NCT03425331

Biomarkers of Response to Ipilimumab and Nivolumab in First-line NSCLC

Terminated Phase 2 Results posted Last updated 8 January 2026

What this trial tests

Phase 2 trial testing Ipilimumab in Non-small Cell Lung Cancer in 5 participants. Terminated before completion.

Timeline

10 April 2018

Primary endpoint
18 May 2020

28 November 2021

Quick facts

Lead sponsor	Dana-Farber Cancer Institute
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	10 April 2018
Primary completion	18 May 2020
Estimated completion	28 November 2021
Sites	3 locations across United States

Drugs / interventions tested

• Ipilimumab — full drug profile →
• Nivolumab (nivolumab) — full drug profile →

Conditions studied

• Non-small Cell Lung Cancer — all drugs for Non-small Cell Lung Cancer →

Sponsor

Dana-Farber Cancer Institute

Who can join

18 and older, any sex, with Non-small Cell Lung Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Best Response Primary · Disease was evaluated radiologically at baseline and then every 6 weeks for the first 6 cycles of therapy. Median treatment duration for this study cohort is 8.05 months with range (2.76 months - 15.90 months).

Best response on treatment is based on RECISTv1.1 criteria: Complete Response (CR) is complete disappearance of all target lesions; Partial Response (PR) is at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. Both require confirmation no fewer than 4 weeks apart. Progressive Disease (PD) is at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study, and the sum must also demonstrate an absolute increase of at least 5 mm since the treatment started or the appearance of one or mor

Partial Response

Group	Value	95% CI
Nivolumab+Ipilimumab	3

Stable Disease

Group	Value	95% CI
Nivolumab+Ipilimumab	2

Median Progression-free Survival (PFS) Secondary · Disease was evaluated at baseline and then every 6 weeks for the first 6 cycles of therapy and in long-term follow-up every 3 months. Median follow-up for survival is of 15.97 months with range (4.00 months - 20.63 months).

PFS based on the Kaplan-Meier method is defined as the duration between registration and documented disease progression (PD) defined per RECIST 1.1 criteria or death, or is censored at time of last disease assessment.

Group	Value	95% CI
Nivolumab+Ipilimumab	6.90	4.01 – NA

Median Overall Survival (OS) Secondary · Median follow-up for survival is of 15.97 months with range (4.00 months - 20.63 months).

OS based on the Kaplan-Meier method is defined as the time from study entry to death or censored at date last known alive.

Group	Value	95% CI
Nivolumab+Ipilimumab	15.97	15.31 – NA

Median Duration of Response (DOR) Secondary · Disease was evaluated radiologically at baseline and then every 6 weeks for the first 6 cycles of therapy. Median treatment duration for this study cohort is 8.05 months with range (2.76 months - 15.90 months).

DOR, estimated using the Kaplan Meier method, is measured from the time measurement criteria are met for CR or PR (whichever is first recorded) per RECISTv1.1, until the first date that recurrent or progressive disease is objectively documented. Patients without PD are censored at the date of last disease assessment.

Group	Value	95% CI
Nivolumab+Ipilimumab	4.11	2.74 – 8.39

Incidence of Grade 4-5 Treatment-related Toxicity Rate Secondary · AE evaluated on day 1, 15, 29 each cycle. The median of treatment duration is 8.05 months with range (2.76 months - 15.90 months).

All grade 4-5 adverse events (AE) with treatment attribution of possibly, probably or definite based on CTCAEv4 that are not resolved in accordance with treatment guidelines were counted. Rate is the proportion of treated participants experiencing at least one of these adverse events as defined during the time of observation.

Group	Value	95% CI
Nivolumab+Ipilimumab	0.2

Adverse events — posted to ClinicalTrials.gov

Time frame: AE evaluated on day 1, 15, 29 each cycle on treatment. The median of the observation period for all-cause mortality is 15.97 months with range (4.00 months - 20.63 months). The median of observation time for AE is 8.05 months with range (2.76 months - 15.90 months).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Nivolumab+Ipilimumab

Serious: 2/5 (40%)

Deaths: 3/5

Serious adverse events (4 terms)

Reaction	System	Nivolumab+Ipilimumab
Adrenal insufficiency	Endocrine disorders	—
Fever	General disorders and administration site conditions	—
Lung infection	Infections and infestations	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—

Other adverse events (71 terms — click to expand)

Reaction	System	Nivolumab+Ipilimumab
Diarrhea	Gastrointestinal disorders	—
Fatigue	General disorders and administration site conditions	—
Non-cardiac chest pain	General disorders and administration site conditions	—
Hyponatremia	Metabolism and nutrition disorders	—
Anemia	Blood and lymphatic system disorders	—
Abdominal pain	Gastrointestinal disorders	—
Constipation	Gastrointestinal disorders	—
Vomiting	Gastrointestinal disorders	—
Edema limbs	General disorders and administration site conditions	—
Anorexia	Metabolism and nutrition disorders	—
Hypercalcemia	Metabolism and nutrition disorders	—
Hyperglycemia	Metabolism and nutrition disorders	—
Hypoalbuminemia	Metabolism and nutrition disorders	—
Back pain	Musculoskeletal and connective tissue disorders	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—
Peripheral sensory neuropathy	Nervous system disorders	—
Confusion	Psychiatric disorders	—
Depression	Psychiatric disorders	—
Insomnia	Psychiatric disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Productive cough	Respiratory, thoracic and mediastinal disorders	—
Thromboembolic event	Vascular disorders	—
Leukocytosis	Blood and lymphatic system disorders	—
Sinus tachycardia	Cardiac disorders	—
Adrenal insufficiency	Endocrine disorders	—
Hyperthyroidism	Endocrine disorders	—
Hypothyroidism	Endocrine disorders	—
Endocrine disorders - Other, specify	Endocrine disorders	—
Conjunctivitis	Eye disorders	—
Dry mouth	Gastrointestinal disorders	—
Gastroesophageal reflux disease	Gastrointestinal disorders	—
Nausea	Gastrointestinal disorders	—
Fever	General disorders and administration site conditions	—
Pain	General disorders and administration site conditions	—
Bronchial infection	Infections and infestations	—
Lung infection	Infections and infestations	—
Fall	Injury, poisoning and procedural complications	—
Fracture	Injury, poisoning and procedural complications	—
Aspartate aminotransferase increased	Investigations	—
Cholesterol high	Investigations	—

Most-reported serious reactions: Adrenal insufficiency, Fever, Lung infection, Hypoxia.

Data from ClinicalTrials.gov NCT03425331 adverse events section.

Sponsor's own description

This research study is studying two immunotherapy drugs as a possible treatment for advanced non-small cell lung cancer (NSCLC).

The drugs involved in this study are:

* Ipilimumab
* Nivolumab

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Combination checkpoint inhibitors for treatment of non-small-cell lung cancer: an update on dual anti-CTLA-4 and anti-PD-1/PD-L1 therapies.
   Puri S, Shafique M. · · 2020 · cited 29× · PMID 32158484 · DOI 10.7573/dic.2019-9-2
2. 34th Annual Meeting & Pre-Conference Programs of the Society for Immunotherapy of Cancer (SITC 2019): part 1 : National Harbor, MD, USA. 6-10 November 2019.
   · 2019 · cited 19× · PMID 31694725 · DOI 10.1186/s40425-019-0763-1
3. Nivolumab plus ipilimumab combination therapy for the first-line treatment NSCLC: evidence to date.
   Remon J, Esteller L, Taus Á. · · 2019 · cited 10× · PMID 31213908 · DOI 10.2147/cmar.s164935