Trial · NCT03692325

Safety and Efficacy of Nivolumab in Treating Oral Proliferative Verrucous Leukoplakia

NCT03692325, Phase 2, Completed, Dana-Farber Cancer Institute; results first posted 2024-04-22

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety and Efficacy of Nivolumab in Treating Oral Proliferative Verrucous Leukoplakia. Trials, record NCT03692325. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03692325

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety and Efficacy of Nivolumab in Treating Oral Proliferative Verrucous Leukoplakia
NCT number: NCT03692325
Status: Completed
Phases: Phase 2
Enrollment: 33 participants, actual
Lead sponsor: Dana-Farber Cancer Institute
Conditions: Leukoplakia, Oral
Interventions: Nivolumab
First posted: 2018-10-02
Last updated: 2024-10-01
Results first posted: 2024-04-22

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Best Overall Response Rate (BORR)	Participants were followed up to 164 days.	BORR on treatment is the percentage of participants who achieved CR or PR. Best overall response is the best response recorded from study registration until the first disease progression/diagnosis of invasive OSCC (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Best overall response was determined by using composite scores based on both measurement and histology, matching to the response grid as following, (1)CR, a decrease of \>80% or more; (2)PR, a decrease of 40-80%; (3)SD, neither PR or PD, (4)PD, an increase of 10% or more.
Secondary	COMD QLQ Score Change From Baseline to End of Treatment	Assessed at baseline and end of treatment. Treatment duration in days was a median (range) of 105 (21-164).	Quality of Life was evaluated using COMD QLQ (chronic oral mucosal diseases quality of life questionnaire). The range of the possible total score is 0-104, and low score is a good QoL.
Secondary	Grade 1/2 Toxicity Rate	Participants were followed up to 194 days.	The proportion of participants who experienced a maximum grade 1 or 2 adverse events regardless of treatment attribution based on the Common Toxicity Criteria for Adverse events Version 5.0 (CTCAEv5) as reported on case report forms.
Secondary	Grade 3/4 Toxicity Rate	Participants were followed up to 194 days.	The proportion of participants who experienced a maximum grade 3 or 4 adverse events regardless of treatment attribution based on the Common Toxicity Criteria for Adverse events Version 5.0 (CTCAEv5) as reported on case report forms.
Secondary	Time to the Next Surgery for a Head and Neck Malignancy	Participants were followed up to 13.3 months.	Time to Next Surgery is defined as time from the first study treatment to any head \& neck surgery or resection for biopsy-proven carcinoma in situ (CIS) or invasive oral carcinoma.
Secondary	Cancer Free Survival at 2 Years (CFS2)	Participants were followed up to 2 years.	CFS2 is the probability of participants remaining alive and cancer-free at 2 years based on Kaplan-Meier methodology. Cancer-Free Survival (CFS) is defined as the time from study registration to development of invasive oral cancer or death due to any cause. Participants alive without disease progression or recurrence (of invasive oral cancer) are censored at date of last disease evaluation.
Secondary	2-year Overall Survival (OS) Rate	Participants were followed up to 2 years.	2-year OS rate was defined as the percentage of participants alive at 2 years.
Secondary	PD-L1 Combined Positive Scores (CPS)	PD-L1 CPS assessed at baseline.	PD-L1 CPS (programmed death-1 ligand 1 combined positive score) was calculated by dividing the number of PD-L1 staining cells by the total number of viable tumor cells and then multiplying by 100. Its range of possible values was 0-100, where higher scores were better when participants received PD-L1 targeted therapy.

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Best Overall Response Rate (BORR)	Participants were followed up to 164 days.		percentage of participants	NUMBER
SECONDARY	COMD QLQ Score Change From Baseline to End of Treatment	Assessed at baseline and end of treatment. Treatment duration in days was a median (range) of 105 (21-164).	This analysis population represents the subset of participants who have completed questionnaires at both baseline and end of treatment.	Units on a scale	MEDIAN
SECONDARY	Grade 1/2 Toxicity Rate	Participants were followed up to 194 days.		proportion of participants	NUMBER
SECONDARY	Grade 3/4 Toxicity Rate	Participants were followed up to 194 days.		proportion of participants	NUMBER
SECONDARY	Time to the Next Surgery for a Head and Neck Malignancy	Participants were followed up to 13.3 months.	This analysis dataset is comprised of the participants who had next surgery.	Months	MEDIAN
SECONDARY	Cancer Free Survival at 2 Years (CFS2)	Participants were followed up to 2 years.		Probability	NUMBER
SECONDARY	2-year Overall Survival (OS) Rate	Participants were followed up to 2 years.		Percentage of participants	NUMBER
SECONDARY	PD-L1 Combined Positive Scores (CPS)	PD-L1 CPS assessed at baseline.		score on a scale	MEDIAN

Source ClinicalTrials.gov

Results by group8

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Best Overall Response Rate (BORR)	Nivolumab	36.4	95% Confidence Interval 20.4 to 54.9	percentage of participants
COMD QLQ Score Change From Baseline to End of Treatment	Nivolumab	-3	Full Range -36 to 14	Units on a scale
Grade 1/2 Toxicity Rate	Nivolumab	0.75	95% Confidence Interval 0.58 to 0.89	proportion of participants
Grade 3/4 Toxicity Rate	Nivolumab	0.21	95% Confidence Interval 0.09 to 0.39	proportion of participants
Time to the Next Surgery for a Head and Neck Malignancy	Nivolumab	6.5	Full Range 2.0 to 13.3	Months
Cancer Free Survival at 2 Years (CFS2)	Nivolumab	0.728	95% Confidence Interval 0.526 to 0.855	Probability
2-year Overall Survival (OS) Rate	Nivolumab	100	95% Confidence Interval 89.4 to 100	Percentage of participants
PD-L1 Combined Positive Scores (CPS)	Nivolumab	10	Full Range 0 to 80	score on a scale

Source ClinicalTrials.gov

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Nivolumab	33
Overall Study	COMPLETED	Nivolumab	29
Overall Study	NOT COMPLETED	Nivolumab	4

Source ClinicalTrials.gov

Adverse events120

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Blood and lymphatic system disorders - Other, specify	Blood and lymphatic system disorders	Nivolumab	1	33
other	Atrial fibrillation	Cardiac disorders	Nivolumab	2	33
other	Chest pain - cardiac	Cardiac disorders	Nivolumab	3	33
other	Sinus bradycardia	Cardiac disorders	Nivolumab	5	33
other	Ear pain	Ear and labyrinth disorders	Nivolumab	1	33
other	Endocrine disorders - Other, specify	Endocrine disorders	Nivolumab	7	33
other	Hyperthyroidism	Endocrine disorders	Nivolumab	3	33
other	Hypothyroidism	Endocrine disorders	Nivolumab	20	33
other	Blurred vision	Eye disorders	Nivolumab	1	33
other	Dry eye	Eye disorders	Nivolumab	2	33

1 to 10 of 120Source ClinicalTrials.gov

See all 120 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Nivolumab	0	33
Total, other adverse events	Nivolumab	33	33
Total, serious adverse events	Nivolumab	6	33

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37971722	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03692325

GET https://conductscience.org/api/trials/v1/NCT03692325/events?page=2

API reference For AI assistants