Actively Recruiting
Feasibility and Tolerability of IMLYGIC for Treating Cutaneous Neurofibromas in Adults With NF1
Led by Johns Hopkins University · Updated on 2026-05-08
10
Participants Needed
1
Research Sites
56 weeks
Total Duration
On this page
Sponsors
J
Johns Hopkins University
Lead Sponsor
A
Amgen
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are investigating a new treatment approach for adults with neurofibromatosis Type I (NF1) who have cutaneous neurofibromas (cNFs), which are skin tumors causing disfigurement, pain, itching, or other concerns. This Phase 1 study focuses on evaluating the safety, feasibility, and possible effectiveness of IMLYGIC monotherapy in treating these skin tumors. The study is designed to provide important information for developing future treatments for NF1-related cNFs. Participants will receive IMLYGIC through injections directly into the tumors over a series of 28-day cycles. In the first cycle, the treatment is given on days 1 and 21, and in cycles 2 through 4, it is given on days 7 and 21, totaling up to eight treatments across four cycles. This intralesional administration aims to target the tumors locally. The study includes a safety run-in phase and monitors treatment tolerability and feasibility. During the study, participants will undergo medical history review, physical exams, vital sign checks, and lab tests to ensure organ function and overall health. Tumor response will be assessed by measuring lesions, taking photographs, and evaluating biopsies after cycles 2, 4, and at the end of treatment. Participants will be followed for 30 days after treatment to monitor outcomes and safety. The main goals include measuring how many participants complete all four treatment cycles and assessing whether the treatment causes serious side effects. The total study duration for each participant is approximately 16 weeks of treatment plus a follow-up period.
CONDITIONS
Brief 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 meeting NIH clinical criteria for NF1 diagnosis
- Must have at least 8 measurable cutaneous neurofibromas measuring 5 mm to 5 cm in diameter
- Lesions must not be covered by hair, have skin breakdown, be in areas prone to irritation or trauma, or previously treated by certain therapies
- Must have 6 cNFs suitable for surgical removal and be willing to undergo tumor biopsies
- Karnofsky performance status of 80% or higher
- Adequate blood, kidney, and liver function as defined by specific lab values
- Ability to comply with study procedures and provide informed consent
- Women of childbearing potential must not be pregnant or breastfeeding and agree to use effective birth control during and for 3 months after the study
- Men must agree to abstain from sperm donation and use contraception during and 90 days after treatment
- Acceptable birth control methods include sterilization, hormonal contraception, IUD or similar methods
You will not qualify if you...
- Any medical condition that makes study treatment risky or monitoring difficult
- Evidence of immunosuppression, including HIV, active hepatitis B or C, or recent high-dose steroid use
- Open herpetic skin lesions or recent/ongoing anti-herpetic treatment
- Cancer treatment within the past year (except certain skin cancers treated and healed)
- Active optic glioma or other low-grade glioma requiring treatment
- Significant eye problems from long-standing tumors are allowed
- Prolonged QT/QTc heart interval exceeding 470 msec
- Prior treatment with IMLYGIC or GM-CSF
- Current or recent treatment with MEK inhibitors
- Pregnant or breastfeeding women
- Participation in other clinical trials within 30 days prior to study
- Known allergies to study treatment components
- Severe uncontrolled medical or social conditions that could affect participation, such as uncontrolled diabetes or hypertension, severe infection, malnutrition, liver or kidney disease, heart failure, or substance dependence
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 16 weeks (4 cycles)
Participants receive intralesional administration of IMLYGIC on specified days during up to 4 cycles to treat cutaneous neurofibromas.
8 treatment visits over 16 weeks
Duration - 30 days after last treatment
Participants are assessed for post-treatment effects and clinical response after completing treatment.
1 follow-up visit
Trial Site Locations
Total: 1 location
1
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
Research Team
J
Joshua M Roberts, PhD
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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