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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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ClinicalTrials.gov Identifier: NCT04980872
Recruitment Status : Recruiting
First Posted : July 28, 2021
Last Update Posted : August 15, 2022
Sponsor:
Information provided by (Responsible Party):
Merck Sharp & Dohme LLC

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.

Condition or disease Intervention/treatment Phase
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS) Drug: Miransertib Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Multicenter
Masking: None (Open Label)
Masking Description: None (Open-label)
Primary Purpose: Treatment
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
Actual Study Start Date : November 2, 2021
Estimated Primary Completion Date : December 15, 2025
Estimated Study Completion Date : December 15, 2025


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




Primary Outcome Measures :
  1. Number of participants experiencing a Serious Adverse Event (SAE) [ Time Frame: Up to approximately 4 years ]
    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.

  2. Number of participants discontinuing study treatment due to an Adverse Event (AE) [ Time Frame: Up to approximately 4 years ]
    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.



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Ages Eligible for Study:   2 Years to 120 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04980872


Contacts
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Contact: Toll Free Number 1-888-577-8839 Trialsites@merck.com

Locations
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United States, Georgia
Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107) Active, not recruiting
Atlanta, Georgia, United States, 30322
United States, Massachusetts
Boston Children's Hospital ( Site 0089) Active, not recruiting
Boston, Massachusetts, United States, 02115
United States, Ohio
Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102) Active, not recruiting
Cincinnati, Ohio, United States, 45229
United States, Texas
Texas Children's Hospital ( Site 0104) Active, not recruiting
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital ( Site 0103) Active, not recruiting
Seattle, Washington, United States, 98105
Australia, New South Wales
John Hunter Hospital ( Site 0203) Active, not recruiting
Newcastle, New South Wales, Australia, 2305
Australia, Queensland
Bundaberg Base Hospital ( Site 0202) Active, not recruiting
Bundaberg, Queensland, Australia, 4670
Brazil
Hospital Araújo Jorge ( Site 0801) Active, not recruiting
Goiânia, Goias, Brazil, 74605070
Italy
Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052) Recruiting
Roma, Lazio, Italy, 00168
Contact: Study Coordinator    +39063381344      
Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087) Active, not recruiting
Rome, Roma, Italy, 00165
United Kingdom
Great Ormond Street Hospital ( Site 0701) Active, not recruiting
London, London, City Of, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Merck Sharp & Dohme LLC
Investigators
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Study Director: Medical Director Merck Sharp & Dohme LLC
Additional Information:
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Responsible Party: Merck Sharp & Dohme LLC
ClinicalTrials.gov Identifier: NCT04980872    
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 )
First Posted: July 28, 2021    Key Record Dates
Last Update Posted: August 15, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
URL: http://engagezone.msd.com/ds_documentation.php

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Merck Sharp & Dohme LLC:
ARQ 092
ArQule
AKT
mTOR
PIK3CA
PROS
Proteus
Overgrowth
Congenital malformations
MOSAIC
Additional relevant MeSH terms:
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Proteus Infections
Proteus Syndrome
Syndrome
Disease
Pathologic Processes
Enterobacteriaceae Infections
Gram-Negative Bacterial Infections
Bacterial Infections
Bacterial Infections and Mycoses
Infections
Hamartoma Syndrome, Multiple
Hamartoma
Neoplasms
Neoplasms, Multiple Primary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Limb Deformities, Congenital
Musculoskeletal Abnormalities
Abnormalities, Multiple
Congenital Abnormalities