Trial Outcomes & Findings for Nintedanib in Molecularly Selected Patients With Advanced Non-Small Cell Lung Cancer (NCT NCT02299141)

Last Updated: 2025-06-24

Results Overview

* RR = Partial response plus complete response using RECIST 1.1 * Complete response (CR) = disappearance of all target lesions, non-target lesions, and normalization of tumor marker level * Partial response (PR) = at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline of sum diameters

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

After 2 cycles of therapy (approximately Day 56)

Results posted on

2025-06-24

Participant Flow

Participant milestones

Participant milestones
Measure	Nintedanib -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle
Overall Study STARTED	20
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Nintedanib in Molecularly Selected Patients With Advanced Non-Small Cell Lung Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Nintedanib n=20 Participants -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle
Age, Continuous	66 years n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 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	18 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: After 2 cycles of therapy (approximately Day 56)

Outcome measures

Outcome measures
Measure	Nintedanib n=20 Participants -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle	Responder B -Includes the mutations associated with Responder B	Responder C -Includes the mutations associated with Responder C	Progressive Disease A -Includes the mutations associated with participant Progressive Disease A	Progressive Disease B -Includes the mutations associated with participant Progressive Disease B	Progressive Disease C -Includes the mutations associated with participant Progressive Disease C	Progressive Disease D -Includes the mutations associated with participant Progressive Disease D
Response Rate (RR)	3 Participants	—	—	—	—	—	—

SECONDARY outcome

Timeframe: 12 months follow-up minimum

* PFS is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. * Progressive disease (PD) = at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study, appearance of one or more non-target lesion(s) and/or unequivocal progression of existing non-target lesions

Outcome measures

Outcome measures
Measure	Nintedanib n=20 Participants -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle	Responder B -Includes the mutations associated with Responder B	Responder C -Includes the mutations associated with Responder C	Progressive Disease A -Includes the mutations associated with participant Progressive Disease A	Progressive Disease B -Includes the mutations associated with participant Progressive Disease B	Progressive Disease C -Includes the mutations associated with participant Progressive Disease C	Progressive Disease D -Includes the mutations associated with participant Progressive Disease D
Median Progression-free Survival (PFS)	4.3 months Interval 1.8 to 7.9	—	—	—	—	—	—

SECONDARY outcome

Timeframe: At the time of response (approximately day 56)

Population: Only 3 participants had a response to treatment and are evaluable for this outcome measure

Outcome measures

Outcome measures
Measure	Nintedanib n=3 Participants -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle	Responder B -Includes the mutations associated with Responder B	Responder C -Includes the mutations associated with Responder C	Progressive Disease A -Includes the mutations associated with participant Progressive Disease A	Progressive Disease B -Includes the mutations associated with participant Progressive Disease B	Progressive Disease C -Includes the mutations associated with participant Progressive Disease C	Progressive Disease D -Includes the mutations associated with participant Progressive Disease D
Response Rate by Mutation Type TP53 mutation	3 Participants	—	—	—	—	—	—
Response Rate by Mutation Type PDGFR-A mutation	0 Participants	—	—	—	—	—	—
Response Rate by Mutation Type PDGFR-B mutation	0 Participants	—	—	—	—	—	—
Response Rate by Mutation Type FGFR1-3 mutation	1 Participants	—	—	—	—	—	—
Response Rate by Mutation Type VEGFR1-3 mutation	0 Participants	—	—	—	—	—	—
Response Rate by Mutation Type RET alteration	0 Participants	—	—	—	—	—	—

SECONDARY outcome

Timeframe: Baseline and at the time of response (approximately Day 56)

Population: Only 7 participants are evaluable for this outcome measure as they were considered extreme responders.

Correlate baseline genetic mutations with treatment response and progression of disease

Outcome measures

Outcome measures
Measure	Nintedanib n=1 Participants -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle	Responder B n=1 Participants -Includes the mutations associated with Responder B	Responder C n=1 Participants -Includes the mutations associated with Responder C	Progressive Disease A n=1 Participants -Includes the mutations associated with participant Progressive Disease A	Progressive Disease B n=1 Participants -Includes the mutations associated with participant Progressive Disease B	Progressive Disease C n=1 Participants -Includes the mutations associated with participant Progressive Disease C	Progressive Disease D n=1 Participants -Includes the mutations associated with participant Progressive Disease D
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 C242F	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 A159V	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 A27V	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 P72R	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 M160_A161del	0 mutations	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 D281E	0 mutations	0 mutations	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 R342P	0 mutations	0 mutations	0 mutations	0 mutations	1 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 D207fs	0 mutations	0 mutations	0 mutations	0 mutations	1 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) TP53 Tyr234*	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations	1 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) FGFR3 S249C	1 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) FGFR1 amplification	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations	0 mutations	1 mutations
Unique Genetic Variations Associated With Extreme Responders (Both Non-responders and Responders) PDGFRA G166E	0 mutations	0 mutations	0 mutations	1 mutations	0 mutations	0 mutations	0 mutations

SECONDARY outcome

Timeframe: At the time of progression (estimated to be 8 months)

Population: -Samples are available for 2 patients (1 patient with stable disease and 1 with partial response) but sample quantity not sufficient for genomic analysis.

Genomic analysis at time of progression after treatment with nintedanib (after response (complete response/partial response/stable disease) lasting for 6 months or longer) will provide some unique insights into mechanisms underlying acquired resistance.

Outcome measures

Outcome data not reported

Adverse Events

Nintedanib

Serious events: 12 serious events

Other events: 10 other events

Deaths: 12 deaths

Serious adverse events

Serious adverse events
Measure	Nintedanib n=20 participants at risk -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle
Cardiac disorders Atrial fibrillation	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Cardiac disorders Cardiac arrest	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Gastrointestinal disorders Esophagitis	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
General disorders Fever	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Infections and infestations Lung infection	15.0% 3/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Infections and infestations Sepsis	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Infections and infestations Skin infection	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Injury, poisoning and procedural complications Fall	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Alanine aminotransferase increased	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Aspartate aminotransferase increased	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Blood bilirubin increased	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Creatinine increased	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Musculoskeletal and connective tissue disorders Back pain	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Nervous system disorders Confusion	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Nervous system disorders Edema cerebral	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Nervous system disorders Seizure	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Nervous system disorders Stroke	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Renal and urinary disorders Renal calculi	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Respiratory, thoracic and mediastinal disorders Pleural effusion	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Respiratory, thoracic and mediastinal disorders Pneumonia	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Respiratory, thoracic and mediastinal disorders Respiratory failure	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Vascular disorders Thromboembolic event	10.0% 2/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.

Other adverse events

Other adverse events
Measure	Nintedanib n=20 participants at risk -Nintedanib will be administered orally at a dose of 200 mg twice daily during each 28 day cycle
Gastrointestinal disorders Constipation	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Gastrointestinal disorders Diarrhea	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Gastrointestinal disorders Nausea	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
General disorders Fatigue	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Injury, poisoning and procedural complications Drug induced liver injury	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Alanine aminotransferase increased	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Alkaline phosphatase increased	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Investigations Aspartate aminotransferase increased	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Metabolism and nutrition disorders Anorexia	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Metabolism and nutrition disorders Hypokalemia	15.0% 3/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Metabolism and nutrition disorders Hyponatremia	5.0% 1/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.
Vascular disorders Hypertension	15.0% 3/20 • -Adverse events are collected from start of treatment until 30 days following the last day of study treatment. Only grade 3, 4, or 5 adverse events were collected per protocol unless a grade 1 or grade 2 event met the definition of an serious adverse event.

Additional Information

Ramaswamy Govindan

Washington University School of Medicine

Phone: 314-362-5654

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place