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Boston Children's Hospital ( Site 0089)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Texas Children's Hospital ( Site 0104)
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Children's Hospital ( Site 0103)
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States
Facility Name
John Hunter Hospital ( Site 0203)
City
Newcastle
State/Province
New South Wales
ZIP/Postal Code
2305
Country
Australia
Facility Name
Bundaberg Base Hospital ( Site 0202)
City
Bundaberg
State/Province
Queensland
ZIP/Postal Code
4670
Country
Australia
Facility Name
Hospital Araújo Jorge ( Site 0801)
City
Goiânia
State/Province
Goias
ZIP/Postal Code
74605070
Country
Brazil
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
City
Roma
State/Province
Lazio
ZIP/Postal Code
00168
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
City
Rome
State/Province
Roma
ZIP/Postal Code
00165
Country
Italy
Facility Name
Great Ormond Street Hospital ( Site 0701)
City
London
State/Province
London, City Of
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
IPD Sharing URL
http://engagezone.msd.com/ds_documentation.php
Links:
URL
http://merckoncologyclinicaltrials.com
Description
Merck Oncology Clinical Trials Information
URL
https://trialstransparency.merckclinicaltrials.com/Study.aspx?id=7075-006&kw=7075-006
Description
Plain Language Summary
Learn more about this trial
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)
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