Actively Recruiting
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
Led by Johns Hopkins University · Updated on 2026-05-08
10
Participants Needed
1
Research Sites
99 weeks
Total Duration
On this page
Sponsors
J
Johns Hopkins University
Lead Sponsor
A
Amgen
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is designed to establish the foundation for a new therapy for neurofibromatosis Type I (NF1)-related cutaneous neurofibromas (cNFs) by assessing the feasibility and efficacy of IMLYGIC in adults with NF1 and cNFs who desire local treatment of their cNF due to disfigurement, progression, pain, itching or other concerns. This is a single institutional Phase 1 study with a safety run-in to assess the feasibility and efficacy of IMLYGIC monotherapy in NF1 patients with cNFs. Each 28-day treatment cycle will be defined as intralesional administration of IMLYGIC administered on day 1 and 21 of Cycle 1 and days 7 and 21 for Cycles 2-4 for up to 4 cycles.
CONDITIONS
Official Title
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females aged 18 years or older who meet NIH clinical criteria for NF1 diagnosis
- Must have at least 8 measurable cutaneous neurofibromas (cNFs) 5 mm to 5 cm in size, not covered by hair or broken skin, and not located in areas prone to irritation
- Must have 6 cNFs suitable for surgical removal and be willing to undergo biopsies
- Karnofsky performance status of 80% or higher
- Adequate blood, kidney, and liver function based on specific laboratory values
- Willingness and ability to comply with study protocol
- Ability to understand and sign informed consent
- Women of childbearing potential must not be pregnant or breastfeeding, must use effective birth control during and 3 months after the study, and agree not to donate eggs
- Men must agree to avoid sperm donation and use condoms or abstain from heterosexual intercourse during treatment and 90 days after
- Women not of childbearing potential must have 12 months of amenorrhea or be surgically sterile
- Negative pregnancy tests before starting treatment
You will not qualify if you...
- Any medical condition making study drug use unsafe or monitoring difficult
- Evidence of immune system suppression including HIV, active hepatitis B or C, or use of high-dose steroids
- Open herpetic skin lesions or recent/ongoing anti-herpetic treatment
- Cancer treatment within the past year except certain skin cancers with no metastasis
- Active optic glioma or low-grade glioma requiring treatment
- Significant heart rhythm abnormalities (QTc >470 msec)
- Previous treatment with IMLYGIC or GM-CSF
- Current or recent use of MEK inhibitors within 6 months
- Pregnant or breastfeeding women
- Participation in other clinical trials within 30 days
- Known allergies to study drug or components
- Severe uncontrolled medical or social issues that affect study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
Research Team
J
Joshua M Roberts, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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