Miransertib for Proteus Syndrome

Recruiting at 10 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)

60 Participants Needed

Trial Summary

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.

How does the drug Miransertib differ from other treatments for Proteus Syndrome?

Miransertib is unique because it specifically targets the genetic pathway involved in Proteus Syndrome, which is a rare condition with no standard treatment. Unlike other therapies, Miransertib is designed to inhibit the AKT1 enzyme, potentially addressing the root cause of the disorder. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Medical Director

Principal Investigator

Merck Sharp & Dohme LLC

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

References

1. United States pubmed.ncbi.nlm.nih.gov

Heparan sulfate regulates ADAM12 through a molecular switch mechanism. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Molecular design and structural optimization of potent peptide hydroxamate inhibitors to selectively target human ADAM metallopeptidase domain 17. [2016]

3. United States pubmed.ncbi.nlm.nih.gov

Meprin β induces activities of A disintegrin and metalloproteinases 9, 10, and 17 by specific prodomain cleavage. [2020]

4. England pubmed.ncbi.nlm.nih.gov

Harnessing the natural inhibitory domain to control TNFα Converting Enzyme (TACE) activity in vivo. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Selective inhibition of ADAM12 catalytic activity through engineering of tissue inhibitor of metalloproteinase 2 (TIMP-2). [2021]