PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus SyndromePhase 1/2MOSAICNCT03094832
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
A Phase 1/2 study testing Miransertib in people with PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome, run by ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA) at 11 sites in 4 countries.
Trial summary
This is an open label, Phase 1/2 study 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) and Proteus Syndrome (PS) (MOSAIC). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
- What is being tested?
- Miransertib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 16 May 2017to 11 Apr 2022
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Part A
- Signed informed consent and, when applicable, signed assent
- Male or female participants ≥ 2 years old with body surface area (BSA) of ≥ 0.33 m^2
- Have a clinical diagnosis of PROS or PS with documented somatic PIK3CA or serine-threonine protein kinase (AKT1) mutations
- Archival or fresh overgrowth tissue sample available to be shipped to Sponsor or designee
- Have poor prognosis, significant morbidity, and/or progressive disease (e.g., worsening of the disease/increase in number or size of the overgrowth lesions in the last 12 months)
Show all 27Show fewer
- Have measurable disease (at least one overgrowth lesion that can be accurately measured in size by imaging and/or linear or circumference measure)
- Adequate organ function based on screening laboratory values
- If a female is of child-bearing potential, documentation of a negative pregnancy test is required prior to enrollment. Sexually active participants (male and female) must agree to use double-barrier contraceptive measures, oral contraception, or avoidance of intercourse while on study and for up to 90 days after ending treatment
- Ability to complete the Quality of Life (QoL) questionnaires by the participant or his/her caregiver
- Part B:
- Signed consent form and when applicable, signed assent
- Archival or fresh overgrowth tissue sample available to be shipped to Sponsor or designee
- Except for Cohort 4, clinically progressive or worsening disease defined as an increase in number or size of the overgrowth lesion(s) in the last 6 months as assessed by the Investigator
- Adequate organ function based on screening laboratory values
- Male or female participants of child-producing potential must agree to use double-barrier contraceptive measures, oral contraception, or avoidance of intercourse during the study and for 90 days after the last dose of miransertib
- Ability to complete the study questionnaires by the participant or his/her caregiver
- Cohort 1 (PROS) specific criteria
- Male or female participants ≥ 2 years and ≤30 years of age with BSA of ≥ 0.33 m2
- Have clinical diagnosis of PROS per Diagnostic Criteria for PROS and documented somatic PIK3CA variant
- Have at least one lesion that can be measured by study- standardized volumetric MRI (eligibility to be confirmed by blinded independent central imaging review
- Cohort 2 (PS) specific criteria
- Male or female participants ≥ 2 years and ≤18 years of age with BSA of ≥ 0.33 m2
- Have clinical diagnosis of PS per Diagnostic Criteria for PS and documented somatic AKT1 variant
- Have at least one plantar cerebriform connective tissue nevus (CCTN) and pre-CCTN lesion that can be measured by standardized photography
- Cohort 3 specific criteria: Male or female participants ≥2 years old with BSA of ≥ 0.33 m2 and who fail to meet the eligibility criteria for Cohorts 1 or 2
- Cohort 4 (PROS or PS) specific criteria: participants previously treated with miransertib or currently receiving miransertib under Compassionate Use/Expanded Access. Participants should meet the age criterion by/on the date of the first dose, Cycle 1 Day 1
Who cannot join
- Part A:
- History of Type 1 or 2 uncontrolled diabetes mellitus requiring regular medication (other than metformin or other oral hypoglycemic agents) or fasting glucose ≥ 160 mg/dL (if > 12 years old) and ≥ 180 mg/dL (if ≤ 12 years old) at the screening visit
- History of significant cardiac disorders:
- Myocardial infarction (MI) or congestive heart failure defined as Class II-IV per the New York Heart Association (NYHA) classification within 6 months of the first dose of miransertib (MI occurring > 6 months of the first dose of miransertib will be permitted)
- Grade 2 (per NCI CTCAE version 4.03) or worse conduction defect (e.g., right or left bundle branch block); left ventricular ejection fraction (LVEF) < 50% assessed by echocardiogram/multigated acquisition (MUGA) scan
- Major surgery, radiotherapy, or immunotherapy within four weeks of the first dose of miransertib
Show all 22Show fewer
- Any experimental systemic therapy for the purpose of treating PROS or PS (e.g., sirolimus, everolimus, high dose steroids) within two weeks of the first dose of miransertib, except for participants who were previously or are currently treated with miransertib under a Compassionate Use/Expanded Access program
- Intolerance of or severe toxicity attributed to v-Akt murine thymoma viral oncogene homolog (AKT) inhibitors (e.g., miransertib, uprosertib, afuresertib, ipatasertib)
- Concurrent severe uncontrolled illness not related to PROS or PS (ongoing or active infection, known human immunodeficiency virus (HIV) infection, malabsorption syndrome, psychiatric illness/substance abuse/social situation that would limit compliance with study requirements)
- Pregnant or breastfeeding
- Inability to comply with study evaluations or to follow drug administration guidelines
- Part B
- History of Type 1 diabetes mellitus or Type 2 uncontrolled diabetes mellitus requiring regular medication (other than metformin or other oral hypoglycemic agents) or fasting glucose ≥ 160 mg/dL (if > 12 years old) and ≥ 180 mg/dL (if ≤ 12 years old) at the screening visit
- History of significant cardiac disorders:
- Myocardial infarction (MI) or congestive heart failure defined as Class II-IV per the New York Heart Association (NYHA) classification within 6 months of the first dose of miransertib (MI occurring > 6 months of the first dose of miransertib will be permitted)
- Grade 2 (per current version of National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE]) or worse conduction defect (e.g., right or left bundle branch block)
- Major surgery or locoregional therapy within four weeks of the first dose of miransertib
- Any experimental systemic therapy for the purposes of treating PROS or PS (e.g., sirolimus, everolimus, high dose steroids) within two weeks of the first dose of miransertib
- Intolerance of or severe toxicity attributed to AKT inhibitors (e.g., miransertib, uprosertib, afuresertib, ipatasertib)
- Concurrent severe uncontrolled illness not related to PROS or PS (e.g. ongoing or active infection, known HIV infection, malabsorption syndrome, psychiatric illness/substance abuse/social situation that would limit compliance with study requirements)
- Pregnant or breastfeeding
- Inability to comply with study evaluations or to follow drug administration guidelines
Trial locations and contacts
11 sites in 4 countries.
11 sites in 4 countries
United States6 sites
Children's Hospital of Atlanta ( Site 0107)
Atlanta, Georgia, 30342
Boston Children's Hospital ( Site 0089)
Boston, Massachusetts, 02115
Ann & Robert H. Lurie Children's Hospital of Chicago ( Site 0101)
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center ( Site 0102)
Cincinnati, Ohio, 45229
Texas Children's Hospital ( Site 0104)
Houston, Texas, 77030
Seattle Childrens Hospital ( Site 0103)
Seattle, Washington, 98105
Italy3 sites
Universita di Catania ( Site 0088)
Catania, 95123
Fondazione Policlinico Universitario A. Gemelli ( Site 0052)
Roma, 00168
Ospedale Pediatrico Bambino Gesu ( Site 0087)
Rome, Roma, 00165
Australia1 site
Hunter Genetics ( Site 0201)
Waratah NSW, New South Wales, 2298
Spain1 site
Hospital Sant Joan ( Site 0601)
Esplugues de Llobregat, Barcelona, 08950
About this study
Official title: A Phase 1/2 Study of ARQ 092 (Miransertib) in Subjects With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome
This is an open label, Phase 1/2 study 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) and Proteus Syndrome (PS) (MOSAIC).
Read the detailed description
The study consists of two parts: Part A and Part B. Part A was closed to enrollment under Amendment 6. As of Amendment 7, the endpoints for Part A and Part B have been combined to assess the safety and tolerability of miransertib in participants with PROS and PS. Previous efficacy and pharmacokinetic (PK) objectives and endpoints have been removed. Amendment 7 will complete the final enrollment into the MOSAIC study and Compassionate Use/Expanded Access Program.
- Being tested
- Miransertib
- Keywords
- ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations, Miransertib, MOSAIC
- Medical subject headings
- Hereditary Sensory and Autonomic Neuropathies, Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MiransertibDrug
Also called MK-7075, ARQ 092
Miransertib capsules administered orally at an initial dose of 15 mg/m^2 or 25 mg/m^2 QD and then titrated up to 25 mg/m^2 or 35 mg/m^2 QD at the investigator's discretion.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Miransertib PROS/PS | Experimental | During Cycles 1-3, participants with either PROS (phosphatidylinositol- 4,5-bisphosphate 3-kinase, catalytic subunit alpha [PIK3CA]-related Overgrowth Spectrum) or PS (Proteus syndrome) received miransertib 15 mg/m^2 once daily (QD) (each cycle length = 28 days). From Cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 and then titrated to 35 mg/m^2 orally QD at the investigator's discretion. Receives: Drug: Miransertib |
| Part B: Miransertib PROS (Cohort 1) | Experimental | During Cycles 1-3, participants with PROS who have a measurable lesion by volumetric magnetic resonance imaging (MRI) received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. Receives: Drug: Miransertib |
| Part B: Miransertib PS (Cohort 2) | Experimental | During Cycles 1-3, participants with PS who have a measurable lesion by standardized digital photography received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. Receives: Drug: Miransertib |
| Part B: Miransertib PROS/PS (Cohort 3) | Experimental | During Cycles 1-3, participants with PROS or PS who do not meet all the eligibility criteria for Cohorts 1 or 2 received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. Receives: Drug: Miransertib |
| Part B: Miransertib Compassionate Use/Expanded Access (Cohort 4) | Experimental | During cycles 1-48 (each cycle length = 28 days) or until disease progression, unacceptable toxicity, or discontinuation, participants previously treated with miransertib or currently receiving miransertib under Compassionate Use/Expanded Access continued to receive the current dose of miransertib (did not exceed 25 mg/m^2). Receives: Drug: Miransertib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Experienced an Adverse Event (AE)
An AE was 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.
Measured: Up to approximately 48 months
Number of Participants Who Discontinued Study Treatment Due to an AE
An AE was 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.
Measured: Up to approximately 45 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Mar 2017
- Study start16 May 2017
- Primary completion11 Apr 2022
- Study completion11 Apr 2022
Why it stopped: Early termination due to business reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03094832
- Sponsor's protocol ID
- 7075-002
- Other IDs
- MOSAIC, ARQ 092-103, MK-7075-002, 2016-000558-37, 2016
- Acronym
- MOSAIC
- Status last verified
- Apr 2023
- First submitted
- 17 Mar 2017
- Last updated
- 13 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
- Expanded access
- Available
Publications
Eng W, Iacobas I, Perkins J, Zampino G, Leoni C, Buonuomo PS, Simonetti A, Goel H, Briones M, Huang M, Goldmacher G, Liaw D, Hammill A. Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome. Orphanet J Rare Dis. 2025 Jul 25;20(1):375. doi: 10.1186/s13023-025-03831-z.
PubMed 40713644Forde K, Resta N, Ranieri C, Rea D, Kubassova O, Hinton M, Andrews KA, Semple R, Irvine AD, Dvorakova V. Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome. Orphanet J Rare Dis. 2021 Feb 27;16(1):109. doi: 10.1186/s13023-021-01745-0.
PubMed 33639990
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of ARQ 092 (Miransertib) in Subjects With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome
- ClinicalTrials.gov ID
- NCT03094832
- Acronym / sponsor ID
- MOSAIC / 7075-002
- Keywords
- ARQ 092, ArQule, AKT, PIK3CA, Overgrowth, Congenital malformations, Miransertib, MOSAIC
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Early termination due to business reasons
- Enrolment
- 50 participants (actual)
- Lead sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)Industry (pharma, biotech, devices)
- Collaborators
- Worldwide Clinical Trials
- Study start
- 16 May 2017 (actual)
- Primary completion
- 11 Apr 2022 (actual)
- Study completion
- 11 Apr 2022 (actual)
- First posted
- 29 Mar 2017 (actual)
- Results
- Posted 31 Mar 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label, Phase 1/2 study 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) and Proteus Syndrome (PS) (MOSAIC).
Detailed description
The study consists of two parts: Part A and Part B. Part A was closed to enrollment under Amendment 6. As of Amendment 7, the endpoints for Part A and Part B have been combined to assess the safety and tolerability of miransertib in participants with PROS and PS. Previous efficacy and pharmacokinetic (PK) objectives and endpoints have been removed. Amendment 7 will complete the final enrollment into the MOSAIC study and Compassionate Use/Expanded Access Program.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Part A: Miransertib PROS/PS | Experimental | Drug: MiransertibDuring Cycles 1-3, participants with either PROS (phosphatidylinositol- 4,5-bisphosphate 3-kinase, catalytic subunit alpha [PIK3CA]-related Overgrowth Spectrum) or PS (Proteus syndrome) received miransertib 15 mg/m^2 once daily (QD) (each cycle length = 28 days). From Cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 and then titrated to 35 mg/m^2 orally QD at the investigator's discretion. |
| Part B: Miransertib PROS (Cohort 1) | Experimental | Drug: MiransertibDuring Cycles 1-3, participants with PROS who have a measurable lesion by volumetric magnetic resonance imaging (MRI) received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. |
| Part B: Miransertib PS (Cohort 2) | Experimental | Drug: MiransertibDuring Cycles 1-3, participants with PS who have a measurable lesion by standardized digital photography received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. |
| Part B: Miransertib PROS/PS (Cohort 3) | Experimental | Drug: MiransertibDuring Cycles 1-3, participants with PROS or PS who do not meet all the eligibility criteria for Cohorts 1 or 2 received miransertib 15 mg/m^2 QD (each cycle length = 28 days). From cycles 4-48 or until disease progression, unacceptable toxicity, or discontinuation, participants received miransertib dose titrated to 25 mg/m^2 orally QD at the investigator's discretion. |
| Part B: Miransertib Compassionate Use/Expanded Access (Cohort 4) | Experimental | Drug: MiransertibDuring cycles 1-48 (each cycle length = 28 days) or until disease progression, unacceptable toxicity, or discontinuation, participants previously treated with miransertib or currently receiving miransertib under Compassionate Use/Expanded Access continued to receive the current dose of miransertib (did not exceed 25 mg/m^2). |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (27 listed)
- Part A
- Signed informed consent and, when applicable, signed assent
- Male or female participants ≥ 2 years old with body surface area (BSA) of ≥ 0.33 m^2
- Have a clinical diagnosis of PROS or PS with documented somatic PIK3CA or serine-threonine protein kinase (AKT1) mutations
- Archival or fresh overgrowth tissue sample available to be shipped to Sponsor or designee
Key exclusion criteria (22 listed)
- History of Type 1 or 2 uncontrolled diabetes mellitus requiring regular medication (other than metformin or other oral hypoglycemic agents) or fasting glucose ≥ 160 mg/dL (if > 12 years old) and ≥ 180 mg/dL (if ≤ 12 years old) at the screening visit
- History of significant cardiac disorders:
- Major surgery, radiotherapy, or immunotherapy within four weeks of the first dose of miransertib
- Any experimental systemic therapy for the purpose of treating PROS or PS (e.g., sirolimus, everolimus, high dose steroids) within two weeks of the first dose of miransertib, except for participants who were previously or are currently treated with miransertib under a Compassionate Use/Expanded Access program
- Intolerance of or severe toxicity attributed to v-Akt murine thymoma viral oncogene homolog (AKT) inhibitors (e.g., miransertib, uprosertib, afuresertib, ipatasertib)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MiransertibAlso: MK-7075, ARQ 092Miransertib capsules administered orally at an initial dose of 15 mg/m^2 or 25 mg/m^2 QD and then titrated up to 25 mg/m^2 or 35 mg/m^2 QD at the investigator's discretion. | Drug | Part A: Miransertib PROS/PS, Part B: Miransertib Compassionate Use/Expanded Access (Cohort 4), Part B: Miransertib PROS (Cohort 1), Part B: Miransertib PROS/PS (Cohort 3), Part B: Miransertib PS (Cohort 2) |
Primary outcome
Number of Participants Who Experienced an Adverse Event (AE)
Time frame: Up to approximately 48 months
An AE was 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.
Number of Participants Who Discontinued Study Treatment Due to an AE
Time frame: Up to approximately 45 months
An AE was 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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40713644Eng W, Iacobas I, Perkins J, Zampino G, Leoni C, Buonuomo PS, Simonetti A, Goel H, Briones M, Huang M, Goldmacher G, Liaw D, Hammill A. Safety findings from the phase 1/2 MOSAIC study of miransertib for patients with PIK3CA-related overgrowth spectrum or Proteus syndrome. Orphanet J Rare Dis. 2025 Jul 25;20(1):375. doi: 10.1186/s13023-025-03831-z.
- Derived from this study: PubMed 33639990Forde K, Resta N, Ranieri C, Rea D, Kubassova O, Hinton M, Andrews KA, Semple R, Irvine AD, Dvorakova V. Clinical experience with the AKT1 inhibitor miransertib in two children with PIK3CA-related overgrowth syndrome. Orphanet J Rare Dis. 2021 Feb 27;16(1):109. doi: 10.1186/s13023-021-01745-0.
- Individual participant dataSharing planned: Yes. http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
Related trials for PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Active, not recruitingPhase 2
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)
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Merck Sharp & Dohme LLC
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Miransertib in people with PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome, run by ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA) at 11 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 27 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 13 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Italy, Australia, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Mar 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Experienced an Adverse Event (AE) (time frame: Up to approximately 48 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03094832, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2023.