A Multi-Cohort Phase 2 Dose-Escalation Study of MK-7075 (Miransertib) in Proteus Syndrome

Who is this study for? Patients with Proteus Syndrome
What treatments are being studied? Miransertib
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

Background: Proteus syndrome is a rare overgrowth disorder. Most people begin to have symptoms between 6 months and 2 years of age. There are very few living adults with this disease. There is also no known treatment for it. Researchers want to see if a new drug can slow down or stop overgrowth in people with Proteus syndrome.

Objective: The learn if miransertib is a safe and effective treatment for Proteus syndrome.

Eligibility: People ages 3 and older with Proteus syndrome

Design: Participants will be screened with a medical checkup. They will answer questions about their medical history and current health. They will have a physical exam with vital signs. They will have an electrocardiogram to measure their heartbeat. They will give blood and urine samples. They will repeat the screening tests during the study. Participants will take a miransertib pill once a day. They will bring their empty pill bottles with them to the NIH when they visit. If they can t swallow a pill, researchers will try to find other ways for them to take the drug. Participants will have X-rays, ultrasounds, and imaging scans. Photos may be taken of their feet and other parts of the body that have or develop signs of Proteus syndrome. Participants will have lung function tests to measure how much and how fast air moves out of their lungs. Participants will complete surveys about their levels of pain, physical functioning, and quality of life. Participants may have additional tests performed to assess their individual disease. They may have consultations with other specialists. Participation lasts about 4 years. Participants will have 20-30 visits at the NIH. ...

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Healthy Volunteers: No
View:

⁃ All participants in all Cohorts must meet the criteria below:

⁃ Signed informed consent, and when applicable, signed assent

⁃ Have a molecular diagnosis of Proteus syndrome with documented somatic AKT1 mutation from a CLIA-certified laboratory.

⁃ Have progressive and measurable disease (e.g., a measurable manifestation of Proteus syndrome with evidence or report of worsening of manifestation(s)/ in the last 12 months)

⁃ Adequate organ function as indicated by the following laboratory values:

⁃ Hematological:

⁃ Hemoglobin (Hgb): >=10.0 g/dL

⁃ Glycated hemoglobin (HbA1c): <=8% (<=64 mmol/mol)

⁃ Absolute neutrophil count (ANC): >=1.5 x 10^9/L

⁃ Platelet count >=150 x 10^9/L

⁃ Hepatic:

⁃ Total bilirubin <=2 x upper limit of normal (ULN)

⁃ Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) <=3 x ULN

⁃ Renal:

⁃ Serum creatinine depending on age:

⁃ 2-5 years male and female: <=0.50 mg/dL

⁃ Metabolic (lipids):

⁃ Cholesterol: <=400 mg/dL (<=10.34 mmol/L)

⁃ Triglyceride: <=500 mg/dL (<=5.7 mmol/L)

⁃ 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 questionnaires by the participant and/or his/her caregiver

⁃ The following specific criteria will be used to assign participants to Cohorts:

⁃ Cohort 1 (Proteus syndrome with plantar CCTN) specific criteria:

• Have at least one plantar CCTN that can accurately be measured by standardized photography. The CCTN is defined as a nevus with at least two gyri and three sulci affecting 10% - 70% of the total surface area of the foot.

• Male or female participants age greater than or equal to 3 and less than or equal to 16 years old and BSA of greater than or equal to 0.33 m^2

⁃ Cohort 2 (Proteus syndrome without plantar CCTN) specific criteria:

• Does not meet the eligibility criteria for Cohorts 1 or 3

• Male or female participants age greater than or equal to 3 years old and BSA of greater than or equal to 0.33 m^2

⁃ Cohort 3 (Proteus syndrome previously treated with miransertib) specific criteria:

• Participants previously treated with miransertib or currently receiving miransertib under Compassionate Use/Expanded Access or an existing trial (i.e., 16-HG-0014)

• Male or female participants greater than or equal to 3 years old and BSA of greater than or equal to 0.33 m^2

⁃ Note: All participants must meet Cohort-related age criteria by/on the date of the first dose, Cycle 1 Day 1

Locations
United States
Maryland
National Institutes of Health Clinical Center
Recruiting
Bethesda
Contact Information
Primary
Christopher A Ours, M.D.
chris.ours@nih.gov
(301) 443-8750
Backup
Leslie G Biesecker, M.D.
lesb@mail.nih.gov
(301) 402-2041
Time Frame
Start Date: May 20, 2022
Estimated Completion Date: March 31, 2028
Participants
Target number of participants: 45
Treatments
Experimental: MK-7075 (miransertib)
This is a single-arm study. All study participants will be taking the experimental drug, MK-7075 (miransertib).
Authors
Leslie G Biesecker
Sponsors
Leads: National Human Genome Research Institute (NHGRI)

This content was sourced from clinicaltrials.gov

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