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M7824 in People With Recurrent Respiratory Papillomatosis

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ClinicalTrials.gov Identifier: NCT03707587
Recruitment Status : Active, not recruiting
First Posted : October 16, 2018
Results First Posted : October 14, 2020
Last Update Posted : October 19, 2021
Sponsor:
Information provided by (Responsible Party):
Scott Norberg, D.O., National Cancer Institute (NCI)

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Recurrent Respiratory Papillomatosis
Respiratory Papillomatosis
Laryngeal Papilloma, Recurrent
Human Papilloma Virus
Intervention Drug: M7824
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description 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. 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.
Period Title: Overall Study
Started 7 2
Completed 7 1
Not Completed 0 1
Reason Not Completed
Death             0             1
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition Total
Hide Arm/Group Description 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. 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 of all reporting groups
Overall Number of Baseline Participants 7 2 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 9 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
7
 100.0%
1
  50.0%
8
  88.9%
>=65 years
0
   0.0%
1
  50.0%
1
  11.1%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants 2 participants 9 participants
32.42  (13.04) 47.17  (16.32) 39.79  (14.68)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 9 participants
Female
3
  42.9%
1
  50.0%
4
  44.4%
Male
4
  57.1%
1
  50.0%
5
  55.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 9 participants
Hispanic or Latino
0
   0.0%
1
  50.0%
1
  11.1%
Not Hispanic or Latino
7
 100.0%
1
  50.0%
8
  88.9%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 2 participants 9 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
1
  14.3%
1
  50.0%
2
  22.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
5
  71.4%
1
  50.0%
6
  66.7%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
  14.3%
0
   0.0%
1
  11.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants 2 participants 9 participants
7 2 9
Median Baseline Derkay Score   [1] 
Median (Full Range)
Unit of measure:  Scores on a scale
Number Analyzed 7 participants 2 participants 9 participants
13
(2 to 24)
13.5
(12 to 15)
13
(2 to 24)
[1]
Measure Description: The Derkay staging system incorporates an objective score based on the number of sites and bulkiness of papillomas within the pharynx, larynx and trachea, ranging from 0-3 for each site. The total score (combined value of the number of sites and bulkiness of papillomas within the pharynx, larynx and trachea) can range from 0 to 75. Higher values indicate worse disease.
1.Primary Outcome
Title Proportion of Participants With a Complete Response
Hide Description 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.
Time Frame Response was assessed by flexible endoscopy with or without imaging studies before treatment and 6 and 12 weeks after starting treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 7 2
Measure Type: Number
Unit of Measure: proportion of participants
0 0
2.Secondary Outcome
Title Number of Participants With a Partial Response
Hide Description 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.
Time Frame Response was assessed by flexible endoscopy with or without imaging studies before treatment and 6 and 12 weeks after starting treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 7 2
Measure Type: Count of Participants
Unit of Measure: Participants
1
  14.3%
0
   0.0%
3.Secondary Outcome
Title Number of Participants With ≥Grade 3 Adverse Events
Hide Description 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.
Time Frame 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.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 7 2
Measure Type: Count of Participants
Unit of Measure: Participants
Grade 3 Dyspnea
1
  14.3%
0
   0.0%
Grade 3 Hypertension
1
  14.3%
1
  50.0%
Grade 3 Laryngeal obstruction
4
  57.1%
1
  50.0%
Grade 3 Lung infection
1
  14.3%
0
   0.0%
Grade 3 Pain
1
  14.3%
0
   0.0%
Grade 3 Sore throat
1
  14.3%
1
  50.0%
4.Secondary Outcome
Title Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
Hide Description 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.
Time Frame 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.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description:
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.
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 Number of Participants Analyzed 7 2
Measure Type: Count of Participants
Unit of Measure: Participants
7
 100.0%
2
 100.0%
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Hide Arm/Group Description 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. 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.
All-Cause Mortality
Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Affected / at Risk (%) Affected / at Risk (%)
Total   0/7 (0.00%)      1/2 (50.00%)    
Hide Serious Adverse Events
Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/7 (42.86%)      2/2 (100.00%)    
General disorders     
Sudden death NOS † 1  0/7 (0.00%)  0 1/2 (50.00%)  1
Infections and infestations     
Lung infection † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Laryngeal obstruction † 1  3/7 (42.86%)  3 1/2 (50.00%)  1
1
Term from vocabulary, CTCAE (5.0)
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Cohort 1 - Checkpoint Inhibitor Naive Patients Cohort 2 - Patients Refractory to Checkpoint Inhibition
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/7 (100.00%)      2/2 (100.00%)    
Eye disorders     
Dry eye † 1  0/7 (0.00%)  0 1/2 (50.00%)  1
Gastrointestinal disorders     
Diarrhea † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
General disorders     
Pain † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
Infections and infestations     
Lung infection † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
Metabolism and nutrition disorders     
Hypophosphatemia † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify † 1 [1]  1/7 (14.29%)  1 0/2 (0.00%)  0
Nervous system disorders     
Headache † 1  0/7 (0.00%)  0 1/2 (50.00%)  1
Respiratory, thoracic and mediastinal disorders     
Dyspnea † 1  3/7 (42.86%)  4 2/2 (100.00%)  2
Laryngeal obstruction † 1  1/7 (14.29%)  1 0/2 (0.00%)  0
Sore throat † 1  6/7 (85.71%)  12 1/2 (50.00%)  1
Cough † 1  0/7 (0.00%)  0 1/2 (50.00%)  1
Vascular disorders     
Hypertension † 1  4/7 (57.14%)  4 2/2 (100.00%)  3
1
Term from vocabulary, CTCAE (5.0)
†
Indicates events were collected by systematic assessment
[1]
ruptured umbilical cysts
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Scott Norberg
Organization: National Cancer Institute
Phone: 301-275-9668
EMail: scott.norberg@nih.gov
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Scott Norberg, D.O., National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT03707587    
Other Study ID Numbers: 190002
19-C-0002
First Submitted: October 12, 2018
First Posted: October 16, 2018
Results First Submitted: August 13, 2020
Results First Posted: October 14, 2020
Last Update Posted: October 19, 2021