Imlygic for Neurofibromatosis

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Johns Hopkins University

Must not be taking: Anti-herpetics, MEK inhibitors

Disqualifiers: Immunosuppression, Active cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 10

Duration: 16 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called Imlygic, a type of oncolytic virus therapy, for individuals with neurofibromatosis Type I (NF1), specifically targeting skin tumors known as cutaneous neurofibromas (cNFs). Researchers aim to determine if Imlygic can effectively and safely reduce or improve these skin tumors, which may cause pain or cosmetic concerns. Participants should have NF1 with at least eight measurable cNFs that have not been previously treated and are not located in areas prone to irritation or trauma. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot take anti-herpetic drugs or be on certain treatments like MEK inhibitors. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that Imlygic, also known as talimogene laherparepvec, is safely used to treat conditions like melanoma, a type of skin cancer. Most recipients experience common side effects such as tiredness, chills, fever, nausea, and flu-like symptoms. These side effects occur in about 25% of patients, making them fairly common but usually manageable.

Imlygic is not recommended for pregnant individuals or those with weakened immune systems due to the involvement of a modified virus, which could pose risks for these groups. Overall, research has found that Imlygic is generally safe, with most people managing the side effects without serious problems. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for neurofibromatosis, which include surgery, radiation therapy, or medications like pain relievers and anti-seizure drugs, Imlygic is unique because it uses a modified virus to directly target and destroy tumor cells. This virus-based treatment, also known as oncolytic virus therapy, is designed to selectively infect and kill cancer cells while sparing healthy ones. Researchers are excited about Imlygic because it offers a novel approach that could potentially reduce tumor size and improve symptoms with fewer side effects compared to traditional therapies.

What evidence suggests that Imlygic might be an effective treatment for neurofibromatosis?

Research has shown that Imlygic, also known as talimogene laherparepvec, may help treat skin-related conditions. In earlier studies, about 29% of patients experienced complete remission of their condition. Imlygic uses a virus to target and destroy cancer cells, which might benefit tumors related to neurofibromatosis Type I. Although data on its use specifically for neurofibromatosis is limited, its success in other skin conditions is promising. In this trial, the treatment is administered directly into the tumors to help reduce their size and relieve symptoms such as pain or itching. ^{4 6 7 8 9}

Who Is on the Research Team?

Carlos Romo, MD

Principal Investigator

Johns Hopkins University

Are You a Good Fit for This Trial?

Adults with Neurofibromatosis Type I (NF1) who have cutaneous neurofibromas (cNFs) that cause disfigurement, pain, itching or other issues are eligible for this trial. The study is designed to test the feasibility and efficacy of a new therapy using T-VEC.

Inclusion Criteria

Must have ≥8 measureable cNFs meeting specific criteria

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)

Exclusion Criteria

Any underlying medical condition making administration of the study drug hazardous or monitoring adverse effects difficult

Evidence of immunosuppression for any reason

Marked baseline prolongation of QT/QTc interval

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Intralesional administration of IMLYGIC on Days 1 and 21 of each 28-day cycle for 4 cycles

16 weeks

8 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Imlygic

Trial Overview The trial is testing the use of Imlygic (T-VEC), an oncolytic virus therapy, as a treatment for cNFs in NF1 patients. It's a Phase 2 study where participants receive intralesional injections of T-VEC over four cycles lasting up to 28 days each.

How Is the Trial Designed?

Group I: IMLYGIC Experimental Treatment 1 Intervention

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).

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials

2,366

Recruited

15,160,000+

Neurofibromatosis Therapeutic Acceleration Program

Collaborator

Trials

3

Recruited

600+

Amgen

Industry Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8977104/

Talimogene Laherparepvec (T-VEC) and Other Oncolytic ... The primary outcome measures of the phase III study will be progression-free and overall survival with up to 24 months of treatment allowed [98]. The timing of ...

2. imlygic.com imlygic.com/efficacy-clinical-trial-results

Efficacy & Clinical Results Of the 48 patients who achieved a durable response with IMLYGIC®, 14 patients (29.1%) had a durable complete response. This means that all evidence of ...

3. jamanetwork.com jamanetwork.com/journals/jamaoncology/fullarticle/2276102

Therapeutic Advances and Treatment Options in Metastatic ... Combined talimogene laherparepvec and ipilimumab therapy demonstrated a 41% ORR in early results, and a trial evaluating pembrolizumab with ...

4. amgen.com amgen.com/newsroom/press-releases/2015/10/fda-approves-imlygic-talimogene-laherparepvec-as-first-oncolytic-viral-therapy-in-the-us

FDA Approves IMLYGIC™ (Talimogene Laherparepvec) ... In the study, the median time to response was 4.1 (range: 1.2 to 16.7) months in the IMLYGIC arm. The most common adverse drug reactions in ...

5. cinj.org cinj.org/sites/cinj/files/documents/Gaining%20momentum%20-%20New%20options%20and%20opportunities%20for%20the%20treatment%20of%20advanced%20melanoma.pdf

Gaining momentum Durable disease control can substan- tially increase life expectancy, an outcome as important as an objective response. Emerging data suggest that ...

6. imlygichcp.com imlygichcp.com/safety

Safety & Side Effects The most common adverse events reported (≥ 25%) in IMLYGIC ®-treated patients were fatigue, chills, pyrexia, nausea, influenza-like illness, and injection site ...

7. imlygic.com imlygic.com/

IMLYGIC® (talimogene laherparepvec) | Patient Information You should not get IMLYGIC® if you are pregnant or have a weakened immune system (for example, an immune deficiency, blood or bone marrow cancer, steroid use, ...

8. go.drugbank.com go.drugbank.com/drugs/DB13896

Talimogene laherparepvec: Uses, Interactions, Mechanism ... Talimogene laherparepvec is a genetically modified virus used to treat recurrent melanoma, or metastatic melanoma.

9. fda.gov fda.gov/downloads/biologicsbloodvaccines/cellulargenetherapyproducts/approvedproducts/ucm469575.pdf

Package Insert - IMLYGIC Safety and effectiveness of IMLYGIC have not been established ... IMLYGIC (talimogene laherparepvec) is a sterile suspension for intralesional injection.