Low-grade Upper Tract Urothelial CarcinomaPhase 1/2LUNARNCT06668493
Low-grade UTUC Treated With Nadofaragene Firadenovec Administered to Renal Pelvis
A Phase 1/2 study testing Nadofaragene Firadenovec in people with Low-grade Upper Tract Urothelial Carcinoma, run by Ferring Pharmaceuticals at 9 sites in 2 countries.
Trial summary
The primary purpose of this trial is to evaluate the safety \& tolerability of Nadofaragene Firadenovec in subjects with LG-UTUC. To help with this evaluation, a safety lead-in period will be conducted for the first 6 subjects. Complete response is at 3 or 6 months defined as absence of any UTUC in the renal pelvis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Low-grade Upper Tract Urothelial Carcinoma
- What is being tested?
- Nadofaragene Firadenovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 9 sitesin 2 countries
- Study dates
- 12 Jun 2025to 30 Nov 2029
- Phase
- Phase 1/2
- 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
- Aged ≥18 years at the time of signing informed consent.
- Able to give written informed consent.
- Have biopsy-proven low-grade upper tract urothelial cancer (LG-UTUC) confirmed by a pathology report ≤2 months prior to enrolment.
- Have ≥1 measurable papillary low-grade tumour (5-15 mm in maximum diameter), evaluated visually above the ureteropelvic junction before enrolment.
- Subjects with low-grade tumour larger than 15 mm will be eligible if endoscopic downsizing of the tumour to 5-15 mm in maximum diameter has been performed before enrolment.
- Willing to be available for at least 18 months after first dosing.
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- Have life expectancy >2 years, in the opinion of the investigator.
- Have an Eastern Cooperative Oncology Group (ECOG) status of 2 or less.
- Females of reproductive potential must have a negative highly sensitive urine or serum pregnancy test upon entry into this trial and be willing to use highly effective contraception during treatment with the investigational medicinal product (IMP) and for 6 months following the last dose. Otherwise, female subjects must be postmenopausal (no menstrual period for a minimum of 12 months) or surgically sterile. Highly effective methods of contraception include: combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, and sexual abstinence.
- Male subjects with female partners of reproductive potential must be surgically sterile or willing to use a condom in addition to effective contraception in their female partner during treatment with the IMP and for 3 months following the last dose.
- Adequate laboratory values:
- haemoglobin ≥10 g/dL
- white blood cells (WBC) ≥4000/μL
- absolute neutrophil count (ANC) ≥2000/μL
- platelet count ≥100,000/μL
- international normalized ratio (INR)* below institutional upper limit of normal (ULN)
- activated partial thromboplastin time (aPTT)* below institutional ULN
- aspartate aminotransferase (AST) ≤1.5 x ULN
- alanine aminotransferase (ALT) ≤1.5 x ULN
- total bilirubin ≤1.5 x ULN
- sodium >135 mmol/L
- potassium between 3.6 and 5.0 mmol/L
- Have an estimated glomerular filtration rate (eGFR) ≥45 mL/min/1.73 m2 (for inclusion in the safety lead-in the eGFR must be ≥60 mL/min/1.73 m2 [see exclusion criteria #20]).
Who cannot join
- UTUC characterised by one or more of the following:
- High-grade cytology or high-grade histology
- Multi-focal UTUC
- Exception: Subjects with low-grade multi-focal tumours will be eligible if any ureteral tumours can be ablated before enrolment and if the total diameter of the multifocal tumours above the ureteropelvic junction is not exceeding 15 mm in diameter.
- Bilateral disease
- Exception: Subjects who have had bilateral disease are eligible (not in the safety lead-in) if one renal unit is removed or rendered disease-free by endoscopic ablation before enrolment.
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- Current or previous evidence of carcinoma in situ, of muscle invasive (muscularis propria) urothelial cancer in the urogenital tract presented at the screening visit.
- Concomitant lower tract urothelial carcinoma and/or concomitant or prior urothelial carcinoma within the prostatic urethra.
- History of high grade papillary urothelial cancer within 2 years prior to screening.
- Current or prior treatment with mitomycin gel and/or any investigational drug for the treatment of UTUC.
- Current systemic chemo- or immunotherapy for bladder cancer or any other malignancy.
- Current or prior investigational treatment for Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC) or any other investigational drug within 1 month prior to screening.
- Current or prior retroperitoneal external beam radiotherapy within 5 years of screening.
- Prior treatment with adenovirus-based drugs including use of other adenovirus vector medications, including COVID-19 vaccines, within 2 weeks before instillation.
- Suspected and/or a medical history of hypersensitivity to nadofaragene firadenovec, interferon-α2b (IFN-α2b) and/or adenovector medications.
- Urinary tract infection or bacterial cystitis (once satisfactorily treated, subjects can enter the trial).
- Clinically significant and unexplained elevated liver or renal function tests at screening.
- Women who are pregnant (highly sensitive urine or serum pregnancy test at screening) or breastfeeding.
- Any other significant disease or other clinical findings which in the opinion of the investigator would prevent trial entry.
- History of malignancy in any other organ system than the upper urinary tract within the past 5 years prior to screening. However, subjects with the following exceptions will be allowed inclusion in the trial:
- Treated basal cell carcinoma or squamous cell carcinoma of the skin.
- History of ≤pT2 upper tract urothelial carcinoma, at least 24 months after radical nephroureterectomy (RNU).
- Cervical intraepithelial carcinoma (CIN) without evidence of invasive carcinoma.
- Prostate cancer that is under active surveillance or urothelial cancer. All other genitourinary cancers are excluded.
- Inability to deliver IMP to the pyelocaliceal system.
- Previous BCG treatment during 6 months before the initiation of treatment.
- Any immunosuppressive therapy within 3 months prior to screening.
- Subjects who are immunocompromised or immunodeficient at screening.
- Subjects with solitary kidney and/or an eGFR <60 mL/min/1.73 m2 (only applicable for subjects in the safety lead-in period).
Trial locations and contacts
9 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 2 countries
United States8 sites, 8 recruiting
- Recruiting
Baylor College of Medicine (Houston)
Houston, Texas, 77030
- Recruiting
MD Anderson Cancer Center - Genitourinary (GU) Cancer Center
Houston, Texas, 77030
- Recruiting
Mayo Clinic
Jacksonville, Florida, 32224
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
- Recruiting
University of Pennsylvania - Perelman Center for Advanced Medicine - Penn Urology
Philadelphia, Pennsylvania, 19104
- Recruiting
Mayo Clinic - Rochester Minnesota
Rochester, Minnesota, 55905
- Recruiting
Providence Saint John's Cancer Institute
Santa Monica, California, 90404
- Recruiting
Mayo Clinic - Scottsdale Arizona
Scottsdale, Arizona, 85054
France1 site, 1 recruiting
- Recruiting
Hospital Tenon, Sorbone University
Paris, 75020
About this study
Official title: A Phase 1/2, Single-arm, Open-Label Trial to Evaluate the Safety and Efficacy of Nadofaragene Firadenovec Instilled to the Renal Pelvis in Adult Subjects With Low-grade Upper Tract Urothelial Carcinoma (LG-UTUC)
The primary purpose of this trial is to evaluate the safety \& tolerability of Nadofaragene Firadenovec in subjects with LG-UTUC. To help with this evaluation, a safety lead-in period will be conducted for the first 6 subjects. Complete response is at 3 or 6 months defined as absence of any UTUC in the renal pelvis.
- Being tested
- Nadofaragene Firadenovec
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nadofaragene FiradenovecDrug
Also called Adstiladrin
Repeat dose trial to investigate the safety and efficacy of nadofaragene firadenovec instilled into the renal pelvis
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Not described Receives: Drug: Nadofaragene Firadenovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
The number of treatment-emergent adverse events reported by each subject during the trial.
Measured: Up to 30 months
Complete response
defined as absence of any UTUC in the renal pelvis, i.e. negative urine cytology for high-grade urothelial carcinoma (centrally assessed), and either no suspicious lesions on ureteroscopy (investigator assessed) or a negative for-cause biopsy (centrally assessed).
Measured: Up to 6 months
Secondary outcomes (8)
Occurrence of anti-adenoviral antibodies.
Measured: Up to 30 months
Occurrence of anti-interferon-α2b (IFN-α2b) antibodies.
Measured: Up to 30 months
Shedding of adenoviral vector with IFN-α2b.
Measured: Before dose and up to 15 days after dose
Systemic exposures to IFN-α2b protein.
Measured: Before dose and up to 15 days after dose
Systemic exposures to adenoviral vector with IFN-α2b.
Measured: Before dose and up to 15 days after dose
Systemic exposures to Syn3NODA.
Measured: Before dose and up to 15 days after dose
Duration of response, defined as the time from first achieved complete response to disease recurrence, disease progression (defined as any high-grade disease) or disease-specific death, whichever occurs first.
Measured: Up to 30 months
Urinary excretion of IFN-α2b protein.
Measured: Before dose and up to 15 days after dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2024
- Study start12 Jun 2025
- Primary completion30 Nov 2029 (estimated)
- Study completion30 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Low-grade Upper Tract Urothelial CarcinomaStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ferring Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Compliance, Study director, Ferring Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06668493
- Sponsor's protocol ID
- 000425
- Other IDs
- U1111-1292-0846
- Acronym
- LUNAR
- Status last verified
- Sep 2026
- First submitted
- 30 Oct 2024
- Last updated
- 15 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Single-arm, Open-Label Trial to Evaluate the Safety and Efficacy of Nadofaragene Firadenovec Instilled to the Renal Pelvis in Adult Subjects With Low-grade Upper Tract Urothelial Carcinoma (LG-UTUC)
- ClinicalTrials.gov ID
- NCT06668493
- Acronym / sponsor ID
- LUNAR / 000425
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Ferring PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 12 Jun 2025 (actual)
- Primary completion
- 30 Nov 2029 (estimated)
- Study completion
- 30 Nov 2029 (estimated)
- First posted
- 31 Oct 2024 (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.
The primary purpose of this trial is to evaluate the safety \& tolerability of Nadofaragene Firadenovec in subjects with LG-UTUC. To help with this evaluation, a safety lead-in period will be conducted for the first 6 subjects. Complete response is at 3 or 6 months defined as absence of any UTUC in the renal pelvis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Drug: Nadofaragene Firadenovec |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (23 listed)
- Aged ≥18 years at the time of signing informed consent.
- Able to give written informed consent.
- Have biopsy-proven low-grade upper tract urothelial cancer (LG-UTUC) confirmed by a pathology report ≤2 months prior to enrolment.
- Have ≥1 measurable papillary low-grade tumour (5-15 mm in maximum diameter), evaluated visually above the ureteropelvic junction before enrolment.
- Willing to be available for at least 18 months after first dosing.
Key exclusion criteria (29 listed)
- UTUC characterised by one or more of the following:
- Current or previous evidence of carcinoma in situ, of muscle invasive (muscularis propria) urothelial cancer in the urogenital tract presented at the screening visit.
- Concomitant lower tract urothelial carcinoma and/or concomitant or prior urothelial carcinoma within the prostatic urethra.
- History of high grade papillary urothelial cancer within 2 years prior to screening.
- Current or prior treatment with mitomycin gel and/or any investigational drug for the treatment of UTUC.
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 |
|---|---|---|
| Nadofaragene FiradenovecAlso: AdstiladrinRepeat dose trial to investigate the safety and efficacy of nadofaragene firadenovec instilled into the renal pelvis | Drug | Treatment |
Primary outcome
The number of treatment-emergent adverse events reported by each subject during the trial.
Time frame: Up to 30 months
Complete response
Time frame: Up to 6 months
defined as absence of any UTUC in the renal pelvis, i.e. negative urine cytology for high-grade urothelial carcinoma (centrally assessed), and either no suspicious lesions on ureteroscopy (investigator assessed) or a negative for-cause biopsy (centrally assessed).
8 secondary outcomes
Occurrence of anti-adenoviral antibodies.
Time frame: Up to 30 months
Occurrence of anti-interferon-α2b (IFN-α2b) antibodies.
Time frame: Up to 30 months
Shedding of adenoviral vector with IFN-α2b.
Time frame: Before dose and up to 15 days after dose
Systemic exposures to IFN-α2b protein.
Time frame: Before dose and up to 15 days after dose
Systemic exposures to adenoviral vector with IFN-α2b.
Time frame: Before dose and up to 15 days after dose
Systemic exposures to Syn3NODA.
Time frame: Before dose and up to 15 days after dose
Duration of response, defined as the time from first achieved complete response to disease recurrence, disease progression (defined as any high-grade disease) or disease-specific death, whichever occurs first.
Time frame: Up to 30 months
Urinary excretion of IFN-α2b protein.
Time frame: Before dose and up to 15 days after dose
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 15 Sep 2026.
- Individual participant dataSharing planned: No
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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.
Global Clinical Compliance study director
Ferring Pharmaceuticals
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Nadofaragene Firadenovec in people with Low-grade Upper Tract Urothelial Carcinoma, run by Ferring Pharmaceuticals at 9 sites in 2 countries.
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 23 inclusion and 29 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 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, France. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Ferring Pharmaceuticals (1-888-337-7464, Disclosure@ferring.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The number of treatment-emergent adverse events reported by each subject during the trial. (time frame: Up to 30 months). 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 15 Sep 2026.