Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
Johns Hopkins University
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.
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 con…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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 1. Not covered by hair 2. There is no skin breakdown in the tumor or the skin around it 3. Not located in an area that is pron…
Interventions
- DrugImlygic
intralesional administration of IMLYGIC administered on Days 1 and 21 of each cycle for 4 cycles (8 treatments).
Location
- Johns Hopkins UniversityBaltimore, Maryland