← Back to Search

PI3K Inhibitor

Miransertib for Proteus Syndrome

Phase 2

Waitlist Available

Research Sponsored by Merck Sharp & Dohme Corp.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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)

Must not have

Has previously discontinued miransertib due to related SAEs or other intolerance of miransertib

Receives continuous high dose steroids

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to approximately 4 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying the safety of a drug called miransertib in children with a genetic condition called PIK3CA-related overgrowth spectrum (PROS) or Proteus syndrome (PS).

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects aren't listed here, the exclusion criteria suggest that past serious adverse events (SAEs) related to Miransertib could be a concern. Typically, such drugs may cause issues ranging from mild discomforts to more significant organ-specific reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

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

Select...

I am on a high dose of steroids regularly.

Select...

I am currently taking medication that targets the mTOR pathway.

Select...

I am currently on immunosuppressive therapy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to approximately 4 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to approximately 4 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 4 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of participants discontinuing study treatment due to an Adverse Event (AE)

Number of participants experiencing a Serious Adverse Event (SAE)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MiransertibExperimental Treatment1 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 48 cycles. A cycle is 28 days long.

0 / 5Eligibility criteria met