Miransertib for Proteus Syndrome

Not currently recruiting at 11 trial locations

Overseen By Toll Free Number

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Merck Sharp & Dohme Corp.

Must not be taking: mTOR inhibitors, Immunosuppressives, Steroids

Disqualifiers: Discontinued miransertib, Investigational agents, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 96 cycles (28 days per cycle)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests an oral medication called miransertib to determine its safety and tolerability for individuals with Proteus Syndrome or PROS, a condition causing abnormal tissue growth. The study extends to those who participated in previous miransertib trials or have received approval to begin it. Suitable candidates include those with Proteus Syndrome or PROS already involved in related studies. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as systemic inhibitors of the mTOR pathway, immunosuppressive therapies, and continuous high dose steroids.

Is there any evidence suggesting that miransertib is likely to be safe for humans?

Research has shown that miransertib is safe and easy to handle for people with PROS or Proteus Syndrome. In previous studies, participants with these conditions took miransertib and found it manageable, with no major safety issues reported. Another study supported these results, indicating that both adults and children could use the treatment without problems. Overall, the evidence so far suggests that miransertib is a safe choice for those considering joining a clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Miransertib is unique because it targets the root cause of Proteus Syndrome by inhibiting the AKT1 enzyme, which plays a crucial role in the abnormal cell growth seen in this condition. Unlike standard treatments that primarily focus on managing symptoms or complications, Miransertib directly addresses the underlying molecular pathways. Researchers are excited about Miransertib's potential to not only slow disease progression but also to improve overall quality of life for patients with Proteus Syndrome through its targeted approach.

What evidence suggests that miransertib might be an effective treatment for Proteus Syndrome?

Research has shown that miransertib might help treat Proteus Syndrome (PS) and PIK3CA-related overgrowth spectrum (PROS). In some studies, patients experienced benefits such as reduced pain and smaller growths in connective tissue. One report noted improved overall health and better movement in areas like the ankle and spine after nearly a year of treatment. Early findings suggest that taking 5 mg/m²/day of miransertib can be beneficial. These results indicate that miransertib could be a promising treatment option for people with these conditions. Participants in this trial will take miransertib orally once daily, with dosing based on previously approved levels, for up to 96 cycles. ^{1 2 4 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Merck Sharp & Dohme LLC

Are You a Good Fit for This Trial?

This trial is for people at least 2 years old with PROS or Proteus Syndrome who are already taking Miransertib in other studies. It's not open to those on mTOR inhibitors like sirolimus, high dose steroids, immunosuppressives, or anyone who stopped Miransertib due to severe side effects.

Inclusion Criteria

I am currently being treated with miransertib for PROS or PS as part of a specific study or program.

Exclusion Criteria

I stopped taking miransertib because of severe side effects or intolerance.

I am on a high dose of steroids regularly.

I am currently taking medication that targets the mTOR pathway.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral miransertib once daily between 5 and 35 mg/m^2 based on prior approved dosing for up to 96 cycles

Up to 96 cycles (28 days per cycle)

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to approximately 94 months

Open-label extension

Participants continue to receive miransertib as part of the long-term safety study

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Miransertib

Trial Overview

The study tests the safety and tolerability of an oral drug called Miransertib (MK-7075) for patients with PIK3CA-related overgrowth spectrum or Proteus Syndrome. This extends previous trials and includes new participants approved but not yet started on treatment.

How Is the Trial Designed?

Group I: Miransertib Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Merck Sharp & Dohme Corp.

Lead Sponsor

Trials

2,287

Recruited

4,582,000+

Chirfi Guindo

Merck Sharp & Dohme Corp.

Chief Medical Officer

Engineering degree from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis

Merck Sharp & Dohme Corp.

Chief Executive Officer since 2021

J.D. from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Merck Sharp & Dohme LLC

Lead Sponsor

Trials

4,096

Recruited

5,232,000+

Chirfi Guindo

Merck Sharp & Dohme LLC

Chief Marketing Officer since 2022

Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business

Robert M. Davis

Merck Sharp & Dohme LLC

Chief Executive Officer since 2021

JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40713644/

Safety findings from the phase 1/2 MOSAIC study of ...

Conclusion: Miransertib was safe and tolerable in participants with a confirmed diagnosis of PROS or Proteus syndrome. Future investigations are ...

2.

ojrd.biomedcentral.com

ojrd.biomedcentral.com/articles/10.1186/s13023-025-03831-z

Safety findings from the phase 1/2 MOSAIC study of ...

Conclusion. Miransertib was safe and tolerable in participants with a confirmed diagnosis of PROS or Proteus syndrome. Future investigations are ...

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S0002929719300151

Pharmacodynamic Study of Miransertib in Individuals with ...

We observed a decrease in a cerebriform connective tissue nevus and a reduction in pain in children. We conclude that 5 mg/m2/day of miransertib is an ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6996520/

Clinical report: one year of treatment of Proteus syndrome ...

After 11 mo of treatment, the patient reported improved general well-being, increased mobility of the ankle, spine, and hands, a subjective decrease in size of ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04316546

NCT04316546 | MK-7075 (Miransertib) in Proteus Syndrome

This is a Phase 2 trial investigating the efficacy of miransertib as a treatment for adult and pediatric patients with Proteus syndrome. What is the study ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04980872

NCT04980872 | A Study of the Safety and Tolerability ...

This is a study of the safety and tolerability of oral miransertib ... PROS or Proteus Syndrome Who Are Currently Being Treated With Miransertib in Other Studies.

7.

clinicaltrials.eu

clinicaltrials.eu/trial/long-term-safety-study-of-miransertib-for-patients-with-pik3ca-related-overgrowth-spectrum-or-proteus-syndrome/

Long-term Safety Study of Miransertib for Patients with ...

The study is designed to provide valuable information about the safety of miransertib for individuals with PROS or Proteus Syndrome. Participants will ...