PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)Phase 2NCT04980872
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 Phase 2 study testing Miransertib in people with PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS), run by Merck Sharp & Dohme LLC at 11 sites in 5 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 2 to 120 years
- Conditions the study is for
- PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
- 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
- 60 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 2 Nov 2021to 7 Feb 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
United States5 sites
Children's Healthcare of Atlanta - Egleston Hospital ( Site 0107)
Atlanta, Georgia, 30322
Boston Children's Hospital ( Site 0089)
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center-Hematology ( Site 0102)
Cincinnati, Ohio, 45229
Texas Children's Hospital ( Site 0104)
Houston, Texas, 77030
Seattle Children's Hospital ( Site 0103)
Seattle, Washington, 98105
Australia2 sites
Bundaberg Base Hospital ( Site 0202)
Bundaberg, Queensland, 4670
John Hunter Hospital ( Site 0203)
Newcastle, New South Wales, 2305
Italy2 sites
Fondazione Policlinico Universitario Agostino Gemelli ( Site 0052)
Rome, Lazio, 00168
Ospedale Pediatrico Bambino Gesù-Centro Trials ( Site 0087)
Rome, Roma, 00165
United Kingdom1 site
Great Ormond Street Hospital ( Site 0701)
London, London, City of, WC1N 3JH
About this study
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
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.
- Being tested
- Miransertib
- Keywords
- ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth, Congenital malformations, MOSAIC
- Medical subject headings
- Hereditary Sensory and Autonomic Neuropathies, Proteus Infections, 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 either 1 hour before or 2 hours after a meal.
| Study group | Role | What they receive |
|---|---|---|
| Miransertib | Experimental | 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 96 cycles. A cycle is 28 days long. Receives: Drug: Miransertib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to approximately 94 months
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.
Measured: Up to approximately 90 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2021
- Study start2 Nov 2021
- Primary completion7 Feb 2030 (estimated)
- Study completion7 Feb 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)Studies of the same condition, open and under way.
- More trials run by Merck Sharp & Dohme LLCThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Multicenter
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Merck Sharp & Dohme LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04980872
- Sponsor's protocol ID
- 7075-006
- Other IDs
- MK-7075-006, 2022-500689-87-00, 2021-001369-19
- Status last verified
- Feb 2025
- First submitted
- 23 Jul 2021
- Last updated
- 7 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04980872
- Acronym / sponsor ID
- 7075-006
- Keywords
- ARQ 092, ArQule, AKT, mTOR, PIK3CA, PROS, Proteus, Overgrowth
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Merck Sharp & Dohme LLCIndustry (pharma, biotech, devices)
- Study start
- 2 Nov 2021 (actual)
- Primary completion
- 7 Feb 2030 (estimated)
- Study completion
- 7 Feb 2030 (estimated)
- First posted
- 28 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multicenter
| Arm | Type | Receives |
|---|---|---|
| Miransertib | Experimental | Drug: MiransertibParticipants 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 96 cycles. A cycle is 28 days long. |
Who may qualify
- Ages
- 2 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (5 listed)
- 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
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 either 1 hour before or 2 hours after a meal. | Drug | Miransertib |
Primary outcome
Number of participants experiencing a Serious Adverse Event (SAE)
Time frame: Up to approximately 94 months
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)
Time frame: Up to approximately 90 months
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2025.
- Merck Oncology Clinical Trials InformationLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
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 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Miransertib in people with PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS), run by Merck Sharp & Dohme LLC at 11 sites in 5 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 3 inclusion and 5 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: under way, not looking for participants, as of the sponsor's last update on 7 Feb 2025. 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, Australia, Italy, Brazil, United Kingdom. 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
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 experiencing a Serious Adverse Event (SAE) (time frame: Up to approximately 94 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?
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 7 Feb 2025.