NF1Phase 1NCT07102394
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
A Phase 1 study testing Imlygic in people with NF1 and Neurofibromatosis, run by Johns Hopkins University at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- NF1, Neurofibromatosis, Cutaneous Neurofibroma
- What is being tested?
- Imlygic
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Feb 2026to 31 Dec 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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;
- Serum albumin ≥ 2.8 g/dL;
- Calculated creatinine clearance at Screening ≥ 60 mL/min (by Cockcroft-Gault formula) OR a normal serum creatinine.
- Alanine aminotransferase (ALT) ≤ 2x upper limit of institutional norm
- Total bilirubin value of ≤1.5 x Upper Limit of Normal (ULN) (isolated bilirubin ≥1.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%)
- Participant is willing and able to comply with all aspects of the protocol
- Ability to understand and willingness to sign written informed consent document(s).
- Women of childbearing potential (WOCBP) must not be pregnant or breastfeeding during any portion of the study and must use an adequate method to prevent pregnancy during the study period and for 3 months after treatment conclusion and agree not to donate eggs (ova, oocytes) for the purpose of reproduction during the study and for a period of 3 months after last dose of study treatment. The Investigator should evaluate the effectiveness of the contraceptive method in relationship to the first dose of study treatment (see the Approved Methods of birth control listed below).
- For a woman to be determined not of childbearing potential, she must have ≥ 12 months of non-therapy-induced amenorrhea or be surgically or medically sterile.
- WOCBP must have a negative serum pregnancy test result at Screening and a negative urine pregnancy test result at the Baseline visit prior to the first dose of study treatment if the Screening Day serum test was done more than 28 days prior.
- Male participants are eligible to participate if agreed to the following during the treatment period and for at least 90 days after the last dose of study treatment:
- -- Refrain from donating sperm
- PLUS, either:
- Be abstinent from heterosexual intercourse and agree to remain abstinent until 90 days after the last study drug treatment; OR
- Must agree to use a male condom when having sexual intercourse with a WOCBP and their female partner must utilize one the approved methods of birth control below:
- Approved Methods of birth control for this study are:
- Total abstinence
- Male or female sterilization (vasectomy in males or surgical removal of ovaries or uterus in females)
- Unsterilized male study participants must use a male condom, and their female partner must use one of the methods below:
- Unsterilized female study participants must use one of the following highly effective methods listed below:
- Acceptable birth control methods which are considered highly effective if methods result in a failure rate of less than 1% per year when used consistently and correctly:
- Combined (estrogen and progestogen containing) hormonal contraceptive that stops the release of eggs from the ovary (oral, intravaginal, or transdermal)
- Progestogen-only hormonal contraception that stops the release of eggs from the ovary (oral, injectable, implantable)
- Intrauterine device (IUD)
- Intrauterine hormone-releasing system (IUS)
- Bilateral tubal occlusion or bilateral tubal ligation
Who cannot join
- 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
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- 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)
- Currently receiving therapy with a Mitogen-Activated Protein Kinase (MEK) inhibitor or treated with a MEK inhibitor in the 6 months prior to the first dose of study treatment.
- Pregnant or breastfeeding women may not take study drug.
- Current enrollment or past participation in any other clinical study (excluding observational studies) within 30 days of the first dose of study treatment.
- Known sensitivity to the study treatment, or components thereof, or drug or other allergy that, could compromise safety of the subject
- Participants are excluded if they have severe and/or uncontrolled medical disease or social situation, which could compromise participation in the study (e.g., uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration, congestive heart failure, drug or alcohol dependence, etc.).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21287
Joshua M Roberts, PhD, 609-876-9334, jrbrts004@gmail.com
About this study
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.
Read the detailed description
This is a single institutional 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 day 1 and 21 of Cycle 1 and days 7 and 21 for Cycles 2-4 for up to 4 cycles. (8 treatments). All patients must be at least 18 years of age or older with at least 8 cNFs measuring 5 mm at minimum in longest diameter, Karnofsky performance status of 80% or higher, and have adequate hematologic, renal and hepatic function. After providing written informed consent to participate in the study, patients will be evaluated for study eligibility during the screening visits.
During screening, a complete medical history will be documented, and a complete physical examination will be conducted. Measurement of vital signs and Karnofsky performance status will be obtained. Laboratory samples will be collected to assess hematologic, renal, and hepatic function. The investigators will also collect a sample to evaluate Herpes Simplex-1 (HSV1) DNA Polymerase Chain Reaction (PCR), HSV1 Immunoglobin G (IgG) and Immunoglobin M (IgM) antibodies, these will be Clinical Laboratory Improvement Amendments (CLIA) certified laboratory tests at baseline and at the end of the study. Patients should be treated until week 16 (4 months). After 16 weeks, patients will remain on study for 30-days to allow post-treatment assessment. If a participant achieves a complete response (cutaneous neurofibromas resolve and are no longer visible) for all of the target tumors before the end of the planned treatment, then the study drug will be stopped at that point, but the patient will be seen at the 30 day mark post last treatment in all cases. Efficacy assessment will be based on tumor measurement by physical measurement, photographs of superficial lesions, and representative biopsy of residual masses suspected. After cycles 2, 4 and at End of Treatment (EOT), a thorough assessment of the clinical response status will be made, particularly to determine if any evidence of biological activity of treatment can be observed.
- Conditions
- NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma, Von Recklinghausen Disease, Neurofibromas
- Being tested
- Imlygic
- Keywords
- NF1, neurofibromatosis, cutaneous neurofibroma, IMLYGIC, T-VEC, Neurofibromatosis type 1, neurofibroma
- Medical subject headings
- Neurofibromatoses, Neurofibroma, Neurofibromatosis 1
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ImlygicDrug
intralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments).
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Drug: Imlygic |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of IMLYGIC as assessed by percentage of participants receiving 4 treatments
Feasibility is defined as ≥80% of participants receiving 4 treatments
Measured: 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
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2025
- Study start1 Feb 2026
- Primary completionDec 2026 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NF1Studies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 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).
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Collaborators
- Amgen
- Responsible party
- Sponsor
- Investigators
- Carlos Romo, MD, Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07102394
- Sponsor's protocol ID
- IRB00482551
- Status last verified
- May 2026
- First submitted
- 28 Jul 2025
- Last updated
- 8 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
- ClinicalTrials.gov ID
- NCT07102394
- Acronym / sponsor ID
- IRB00482551
- Conditions
- NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma, Von Recklinghausen Disease, Neurofibromas
- Keywords
- NF1, neurofibromatosis, cutaneous neurofibroma, IMLYGIC, T-VEC, Neurofibromatosis type 1, neurofibroma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Collaborators
- Amgen
- Study start
- 1 Feb 2026 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 3 Aug 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a single institutional 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 day 1 and 21 of Cycle 1 and days 7 and 21 for Cycles 2-4 for up to 4 cycles. (8 treatments). All patients must be at least 18 years of age or older with at least 8 cNFs measuring 5 mm at minimum in longest diameter, Karnofsky performance status of 80% or higher, and have adequate hematologic, renal and hepatic function. After providing written informed consent to participate in the study, patients will be evaluated for study eligibility during the screening visits.
During screening, a complete medical history will be documented, and a complete physical examination will be conducted. Measurement of vital signs and Karnofsky performance status will be obtained. Laboratory samples will be collected to assess hematologic, renal, and hepatic function. The investigators will also collect a sample to evaluate Herpes Simplex-1 (HSV1) DNA Polymerase Chain Reaction (PCR), HSV1 Immunoglobin G (IgG) and Immunoglobin M (IgM) antibodies, these will be Clinical Laboratory Improvement Amendments (CLIA) certified laboratory tests at baseline and at the end of the study. Patients should be treated until week 16 (4 months). After 16 weeks, patients will remain on study for 30-days to allow post-treatment assessment. If a participant achieves a complete response (cutaneous neurofibromas resolve and are no longer visible) for all of the target tumors before the end of the planned treatment, then the study drug will be stopped at that point, but the patient will be seen at the 30 day mark post last treatment in all cases. Efficacy assessment will be based on tumor measurement by physical measurement, photographs of superficial lesions, and representative biopsy of residual masses suspected. After cycles 2, 4 and at End of Treatment (EOT), a thorough assessment of the clinical response status will be made, particularly to determine if any evidence of biological activity of treatment can be observed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 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).
| Arm | Type | Receives |
|---|---|---|
| IMLYGIC | Experimental | Drug: Imlygic28-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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (40 listed)
- 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
- Karnofsky performance level of ≥ 80%.
- Adequate organ and bone marrow and other organ function as defined by the following Screening laboratory values:
Key exclusion criteria (20 listed)
- 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:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imlygicintralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments). | Drug | IMLYGIC |
Primary outcome
Feasibility of IMLYGIC as assessed by percentage of participants receiving 4 treatments
Time frame: 1 year
Feasibility is defined as ≥80% of participants receiving 4 treatments
Tolerability of IMLYGIC as assessed by participants without Dose Limiting Toxicity (DLT) or Serious Adverse Event (SAE)
Time frame: 1 year
Tolerability is defined as no participants have a DLT or SAE
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Carlos Romo, MD principal investigator
Johns Hopkins University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Imlygic in people with NF1 and Neurofibromatosis, run by Johns Hopkins University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 40 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Joshua M Roberts, PhD (443-518-6462, jrobe112@jhmi.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of IMLYGIC as assessed by percentage of participants receiving 4 treatments (time frame: 1 year). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2026.