Trial Outcomes & Findings for Pembrolizumab for HPV-associated Recurrent Respiratory Papilloma Patients (NCT NCT02632344)

NCT ID: NCT02632344

Last Updated: 2025-08-29

Results Overview

A Simon two-stage design was used to assess best objective response rate based on endoscopic lesional burden and Response Evaluation Criteria in Solid Tumors (RECIST). For the endoscopic lesions burden score,T=the range of the score is 0 to 83. A lower score means decreased disease burden.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: Up to 2 years
• Results posted on: 2025-08-29

Participant Flow

Participant milestones

Measure	Pembrolizumab Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Overall Study STARTED	21
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Measure	Pembrolizumab Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Overall Study Adverse Event	3
Overall Study Lack of Efficacy	3
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Pembrolizumab for HPV-associated Recurrent Respiratory Papilloma Patients

Baseline characteristics by cohort

Measure	Pembrolizumab n=21 Participants Pembrolizumab 200 mg every 3 weeks.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	19 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	45 years n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 2 years

A Simon two-stage design was used to assess best objective response rate based on endoscopic lesional burden and Response Evaluation Criteria in Solid Tumors (RECIST). For the endoscopic lesions burden score,T=the range of the score is 0 to 83. A lower score means decreased disease burden.

Outcome measures

Measure	Pembrolizumab n=21 Participants Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Overall Response Rate Via Endoscopic Lesional Burden Score and/or RECIST	21 Participants

PRIMARY outcome

Timeframe: Up to 3 years after enrollment of the last the patient

To determine the safety and tolerability of pembrolizumab in subjects with RRP.

Outcome measures

Measure	Pembrolizumab n=21 Participants Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Number of Participants With Adverse Events Through CTCAE	15 Participants

SECONDARY outcome

Timeframe: Time from observed partial response to the development of progressive disease.

Population: Duration of Response was assessed among those participants who achieved a partial response (PR).

To evaluate the response duration in subjects receiving pembrolizumab.

Outcome measures

Measure	Pembrolizumab n=11 Participants Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Assess Duration of Response	13.1 months Standard Deviation 6.2

Adverse Events

Pembrolizumab

Serious events: 1 serious events

Other events: 15 other events

Deaths: 3 deaths

Serious adverse events

Measure	Pembrolizumab n=21 participants at risk Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
Endocrine disorders Hypophysitis	4.8% 1/21 • Number of events 1 • Up to 3 years after study registration.

Other adverse events

Measure	Pembrolizumab n=21 participants at risk Pembrolizumab 200 mg will be administered as a 30 minute IV infusion every 3 weeks. Treatment will be administered on Day 1 of each cycle after all procedures/assessments have been completed Pembrolizumab: 200 mg will be administered as a 30 minute IV infusion every 3 weeks on day 1 of each cycle after all procedures/assessments have been completed.
General disorders Fatigue	71.4% 15/21 • Up to 3 years after study registration.

Additional Information

Dr. Sara Pai, Principal Investigator

Massachusetts General Hospital

Phone: 617-726-5251

Email: sarapaimdphd@gmail.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place