Trial Outcomes & Findings for Identification of Gene Expression Signature for Panitumumab Sensitivity in Untreated Locally Advanced SCCHN (NCT NCT01305772)

NCT ID: NCT01305772

Last Updated: 2014-08-29

Results Overview

The aim of this outcome measure was to identify a gene expression signature that predicts response to panitumumab in untreated locally advanced squamous cell cancer of the head / neck (SCCHN). Response and progression were evaluated using the largest percentage change among the cases: 1) Pre-panitumumab PET scan activity, and/or; 2) Pre-panitumumab radiologic measurement compared to post-panitumumab measurement and/or; 3) Pre-panitumumab direct measurement of tumor / lymph node compared to post-panitumumab direct measurement of tumor / lymph node. Response and progression were evaluated in this single study using the criteria "changes in only the largest diameter (unidimensional measurement) of the tumor lesions were defined" in the same manner as in RECIST 1.1. No results are reported as only 2 of the 6 subjects had fresh tissue collected after the first dose of panitumumab. The study was amended to remove the biopsy procedure due to the potential risk for the participants.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

6 participants

Primary outcome timeframe

Baseline to 2 years

Results posted on

2014-08-29

Participant Flow

All patients had successful genomic screening and RNA extraction/quality control.

Participant milestones

Participant milestones
Measure
Radiation Therapy
Patients who underwent radiation therapy only, in conjunction with panitumumab therapy. Radiation Therapy : Radiation therapy was initiated within 8 weeks after surgery, or as soon as possible. Panitumumab : Single dose Panitumumab 9mg/kg IV for a total of 3 doses (if tolerated). Dose #1 is to be administered prior to RT for RT arm. Within the surgery arm, second and third doses of panitumumab were administered after surgery during weeks 1 \& 4 of RT. Within the RT arm, second and third doses will be administered at weeks 1 \& 4 during RT.
Surgery
Patients who underwent surgery after research PET/CT scans and subsequent radiation therapy with panitumumab administration Panitumumab : Single dose Panitumumab 9mg/kg IV for a total of 3 doses (if tolerated). Dose #1 is to be administered prior to RT for RT arm. Within the surgery arm, second and third doses of panitumumab were administered after surgery during weeks 1 \& 4 of RT. Within the RT arm, second and third doses will be administered at weeks 1 \& 4 during RT. Surgery : Second biopsy was taken from surgical resection tissue (when possible obtained pre and post panitumumab biopsies from the same site).
Overall Study
STARTED
5
1
Overall Study
Genomic Screening + Biopsy #1
5
1
Overall Study
Research PET/CT #1
5
1
Overall Study
Panitumumab Dose #1
5
1
Overall Study
Research PET/CT #2
5
1
Overall Study
Surgery/Biopsy #2
1
1
Overall Study
RT + Panitumumab Dose #2 / #3
5
1
Overall Study
COMPLETED
5
1
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Identification of Gene Expression Signature for Panitumumab Sensitivity in Untreated Locally Advanced SCCHN

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Radiation Therapy
n=5 Participants
Patients who underwent radiation therapy only, in conjunction with panitumumab therapy
Surgery
n=1 Participants
Patients who underwent surgery after research PET/CT scans and subsequent radiation therapy with panitumumab administration
Total
n=6 Participants
Total of all reporting groups
Age, Continuous
64.60 years
STANDARD_DEVIATION 6.66 • n=5 Participants
69 years
STANDARD_DEVIATION NA • n=7 Participants
65.33 years
STANDARD_DEVIATION 6.22 • n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Sex: Female, Male
Male
5 Participants
n=5 Participants
1 Participants
n=7 Participants
6 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 2 years

Population: No results will be reported for the primary outcome measure as only 2 of the 6 subjects had tissue collected after the first dose of panitumumab due to safety risk to the subject.

The aim of this outcome measure was to identify a gene expression signature that predicts response to panitumumab in untreated locally advanced squamous cell cancer of the head / neck (SCCHN). Response and progression were evaluated using the largest percentage change among the cases: 1) Pre-panitumumab PET scan activity, and/or; 2) Pre-panitumumab radiologic measurement compared to post-panitumumab measurement and/or; 3) Pre-panitumumab direct measurement of tumor / lymph node compared to post-panitumumab direct measurement of tumor / lymph node. Response and progression were evaluated in this single study using the criteria "changes in only the largest diameter (unidimensional measurement) of the tumor lesions were defined" in the same manner as in RECIST 1.1. No results are reported as only 2 of the 6 subjects had fresh tissue collected after the first dose of panitumumab. The study was amended to remove the biopsy procedure due to the potential risk for the participants.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 9 months

Nine month progression-free survival (PFS) was defined from the time from enrollment to the first date of disease progression or death as a result of any cause. Progression was defined in the same manner as in RECIST 1.1: At least a 20% increase in the sum of diameters of target lesions and an absolute increase of at least 5mm (the appearance of one or more new lesions is also considered progression). Time was censored at the date of the last follow-up visit for subjects who were still alive and have not progressed. The 9 month PFS rate is a percentage, representing the fraction of treated subjects who, after 9 months, are disease free or alive.

Outcome measures

Outcome measures
Measure
Overall Study
n=6 Participants
Patients from both the Surgery and RT arms will be combined in reporting primary and secondary outcome results, as one arm only contains 1 subject. Statistical uncertainty cannot be measured about 1 subject.
Nine (9) Month Progression Free Survival (PFS)
60 percentage of treated patients
Interval 12.57 to 88.18

SECONDARY outcome

Timeframe: 9 months

Overall survival (OS) was defined as from the time of enrollment to the date of death resulting from any cause. Time as censored at the date of the last follow-up visit for subjects who were still alive. The 9-month OS rate is a percentage, representing the fraction of treated subjects who, after 9 months, are alive.

Outcome measures

Outcome measures
Measure
Overall Study
n=6 Participants
Patients from both the Surgery and RT arms will be combined in reporting primary and secondary outcome results, as one arm only contains 1 subject. Statistical uncertainty cannot be measured about 1 subject.
Nine (9) Month Overall Survival (OS)
80 percentage of treated patients surviving
Interval 20.38 to 96.92

Adverse Events

Radiation Therapy

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

Surgery

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Radiation Therapy
n=5 participants at risk
Radiation therapy with panitumumab therapy.
Surgery
n=1 participants at risk
Surgery after research PET/CT scans and subsequent radiation therapy with panitumumab therapy.
Blood and lymphatic system disorders
Anemia
80.0%
4/5
0.00%
0/1
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify: Granulocytes
20.0%
1/5
0.00%
0/1
Blood and lymphatic system disorders
Febrile neutropenia
0.00%
0/5
100.0%
1/1
Blood and lymphatic system disorders
Lymph node pain
20.0%
1/5
0.00%
0/1
Ear and labyrinth disorders
Ear pain
20.0%
1/5
0.00%
0/1
Ear and labyrinth disorders
Hearing impaired
20.0%
1/5
0.00%
0/1
Eye disorders
Eye disorders - Other, specify: Eye Itching
60.0%
3/5
0.00%
0/1
Gastrointestinal disorders
Constipation
80.0%
4/5
100.0%
1/1
Gastrointestinal disorders
Diarrhea
20.0%
1/5
0.00%
0/1
Gastrointestinal disorders
Dry mouth
100.0%
5/5
0.00%
0/1
Gastrointestinal disorders
Dyspepsia
60.0%
3/5
0.00%
0/1
Gastrointestinal disorders
Dysphagia
60.0%
3/5
0.00%
0/1
Gastrointestinal disorders
Esophageal pain
40.0%
2/5
0.00%
0/1
Gastrointestinal disorders
Esophagitis
20.0%
1/5
0.00%
0/1
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify: Odonyphagia
100.0%
5/5
100.0%
1/1
Gastrointestinal disorders
Mucositis oral
20.0%
1/5
0.00%
0/1
Gastrointestinal disorders
Nausea
60.0%
3/5
0.00%
0/1
Gastrointestinal disorders
Rectal hemorrhage
20.0%
1/5
0.00%
0/1
Gastrointestinal disorders
Stomach pain
20.0%
1/5
0.00%
0/1
Gastrointestinal disorders
Vomiting
40.0%
2/5
100.0%
1/1
General disorders
Chills
20.0%
1/5
100.0%
1/1
General disorders
Edema face
20.0%
1/5
100.0%
1/1
General disorders
Edema limbs
20.0%
1/5
0.00%
0/1
General disorders
Fatigue
100.0%
5/5
100.0%
1/1
General disorders
Fever
40.0%
2/5
0.00%
0/1
General disorders
Pain
0.00%
0/5
100.0%
1/1
Infections and infestations
Gum infection
60.0%
3/5
0.00%
0/1
Infections and infestations
Infections and infestations - Other, specify: Face
20.0%
1/5
0.00%
0/1
Infections and infestations
Skin infection
60.0%
3/5
0.00%
0/1
Injury, poisoning and procedural complications
Dermatitis radiation
100.0%
5/5
0.00%
0/1
Injury, poisoning and procedural complications
Tracheostomy site bleeding
0.00%
0/5
100.0%
1/1
Investigations
Alanine aminotransferase increased
20.0%
1/5
0.00%
0/1
Investigations
Aspartate aminotransferase increased
20.0%
1/5
100.0%
1/1
Investigations
Blood bilirubin increased
20.0%
1/5
0.00%
0/1
Investigations
Neutrophil count decreased
40.0%
2/5
100.0%
1/1
Investigations
Platelet count decreased
40.0%
2/5
100.0%
1/1
Investigations
Weight loss
100.0%
5/5
100.0%
1/1
Investigations
White blood cell decreased
60.0%
3/5
100.0%
1/1
Metabolism and nutrition disorders
Anorexia
100.0%
5/5
0.00%
0/1
Metabolism and nutrition disorders
Dehydration
80.0%
4/5
100.0%
1/1
Metabolism and nutrition disorders
Hyperglycemia
80.0%
4/5
100.0%
1/1
Metabolism and nutrition disorders
Hyperkalemia
60.0%
3/5
0.00%
0/1
Metabolism and nutrition disorders
Hypoalbuminemia
80.0%
4/5
100.0%
1/1
Metabolism and nutrition disorders
Hypocalcemia
60.0%
3/5
100.0%
1/1
Metabolism and nutrition disorders
Hypomagnesemia
100.0%
5/5
100.0%
1/1
Metabolism and nutrition disorders
Hyponatremia
60.0%
3/5
100.0%
1/1
Metabolism and nutrition disorders
Hypophosphatemia
20.0%
1/5
0.00%
0/1
Musculoskeletal and connective tissue disorders
Arthralgia
0.00%
0/5
100.0%
1/1
Musculoskeletal and connective tissue disorders
Fibrosis deep connective tissue
20.0%
1/5
0.00%
0/1
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify: Muscle weakness - Area N/A
60.0%
3/5
0.00%
0/1
Musculoskeletal and connective tissue disorders
Myalgia
20.0%
1/5
0.00%
0/1
Musculoskeletal and connective tissue disorders
Neck pain
20.0%
1/5
0.00%
0/1
Nervous system disorders
Ataxia
20.0%
1/5
0.00%
0/1
Nervous system disorders
Depressed level of consciousness
20.0%
1/5
0.00%
0/1
Nervous system disorders
Dizziness
80.0%
4/5
0.00%
0/1
Nervous system disorders
Dysgeusia
100.0%
5/5
100.0%
1/1
Nervous system disorders
Headache
20.0%
1/5
0.00%
0/1
Nervous system disorders
Memory impairment
20.0%
1/5
0.00%
0/1
Nervous system disorders
Nervous system disorders - Other, specify: Mood alteration - Type N/A
20.0%
1/5
0.00%
0/1
Nervous system disorders
Peripheral sensory neuropathy
20.0%
1/5
100.0%
1/1
Nervous system disorders
Syncope
0.00%
0/5
100.0%
1/1
Psychiatric disorders
Agitation
0.00%
0/5
100.0%
1/1
Psychiatric disorders
Confusion
40.0%
2/5
100.0%
1/1
Psychiatric disorders
Insomnia
40.0%
2/5
0.00%
0/1
Renal and urinary disorders
Urinary incontinence
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Bronchospasm
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Cough
60.0%
3/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Dyspnea
0.00%
0/5
100.0%
1/1
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Laryngeal obstruction
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Pharyngeal hemorrhage
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Pharyngeal mucositis
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Pharyngolaryngeal pain
40.0%
2/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Pneumonitis
0.00%
0/5
100.0%
1/1
Respiratory, thoracic and mediastinal disorders
Sinus disorder
20.0%
1/5
0.00%
0/1
Respiratory, thoracic and mediastinal disorders
Voice alteration
20.0%
1/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Alopecia
20.0%
1/5
100.0%
1/1
Skin and subcutaneous tissue disorders
Pain of skin
40.0%
2/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
20.0%
1/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Photosensitivity
20.0%
1/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Pruritus
20.0%
1/5
100.0%
1/1
Skin and subcutaneous tissue disorders
Rash acneiform
80.0%
4/5
100.0%
1/1
Skin and subcutaneous tissue disorders
Rash maculo-papular
40.0%
2/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify: Erythema
0.00%
0/5
100.0%
1/1
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
20.0%
1/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Skin hypopigmentation
20.0%
1/5
0.00%
0/1
Skin and subcutaneous tissue disorders
Skin ulceration
20.0%
1/5
0.00%
0/1
Vascular disorders
Hypotension
60.0%
3/5
100.0%
1/1

Additional Information

Neal Ready,MD

Duke University Medical Center

Phone: 919-681-6932

Results disclosure agreements

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