Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

NCT07102394 | Recruiting Phase 1 FDA Drug

• 1 other identifier: IRB00482551
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

NF1; Neurofibromatosis; Cutaneous Neurofibroma; Neurofibroma; Von Recklinghausen Disease; Neurofibromas

Keywords

NF1; neurofibromatosis; cutaneous neurofibroma; IMLYGIC; T-VEC; Neurofibromatosis type 1; neurofibroma

Outcome Measures

Primary Outcomes (2)

• Feasibility of IMLYGIC as assessed by percentage of participants receiving 4 treatments

  Feasibility is defined as ≥80% of participants receiving 4 treatments

  1 year

• Tolerability of IMLYGIC as assessed by participants without Dose Limiting Toxicity (DLT) or Serious Adverse Event (SAE)

  Tolerability is defined as no participants have a DLT or SAE

  1 year

Study Arms (1)

IMLYGIC

EXPERIMENTAL

28-day treatment cycle will be defined as intralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments).

Drug: Imlygic

Interventions

Imlygic DRUG

intralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments).

IMLYGIC

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females age ≥ 18 years who meet the NIH clinical criteria for the diagnosis of NF1
• Must have ≥8 measureable cNFs. Histologic confirmation of tumor is not necessary in the presence of consistent clinical findings or confirmed genetic testing. A Measurable cNF is defined as a cutaneous lesion that meets these criteria:
• Singular lesion surrounded by uninvolved skin measuring ≥ 5 mm in the longest diameter but not larger than 5 cm
• Not covered by hair
• There is no skin breakdown in the tumor or the skin around it
• Not located in an area that is prone to irritation or trauma.
• A tumor that has not received other therapies such as electrodessication, laser treatment, or prior resection or biopsy.
• Diffusely infiltrating cutaneous neurofibromas, subcutaneous neurofibromas, and plexiform neurofibromas are NOT eligible for treatment as photographic assessments and measurements with calipers or ruler are not possible or reliable.
• Participants must have 6 cNFs that are amenable for excision.
• Participants must be willing to undergo pre- and post-treatment tumor excisions providing fresh tumor tissue; there should be no contraindication for serial biopsies.
• Karnofsky performance level of ≥ 80%.
• Adequate organ and bone marrow and other organ function as defined by the following Screening laboratory values:
• Absolute neutrophil count ≥ 1,500 cells/µL;
• Platelets ≥ 100 x 103/µL;
• Hemoglobin ≥ 9.5 g/dL;

You may not qualify if:

• Any underlying medical condition, which in the opinion of the investigator, would make administration of the study drug hazardous or make it difficult to monitor adverse effects.
• Evidence of immunosuppression for any reason:
• Known HIV infection or AIDS
• Acute or chronic active hepatitis B or hepatitis C infection
• Chronic oral or systemic steroid medication use at a dose of \>10 mg/day of prednisone or equivalent (steroids with low systemic absorption \[e.g., triamcinolone hexacetonide\] is allowed)
• Other signs or symptoms of clinical immune system suppression
• Open herpetic skin lesions
• Any anti-herpetic treatment within the last 4 weeks prior to trial participation
• Require intermittent or chronic treatment with an anti-herpetic drug (e.g., acyclovir, valacyclovir, famciclovir, any other antiviral medications), other than intermittent topical use
• One year since the last treatment for any cancer, and not requiring any ongoing secondary prevention, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.
• Patients who have had chemotherapy or radiation therapy must have recovered normal organ function as defined above; people who had excisions of basal cell or squamous cell carcinoma, must have healed and the scars must be at least 3 cm away from any cNF being monitored or treated.
• Active optic glioma or other low-grade glioma or plexiform neurofibroma requiring treatment. a. Participants not requiring treatment are eligible.
• Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor or strabismus) or long-standing orbito-temporal Plexiform Neurofibroma (PN) (such as visual loss, strabismus) are eligible
• Patients with marked baseline prolongation of QT/QTc interval (QTc interval \>470 msec) must be excluded.
• Previous treatment with IMLYGIC or treatment with Granulocyte-macrophage colony-stimulating factor (GM-CSF)

Sponsors & Collaborators

• Johns Hopkins University: lead
• Amgen: collaborator
• Neurofibromatosis Therapeutic Acceleration Program: collaborator

Study Sites (1)

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

MeSH Terms

Conditions

Neurofibromatoses; Neurofibroma; Neurofibromatosis 1

Interventions

talimogene laherparepvec

Condition Hierarchy (Ancestors)

Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Peripheral Nervous System Diseases; Neuromuscular Diseases

Study Officials

• Carlos Romo, MD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Joshua M Roberts, PhD

CONTACT

443-518-6462; jrobe112@jhmi.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a single institutional phase 1 study with a safety feasibility and tolerability study of IMLYGIC monotherapy in adults with NF1 and cNFs in need of treatment. Each 28-day treatment cycle will be defined as intralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments).

Study Record Dates

• First Submitted: July 28, 2025
• First Posted: August 3, 2025
• Study Start: February 1, 2026
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: February 17, 2026

Record last verified: 2026-02

Locations

US (1)