Nadofaragene Firadenovec for Transitional Cell Carcinoma

(LUNAR Trial)

Recruiting at 5 trial locations

Overseen By Ferring Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ferring Pharmaceuticals

Must not be taking: Mitomycin, Chemo, Immunotherapy, others

Disqualifiers: High-grade UTUC, Carcinoma in situ, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

20 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Nadofaragene Firadenovec, a gene therapy, to determine its safety and tolerability for individuals with low-grade upper tract urothelial cancer, a type of bladder cancer located in the kidney area. The goal is to assess whether the cancer disappears after 3 or 6 months. The trial seeks participants with confirmed low-grade cancer in the upper urinary tract, with visible tumors between 5-15 mm in size. Participants must commit to being available for at least 18 months after starting treatment.

As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new therapy.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. However, if you are on certain treatments like systemic chemotherapy, immunotherapy, or investigational drugs, you may not be eligible to participate.

Is there any evidence suggesting that Nadofaragene Firadenovec is likely to be safe for humans?

Research has shown that Nadofaragene Firadenovec is generally well-tolerated. In various studies, most participants experienced only mild side effects, classified as grade 1 or 2, indicating they are not serious. Notably, no reports of very serious side effects (grade 4 or 5) have emerged from the treatment. This suggests that the treatment is relatively safe for humans, based on the data collected so far. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Nadofaragene Firadenovec is unique because it utilizes a gene therapy approach to treat transitional cell carcinoma of the bladder. Unlike traditional treatments like Bacillus Calmette-Guérin (BCG) therapy or chemotherapy, which can involve systemic side effects, Nadofaragene Firadenovec delivers a gene directly into the bladder cells to produce a protein that stimulates the immune system to fight cancer locally. Researchers are excited about this treatment because it offers a targeted approach that could potentially reduce side effects and improve outcomes for patients who have limited options when standard therapies fail.

What evidence suggests that Nadofaragene Firadenovec might be an effective treatment for transitional cell carcinoma?

Research has shown that nadofaragene firadenovec yields promising results for treating certain bladder cancers. In studies with patients whose bladder cancer did not respond to BCG treatment, many had no detectable cancer after 3 months. Specifically, one study found that 79% of patients with carcinoma in situ (a type of cancer where cells haven't spread) achieved a complete response at 3 months. Furthermore, 84% of these patients continued to have no detectable cancer after this period. This trial will evaluate nadofaragene firadenovec for treating transitional cell carcinoma, potentially leading to similar positive outcomes. ^{6 7 8 9 10}

Who Is on the Research Team?

Global Clinical Compliance

Principal Investigator

Ferring Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adults with low-grade upper tract urothelial cancer (LG-UTUC) who can consent, have a measurable tumor of 5-15 mm after possible downsizing, confirmed diagnosis within 2 months, an ECOG status ≤2, and a life expectancy >2 years. Women must test negative for pregnancy and use effective contraception or be postmenopausal/surgically sterile; men must also agree to use contraception.

Inclusion Criteria

My doctor believes I have more than 2 years to live.

I am a man who will use birth control or am sterile if my partner can get pregnant.

I can sign and understand the consent form.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Safety Lead-in

A safety lead-in period for the first 6 subjects to evaluate safety and tolerability

3 months

Treatment

Participants receive Nadofaragene Firadenovec instilled to the renal pelvis

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

What Are the Treatments Tested in This Trial?

Interventions

• Nadofaragene Firadenovec

Trial Overview The trial tests the safety and tolerability of Nadofaragene Firadenovec in patients with LG-UTUC. The first six participants will go through a safety lead-in period. Success is measured by the complete absence of UTUC in the renal pelvis at either 3 or 6 months after treatment.

How Is the Trial Designed?

Group I: Treatment Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Ferring Pharmaceuticals

Lead Sponsor

Trials

323

Recruited

1,242,000+

Pierre-Yves Berclaz

Ferring Pharmaceuticals

Chief Medical Officer since 2023

MD from the University of Lausanne, PhD in Molecular Biology from the Cincinnati College of Medicine

Jean-Frédéric Paulsen

Ferring Pharmaceuticals

Chief Executive Officer since 2023

Master’s degree in Finance from the London School of Economics and Political Science

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.5_suppl.716

Real-world outcomes of nadofaragene firadenovec in BCG ... Early real-world data demonstrates encouraging clinical complete response rates in patients with BCG-unresponsive NMIBC and a favorable safety profile.

2. urotoday.com urotoday.com/conference-highlights/asco-gu-2025/asco-gu-2025-bladder-cancer/158308-asco-gu-2025-real-world-outcomes-of-nadofaragene-firadenovec-in-bcg-unresponsive-nmibc.html

ASCO GU 2025: Real-World Outcomes of Nadofaragene ... This study analyzed patients treated with nadofaragene firadenovec for BCG-unresponsive non-muscle invasive bladder cancer across three Mayo Clinic sites.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10391138/

The effectiveness and value of nadofaragene firadenovec, ... In the pivotal trial of pembrolizumab among patients with CIS ± Ta/T1 disease, 39 (41%) of 96 patients achieved a CR at 3 months, which declined to 19% at 12 ...

4. ferringusa.com ferringusa.com/?press=first-real-world-outcomes-data-for-adstiladrin-nadofaragene-firadenovec-vncg-to-be-presented-at-2025-asco-genitourinary-cancers-symposium

First Real-World Outcomes Data for ADSTILADRIN ... The study is evaluating complete response rates, high-grade recurrence-free survival, cystectomy-free survival, overall survival, and adverse events.

5. onclive.com onclive.com/view/efficacy-and-safety-of-nadofaragene-firadenovec-hold-up-in-nmibc-real-world-study

Efficacy and Safety of Nadofaragene Firadenovec Hold Up ... Nadofaragene firadenovec showed a 79% complete response rate at 3 months in patients with carcinoma in situ, with 84% maintaining response at ...

6. urotoday.com urotoday.com/conference-highlights/asco-2025/asco-2025-bladder-cancer/161003-asco-2025-able-22-safety-and-efficacy-evaluation-of-nadofaragene-firadenovec-alone-or-in-combination-with-chemotherapy-or-immunotherapy-in-bcg-unresponsive-nmibc-a-randomized-open-label-phase-2-study.html

ABLE-22: Safety and Efficacy Evaluation of Nadofaragene ... Nadofaragene firadenovec was well tolerated, with no grade 4 or 5 study drug–related AEs.2. ABLE-22 (NCT06545955) is an ongoing, open-label, ...

7. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.5_suppl.TPS891

ABLE-22: Safety and efficacy evaluation of nadofaragene ... ABLE-22 (NCT06545955) is an interventional study evaluating the safety and efficacy of nadofaragene firadenovec alone or in combination with chemotherapy.

8. ferringusa.com ferringusa.com/?press=final-five-year-analysis-of-phase-3-data-with-adstiladrin-nadofaragene-firadenovec-vncg-shows-durable-bladder-preservation-and-consistent-long-term-safety-in-bcg-unresponsive-nmibc

Final Five-Year Analysis of Phase 3 Data ... The study demonstrated an 80% overall survival rate and 49% cystectomy-free survival rate at Month 60 in adult patients with high-risk, Bacillus Calmette-Gué ...

9. sciencedirect.com sciencedirect.com/org/science/article/pii/S1195947925000072

Safety of nadofaragene firadenovec-vncg: review of data ... Nadofaragene firadenovec was well tolerated, with mild grade 1 or 2 study drug–related AEs reported for most participants, and no grade 4 or 5 ...

10. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40194933/

Safety of nadofaragene firadenovec-vncg: review of data ... Conclusion: Nadofaragene firadenovec demonstrated a favorable and tolerable safety profile across its clinical study program, allowing for broad ...