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New treatment option for NF1
Official title Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
ClinicalTrials.gov ID: NCT07102394
What this study is testing
What is Imlygic?
Imlygic is an investigational medicine, being studied as a potential treatment for nf1.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join 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...
- 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
You likely can't join if
- Any underlying medical condition, which in the opinion of the investigator, would make administration of the study drug hazardous or make it...
- 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...
- Other signs or symptoms of clinical immune system suppression
See the full eligibility criteria
Who can 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...
- 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...
- 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...
- 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...
- 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
What rules you out
- 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...
- 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...
- 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Baltimore, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Baltimore, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.