A randomized, open-label, Phase III trial to assess the efficacy and safety of BupiZenge compared to lidocaine for pain associated with oral mucositis in head and neck cancer

Overview

Sponsor-declared trial summary

Key facts

Sponsor

OncoZenge AB

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Anesthesia and Analgesia [E03], Diseases [C] - Neoplasms [C04]

Decision date (initial)

2026-04-28

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the effect of BupiZenge on patient-reported oral cavity pain

Secondary objectives 9

1. To evaluate the effect of BupiZenge on patient-reported oral cavity pain at home
2. To evaluate the safety and tolerability of BupiZenge
3. To assess the consumption of systemic opioids following administration of BupiZenge
4. To evaluate the effect of BupiZenge on patient-reported mouth/throat symptoms and their functional impact
5. To evaluate the effect of BupiZenge on general health-related quality of life
6. To compare the effect of BupiZenge versus viscous lidocaine on the progression of oral mucositis severity, as measured by WHO OM grade
7. To evaluate the PK characteristics of BupiZenge in a sub-set of participants
8. To evaluate the effect of BupiZenge on patient-reported healthcare resource utilization, as assessed by HRUQ
9. To evaluate the effect of BupiZenge on work productivity and activity impairment

Conditions and MedDRA coding

Head and neck cancer

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Swedish Medical Products Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 1. Participant must provide signed written informed consent prior to trial participation and must be willing and able to comply with all requirements and restrictions of the trial.
2. 2. Male or female aged ≥ 18 on the day of consent and ≤ 80 on the first day of dosing
3. 3. Pathologically confirmed diagnosis of squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, or nasopharynx.
4. 4. About to start IMRT with curative intent with daily fractions of 2.0 Gy to 2.2 Gy to a cumulative intended dose of at least 60 Gy and a maximum of 72 Gy. Proton therapy given at equivalent biological doses is allowed.
5. 5. Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2.
6. 6. Female participants of childbearing potential (WOCBP) must agree to use at least an acceptable effective method of contraception or practice total abstinence from the time of giving informed consent until at least 24 hours after last dose of IMP.

Exclusion criteria 12

1. 1. Participation in another investigational interventional clinical trial within 3 months prior to first dosing, or for a longer period if required by local regulations, or within 5 half-lives of the investigational agent taken (whichever is longer). An exception is studies where patients are randomized to different radiotherapy settings, e.g. participation in DAHANCA 35 is allowed.
2. 2. Previous radiation therapy to the head and/or neck area.
3. 3. Pre-existing OM, active herpes simplex virus (HSV) infection, or untreated or uncontrolled oral candidiasis.
4. 4. Receiving high-dose (> 15 mg per day prednisolone), corticosteroids (for any indication).
5. 5. Known allergy or intolerance to bupivacaine, lidocaine, or any of the excipients in the products.
6. 6. Significant cardiac disease such as AV block II-III or requiring treatment with antiarrhythmic drugs in class III (e.g., amiodarone).
7. 7. Inability to eat or drink, or dependence on an enteral feeding tube (percutaneous endoscopic gastrostomy [PEG] or nasogastric tube) for any reason.
8. 8. Moderate/severe liver or kidney disease defined as: AST/ALT > 3 × upper limit of normal (ULN) or bilirubin > 1.5 × ULN (unless related to Gilbert’s syndrome), glomerular filtration rate (GFR) < 30 mL/min/1.73 m2
9. 9. Known diagnosis of epilepsy.
10. 10. Known phenylketonuria (PKU).
11. 11. Pregnancy or breastfeeding.
12. 12. Any condition or circumstance—based on the investigator’s assessment—that could increase risk to the participant, confound trial results, or interfere with compliance / participation (including inability or unwillingness to follow trial procedures, or any clinically significant physical or psychiatric condition).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Total pain reduction from baseline expressed as area under the curve (AUC) of patient-reported oral cavity pain intensity from pre-dose to 3 hours post-dose, measured by the NRS (patient-reported at site) at the last day of radiotherapy, comparing BupiZenge with lidocaine.

Secondary endpoints 16

1. Total pain reduction from baseline, expressed as AUC of patient-reported oral cavity pain intensity from pre-dose to 3 hours post-dose, measured by the NRS (patient-reported at site) at day 1 and at each of week 1 to 3 of treatment with BupiZenge/lidocaine comparing BupiZenge with lidocaine.
2. Proportion of responders defined as a 30% reduction in pain NRS AUC0-3h compared to baseline at the last day of radiotherapy and at each of weeks 1 to 3, comparing BupiZenge with lidocaine.
3. Change from pre-dose (on same day) in oral cavity pain intensity at 15 minutes post-dose (NRS; patient-reported at home), comparing BupiZenge with viscous lidocaine at the week preceding end of radiotherapy and at each of week 1 to week 3 after radiotherapy (including only data when participants on concomitant radiotherapy) (weekly means).
4. Change from pre-dose (on same day) in oral cavity pain intensity at 60 minutes post-dose (NRS; patient-reported at home), comparing BupiZenge with viscous lidocaine at the week preceding end of radiotherapy and at each of week 1 to week 3 after radiotherapy (including only data when participants on concomitant radiotherapy) (weekly means).
5. Change from pre-dose (day 1) in oral cavity pain intensity to pre-dose (NRS; patient-reported at home), comparing BupiZenge with viscous lidocaine at the week preceding end of radiotherapy and at each of week 1 to week 3 after radiotherapy (including only data when participants on concomitant radiotherapy) (weekly means)
6. Safety: Frequency and severity of AEs and SAEs as per common terminology criteria for AEs (CTCAE) v6, including changes from baseline in safety laboratory values, body weight, and vital signs
7. Tolerability: Incidence of dose modifications due to AEs/SAEs
8. Local tolerability in the oral cavity as per visual inspection by the investigator
9. Oral consumption of opioids during the treatment period with concomitant BupiZenge/lidocaine treatment and radiotherapy quantified as oral morphine milligram equivalents (MME) per day comparing BupiZenge with lidocaine.
10. Time (days) to initiation of opioid medication during concomitant radiotherapy counted from day of randomization, comparing BupiZenge with lidocaine
11. Change from baseline to last day of radiotherapy in mOMDQ Total Score, comparing BupiZenge with lidocaine
12. Change from baseline to last day of radiotherapy in RAND SF-36 Physical Component Summary (PCS) score, comparing BupiZenge with lidocaine
13. Proportion of participants who progress from WHO Grade 2 to Grade 3 or higher during the treatment period with concomitant radiotherapy, comparing BupiZenge with lidocaine
14. PK parameters in plasma including but not limited to; time vs concentration profiles, AUC0-3h, Cmax, and Tmax
15. HRUQ Total Score assessed at 30 days post-treatment
16. WPAI Total Score assessed as change from baseline to 30 days post-treatment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

OncoZenge AB

Sponsor organisation

OncoZenge AB

Address

Gustavslundsvagen 34 5 Tr, Vasterled Vasterled

City

Bromma

Postcode

167 51

Country

Sweden

Scientific contact point

Organisation

OncoZenge AB

Contact name

Marie-Louise Fjällskog

Public contact point

Organisation

OncoZenge AB

Contact name

Stian Kildal

Third parties 7

Locations

4 EU/EEA countries · 12 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 55 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications