Trial Outcomes & Findings for M7824 in People With Recurrent Respiratory Papillomatosis (NCT NCT03707587)

Last Updated: 2022-09-29

Results Overview

Complete response for M7824 in the treatment of patients with RRP was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete Response is defined as no evidence of papillomas on physical exam and/or clinic-based flexible nasopharyngolaryngoscopy and/or tracheoscopy or by exam under anesthesia (sedation or general anesthesia) with endoscopy and biopsies, and absence of disease by imaging if lesions are assessed by imaging.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Response was assessed by flexible endoscopy with or without imaging studies before treatment and 6 and 12 weeks after starting treatment.

Results posted on

2022-09-29

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Overall Study STARTED	7	2
Overall Study COMPLETED	7	1
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Overall Study Death	0	1

Baseline Characteristics

M7824 in People With Recurrent Respiratory Papillomatosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Total n=9 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=99 Participants	1 Participants n=107 Participants	8 Participants n=206 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants
Age, Continuous	32.42 years STANDARD_DEVIATION 13.04 • n=99 Participants	47.17 years STANDARD_DEVIATION 16.32 • n=107 Participants	39.79 years STANDARD_DEVIATION 14.68 • n=206 Participants
Sex: Female, Male Female	3 Participants n=99 Participants	1 Participants n=107 Participants	4 Participants n=206 Participants
Sex: Female, Male Male	4 Participants n=99 Participants	1 Participants n=107 Participants	5 Participants n=206 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants	1 Participants n=107 Participants	1 Participants n=206 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=99 Participants	1 Participants n=107 Participants	8 Participants n=206 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Asian	1 Participants n=99 Participants	1 Participants n=107 Participants	2 Participants n=206 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Black or African American	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) White	5 Participants n=99 Participants	1 Participants n=107 Participants	6 Participants n=206 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants	0 Participants n=107 Participants	0 Participants n=206 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants	0 Participants n=107 Participants	1 Participants n=206 Participants
Region of Enrollment United States	7 participants n=99 Participants	2 participants n=107 Participants	9 participants n=206 Participants
Median Baseline Derkay Score	13 scores on a scale n=99 Participants	13.5 scores on a scale n=107 Participants	13 scores on a scale n=206 Participants

PRIMARY outcome

Timeframe: Response was assessed by flexible endoscopy with or without imaging studies before treatment and 6 and 12 weeks after starting treatment.

Outcome measures

Outcome measures
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Proportion of Participants With a Complete Response	0 proportion of participants	0 proportion of participants

SECONDARY outcome

Timeframe: Response was assessed by flexible endoscopy with or without imaging studies before treatment and 6 and 12 weeks after starting treatment.

Partial response for M7824 in the treatment of patients with RRP was assessed by the Response Evaluation Criteria in Solid Tumors (RECIST). Partial Response is defined as a decrease in Derkay anatomic score of 30 percent or greater, and a partial tumor response by imaging using RECIST 1.1 criteria.

Outcome measures

Outcome measures
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Number of Participants With a Partial Response	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days.

Adverse Events were assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Grade 3 is defined as severe or medically significant but not immediately life-threatening. Grade 4 is defined as life-threatening consequences; urgent intervention indicated.

Outcome measures

Outcome measures
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Pain	1 Participants	0 Participants
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Dyspnea	1 Participants	0 Participants
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Hypertension	1 Participants	1 Participants
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Laryngeal obstruction	4 Participants	1 Participants
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Lung infection	1 Participants	0 Participants
Number of Participants With ≥Grade 3 Adverse Events Grade 3 Sore throat	1 Participants	1 Participants

SECONDARY outcome

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 Participants Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	7 Participants	2 Participants

Adverse Events

Cohort 1 - Checkpoint Inhibitor Naive Patients

Serious events: 3 serious events

Other events: 7 other events

Deaths: 0 deaths

Cohort 2 - Patients Refractory to Checkpoint Inhibition

Serious events: 2 serious events

Other events: 2 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 participants at risk Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 participants at risk Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Respiratory, thoracic and mediastinal disorders Laryngeal obstruction	42.9% 3/7 • Number of events 3 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Infections and infestations Lung infection	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
General disorders Sudden death NOS	0.00% 0/7 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.

Other adverse events

Other adverse events
Measure	Cohort 1 - Checkpoint Inhibitor Naive Patients n=7 participants at risk Patients with Recurrent Respiratory Papillomatosis (RRP) who are checkpoint inhibitor naïve will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.	Cohort 2 - Patients Refractory to Checkpoint Inhibition n=2 participants at risk Patients with Recurrent Respiratory Papillomatosis (RRP) who are refractory to checkpoint inhibition will receive 1200 mg intravenous (IV) of M7824 on day 1 of a 14 day cycle, every other week.
Gastrointestinal disorders Diarrhea	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Respiratory, thoracic and mediastinal disorders Dyspnea	42.9% 3/7 • Number of events 4 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	100.0% 2/2 • Number of events 2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Vascular disorders Hypertension	57.1% 4/7 • Number of events 4 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	100.0% 2/2 • Number of events 3 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Metabolism and nutrition disorders Hypophosphatemia	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Respiratory, thoracic and mediastinal disorders Laryngeal obstruction	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Infections and infestations Lung infection	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
General disorders Pain	14.3% 1/7 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	0.00% 0/2 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Respiratory, thoracic and mediastinal disorders Sore throat	85.7% 6/7 • Number of events 12 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/7 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Eye disorders Dry eye	0.00% 0/7 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.
Nervous system disorders Headache	0.00% 0/7 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.	50.0% 1/2 • Number of events 1 • Per protocol, adverse events are documented from the first administration of the study therapy, Study Day 1, through 42 days after the study therapy was last administered, up to 24 weeks + 42 days. Beyond 42 days after the last administration (end of treatment), only adverse events which are serious and related to the M7824 need to be recorded until the patient is off study.

Additional Information

Dr. Scott Norberg

National Cancer Institute

Phone: 301-275-9668

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place