Pain Relief via Mouthwash or Intravenous Infusion in Mouth Inflammation after Chemotherapy

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amsterdam UMC Stichting

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]

Decision date (initial)

2026-03-13

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: Therapy

To evaluate the non-inferiority of esketamine mouthwash compared to intravenous esketamine in reducing mucositis-related pain in patients undergoing hematopoietic stem cell transplantation.

Secondary objectives 9

1. To assess the safety and tolerability of esketamine mouthwash versus intravenous esketamine.
2. To evaluate the change in OM-related pain over the treatment period.
3. To assess oral intake and the need for enteral or parenteral nutrition.
4. To evaluate patient-reported quality of life.
5. To evaluate patient-reported side effects of esketamine treatment.
6. To compare the use of additional analgesic medication.
7. To compare hospital length of stay between the two treatment groups.
8. To evaluate treatment adherence and duration, including treatment length, breaks, and early termination.
9. To determine plasma concentrations of esketamine and norketamine.

Conditions and MedDRA coding

Oral mucositis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Are 18 years or older.
2. Receive high-dose chemotherapy in preparation for a stem cell transplant.
3. Develop painful mouth or throat pain (oral mucositis WHO ≥ 2) during admission that are not relieved by regular painkillers. To determine this, the researchers and/or doctors will examine your mouth.
4. Can give written consent.
5. Speak and understand Dutch or English sufficiently to participate and to complete questionnaires.

Exclusion criteria 12

1. Are allergic to (es)ketamine or similar medicines.
2. Have used (es)ketamine in the past 30 days.
3. Have (had) a psychosis or a severe, untreated psychiatric illness.
4. Have had problems with drug abuse or ketamine misuse.
5. Have severe liver problems or an unstable heart condition.
6. Have increased intracranial pressure.
7. Have an untreated infection in the mouth or throat.
8. Cannot safely use mouthwash (for example, due to swallowing problems or drowsiness).
9. Are pregnant or breastfeeding.
10. Are simultaneously participating in another study that may interfere with this one.
11. Do not speak enough Dutch or English to participate.
12. Or if the doctor finds participation unsafe or unsuitable for you.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Average oral and pharyngeal pain score (Numerical Rating Scale, NRS) at 1-hour post-first dose, compared between treatment arms to assess non-inferiority. Esketamine mouthwash will be declared non-inferior if the upper bound of the two-sided 95% confidence interval for the difference (mouthwash – IV) is < 1.3 NRS points.

Secondary endpoints 9

1. Number and severity of treatment-emergent adverse events (AEs) and serious adverse events (SAEs), graded using CTCAE v5.0, during the treatment period.
2. Change in average oral and pharyngeal pain score (NRS) from baseline to end of treatment.
3. Daily assessment of oral intake and use of tube feeding or parenteral nutrition, summarized at end of treatment.
4. Scores on the Daily Oral Mucositis Questionnaire (DOMQ), assessed daily and summarized at end of treatment.
5. Frequency, severity, and type of local and systemic side effects, as reported by patients using a standardized 4-point Likert-scale questionnaire, completed daily during the treatment period. Clinical staff will also record any observed side effects.
6. Total morphine-equivalent dose administered during the treatment period.
7. Total number of hospital days from randomization to discharge.
8. Total number of treatment days completed (length of treatment), number of missed doses or pauses (breaks in treatment), and documentation of early termination or discontinuation including reasons.
9. Plasma concentrations of esketamine and norketamine at predefined time points, analyzed to assess systemic exposure and metabolism for each administration route.

Investigational products

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

Test 1

Comparator 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Amsterdam UMC Stichting

Sponsor organisation

Amsterdam UMC Stichting

Address

De Boelelaan 1117

City

Amsterdam

Postcode

1081 HV

Country

Netherlands

Scientific contact point

Organisation

Amsterdam UMC Stichting

Contact name

Saskia T. M. Ammer

Public contact point

Organisation

Amsterdam UMC Stichting

Contact name

David J. Brinkman

Locations

1 EU/EEA country · 1 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 11 files

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

Application history

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