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NCT04316546 · ClinicalTrials.gov registry record · Phase 2
MK-7075 (Miransertib) in Proteus Syndrome
A Phase 2 study of Proteus Syndrome, sponsored by National Human Genome Research Institute (NHGRI).
- Recruiting
- Registry status
- Phase 2
- Development phase
- 45
- Enrollment target
- 1
- Study location
NCT04316546: Recruiting Phase 2 study of Proteus Syndrome, sponsored by National Human Genome Research Institute (NHGRI).
NCT04316546 is a Phase 2 study of Proteus Syndrome that is actively recruiting participants, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 45 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (66% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04316546, a Phase 2 study of Proteus Syndrome, is actively recruiting participants, sponsored by National Human Genome Research Institute (NHGRI).
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 45 participants
- Enrollment target
- 1
- Study location
Study 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: To 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....
Primary Outcome
Change in CCTN involvement of the plantar surface from baseline will be used to classify each subject as either a responder or non-responder (binary) in the treated population. The primary endpoint is response rate (defined as =\< 5% increase in plantar involvement from baseline over 26 cycles). This will be assessed by blinded central photography review.
Conditions Studied
Interventions
- DRUG MK-7075 (miransertib)
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2022-05-20 |
| Est. Completion | 2028-07-01 |
| Phase | Phase 2 |
What NCT04316546 shows while recruiting
NCT04316546 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 45 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (66% lower).
The record links to 1 condition, with Proteus Syndrome appearing as the primary indexed condition, and to 1 intervention - of which MK-7075 (miransertib) is the first listed.
NCT04316546 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT04316546 about?
NCT04316546 is a clinical study titled "MK-7075 (Miransertib) in Proteus Syndrome". 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 ...
What is the current status of trial NCT04316546?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 45 participants. The study started on 2022-05-20. Estimated completion is 2028-07-01.
What conditions does trial NCT04316546 study?
This clinical trial studies the following conditions: Proteus Syndrome.
What interventions are being tested in trial NCT04316546?
The interventions under investigation include: MK-7075 (miransertib) (DRUG).
Who is sponsoring clinical trial NCT04316546?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04316546 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Proteus Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04316546's enrollment target sits among peer trials
45 1296th of 2000 higher than 680 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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