NCT02404441 is a Phase 1, PHASE2 clinical trial of PDR001 in Melanoma, sponsored by Novartis Pharmaceuticals.

Who is sponsoring NCT02404441?

NCT02404441 is sponsored by Novartis Pharmaceuticals.

What drugs are being tested in NCT02404441?

Interventions in NCT02404441: PDR001.

What condition does NCT02404441 study?

NCT02404441 studies Melanoma, Non-small Sell Lung Cancer (NSCLC), Triple Negative Breast Cancer, Anaplastic Thyroid Cancer, Other Solid Tumors.

Is NCT02404441 still recruiting?

NCT02404441 is currently completed. Last updated 3 August 2022.

Are results published for NCT02404441?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-08-03 · How we verify

NCT02404441

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Phase I/II Study of PDR001 in Patients With Advanced Malignancies

Completed Phase 1, PHASE2 Results posted Last updated 3 August 2022

What this trial tests

Phase 1, PHASE2 trial testing PDR001 in Melanoma in 319 participants. Completed in 21 July 2020.

Timeline

27 April 2015

Primary endpoint
21 July 2020

21 July 2020

Quick facts

Lead sponsor	Novartis Pharmaceuticals
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	319
Start date	27 April 2015
Primary completion	21 July 2020
Estimated completion	21 July 2020
Sites	41 locations across France, Italy, Netherlands, Taiwan, Germany, Hungary, Norway, Poland

Drugs / interventions tested

PDR001 — full drug profile →

Conditions studied

Melanoma — all drugs for Melanoma →
Non-small Sell Lung Cancer (NSCLC) — all drugs for Non-small Sell Lung Cancer (NSCLC) →
Triple Negative Breast Cancer — all drugs for Triple Negative Breast Cancer →
Anaplastic Thyroid Cancer — all drugs for Anaplastic Thyroid Cancer →

Sponsor

Novartis Pharmaceuticals — full company profile →

Who can join

18 and older, any sex, with Melanoma or Non-small Sell Lung Cancer (NSCLC). 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.

Phase l: The Exposure (AUC(0-336h)) After First Dose of Treatment at Cycle 3 (Each Cycle = 28 Days) Primary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (cycle 3)

Estimated the recommended phase 2 dose (RP2D) and/or the maximum tolerated dose (MTD) for PDR001. AUC0-336h is the AUC from time zero to 336 hour post dose of a measurable concentration sampling time.

Group	Value	95% CI
1mg/kg q2w	270	± 52.5
3mg/kg q2w	1150	± 51.1
10mg/kg q2w	3110	± 33.1
3mg/kg q4w	575	± 21.8
5mg/kg q4w	1490	± 34.2

Phase l: Incidence of Dose Limiting Toxicities (DLTs) Primary · 8 months

DLT is defined as an adverse event (AE) or abnormal laboratory value of common terminology criteria for adverse events (CTCAE) grade ≥ 3 assessed as unrelated to disease, disease progression, inter-current illness or concomitant medications, which occurs within the first cycle of treatment with PDR001 during the dose escalation part of the study for which relationship to study treatment cannot be ruled out, with some exceptions.

Group	Value	95% CI
1mg/kg q2w	0
3mg/kg q2w	0
10mg/kg q2w	0
3mg/kg q4w	0
5mg/kg q4w	0

Phase ll: Overall Response Rate (ORR) Per Response Evaluation Criteria in Solid Tumors (RECIST v1.1) Primary · 61 months

ORR is the percentage of participants with a best overall response of complete response (CR) or partial response (PR) as per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 CR = at least 2 determinations of CR at least 4 weeks apart before progression where confirmation required or 1 determination of CR prior to progression where confirmation not required. PR = at least 2 determinations of PR or better at least 4 weeks apart before progression (and not qualifying for a CR) where confirmation required or 1 determination of PR prior to progression where confirmation not required. RE

Group	Value	95% CI
NSCLC 400 mg/q4w	15.3	8.2 – 25.1
Melanoma 400 mg/q4w	27.9	18.6 – 38.8
TNBC 400 mg/q4w	0.0	0.0 – 7.2
NSCLC 300 mg/q3w	6.8	2.3 – 14.8
ATC 400 mg/q4w	19.0	9.8 – 31.8
All Phase II Patients	14.6	11.1 – 18.6

Phase I: Serum Pharmacokinetic (PK) Parameter AUCs (AUC0-336h (Cycle 1 Only), AUCinf, AUClast AUCtau) Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (cycle 1 & 3)

AUC0-336h is the AUC from time zero to 336 hour post dose of a measurable concentration sampling time. AUClast: The AUC from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1). AUCinf: The AUC from time zero to infinity (mass x time x volume-1). AUCtau: The AUC calculated to the end of a dosing interval (tau) at steady-state (amount x time x volume-1).

Cycle (C) 1: AUC0-336h (n=16, 13, 10, 6, 10)

Group	Value	95% CI
1mg/kg q2w	126	± 29.5
3mg/kg q2w	324	± 24.4
10mg/kg q2w	1270	± 20.3
3mg/kg q4w	350	± 35.0
5mg/kg q4w	638	± 35.3

C1: AUCinf (n = 1, 0,0,2,3)

Group	Value	95% CI
1mg/kg q2w	123	± 0
3mg/kg q4w	384	± 9.8
5mg/kg q4w	726	± 16.0

C1: AUClast

Group	Value	95% CI
1mg/kg q2w	125	± 29.9
3mg/kg q2w	353	± 31.4
10mg/kg q2w	1240	± 21.6
3mg/kg q4w	522	± 39.1
5mg/kg q4w	943	± 37.4

C1: AUCtau (n = 16, 13, 10, 6, 10)

Group	Value	95% CI
1mg/kg q2w	126	± 29.5
3mg/kg q2w	324	± 24.4
10mg/kg q2w	1270	± 20.3
3mg/kg q4w	524	± 39.6
5mg/kg q4w	984	± 41.9

C3: AUClast

Group	Value	95% CI
1mg/kg q2w	260	± 44.8
3mg/kg q2w	995	± 60.5
10mg/kg q2w	2520	± 58.4
3mg/kg q4w	933	± 21.3
5mg/kg q4w	2560	± 37.2

C3: AUCtau (n = 8, 4, 4, 2, 2)

Group	Value	95% CI
1mg/kg q2w	270	± 52.5
3mg/kg q2w	1150	± 51.1
10mg/kg q2w	3110	± 33.1
3mg/kg q4w	1040	± 19.1
5mg/kg q4w	2770	± 26.6

Phase I: Serum Pharmacokinetic (PK) Parameter Cmax Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (Cycle 1 & 3)

The maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration (mass x volume-1)

C1 (n = 15, 15, 10, 6, 9)

Group	Value	95% CI
1mg/kg q2w	18.2	± 26.5
3mg/kg q2w	53.8	± 23.6
10mg/kg q2w	185	± 18.3
3mg/kg q4w	53.8	± 29.4
5mg/kg q4w	106	± 34.2

C3 (n = 10, 7, 3, 3, 2)

Group	Value	95% CI
1mg/kg q2w	29.7	± 41.0
3mg/kg q2w	112	± 27.3
10mg/kg q2w	312	± 30.0
3mg/kg q4w	69.7	± 9.4
5mg/kg q4w	179	± 45.2

Phase I: Serum Pharmacokinetic (PK) Parameter Tmax Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (cycle 1 & 3)

The time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration after single dose administration (time)

C1 (n = 15, 15, 10, 6, 9)

Group	Value	95% CI
1mg/kg q2w	1.58	1.38 – 2.12
3mg/kg q2w	1.57	1.25 – 1.7
10mg/kg q2w	1.55	1.13 – 1.68
3mg/kg q4w	1.55	1.5 – 1.83
5mg/kg q4w	1.58	1.08 – 1.67

C3 (n = 10, 7, 3, 3, 2)

Group	Value	95% CI
1mg/kg q2w	1.55	1.45 – 1.75
3mg/kg q2w	1.55	0.75 – 1.58
10mg/kg q2w	1.58	1.52 – 1.62
3mg/kg q4w	1.5	1.5 – 1.57
5mg/kg q4w	1.3	0.783 – 1.82

Phase ll: Serum Pharmacokinetic (PK) Parameter AUCs (AUC336h, AUCinf, AUClast, AUCtau) Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (cycle 1 & 3)

C1: AUC0-336h (n =58, 58, 37, 54, 37)

Group	Value	95% CI
NSCLC 400 mg/q4w	681	± 39.4
Melanoma 400 mg/q4w	775	± 31.7
TNBC 400 mg/q4w	752	± 29.3
NSCLC 300 mg/q3w	535	± 38.3
ATC 400 mg/q4w	704	± 28.2

C1: AUCinf (n = 13, 8, 7, 5, 2)

Group	Value	95% CI
NSCLC 400 mg/q4w	1090	± 29.6
Melanoma 400 mg/q4w	1080	± 45.4
TNBC 400 mg/q4w	1240	± 25.2
NSCLC 300 mg/q3w	491	± 22.7
ATC 400 mg/q4w	1160	± 7.1

C1: AUClast

Group	Value	95% CI
NSCLC 400 mg/q4w	980	± 42.5
Melanoma 400 mg/q4w	1020	± 109.9
TNBC 400 mg/q4w	923	± 76.1
NSCLC 300 mg/q3w	602	± 62.2
ATC 400 mg/q4w	865	± 69.8

C1: AUCtau (n= 54, 55, 32, 48, 36)

Group	Value	95% CI
NSCLC 400 mg/q4w	1010	± 39.8
Melanoma 400 mg/q4w	1190	± 35.0
TNBC 400 mg/q4w	1130	± 34.9
NSCLC 300 mg/q3w	689	± 40.4
ATC 400 mg/q4w	1070	± 31.3

C3: AUC0-336h (n = 36, 49, 12, 40, 16)

Group	Value	95% CI
NSCLC 400 mg/q4w	1210	± 36.3
Melanoma 400 mg/q4w	1140	± 43.8
TNBC 400 mg/q4w	1360	± 45.7
NSCLC 300 mg/q3w	850	± 50.6
ATC 400 mg/q4w	1290	± 30.0

C3: AUCinf (n = 1, 1, 1, 1, 0)

Group	Value	95% CI
NSCLC 400 mg/q4w	1050	± NA
Melanoma 400 mg/q4w	1070	± NA
TNBC 400 mg/q4w	2340	± NA
NSCLC 300 mg/q3w	135	± NA

C3: AUClast (n = 37, 51, 16, 44, 19)

Group	Value	95% CI
NSCLC 400 mg/q4w	1860	± 39.9
Melanoma 400 mg/q4w	1650	± 59.7
TNBC 400 mg/q4w	1630	± 73.4
NSCLC 300 mg/q3w	984	± 78.0
ATC 400 mg/q4w	1600	± 88.0

C3: AUCtau (n= 31, 44, 7, 38, 14)

Group	Value	95% CI
NSCLC 400 mg/q4w	1940	± 35.5
Melanoma 400 mg/q4w	1790	± 53.4
TNBC 400 mg/q4w	1920	± 44.4
NSCLC 300 mg/q3w	1100	± 54.5
ATC 400 mg/q4w	2120	± 32.7

Phase ll: Serum Pharmacokinetic (PK) Parameter Cmax Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (Cycle 1 & 3)

The maximum (peak) observed plasma, blood, serum, or other body fluid drug concentration after single dose administration (mass x volume-1)

C1 (n = 52, 58, 32, 55, 35)

Group	Value	95% CI
NSCLC 400 mg/q4w	103	± 37.0
Melanoma 400 mg/q4w	111	± 26.6
TNBC 400 mg/q4w	114	± 23.6
NSCLC 300 mg/q3w	79.9	± 31.8
ATC 400 mg/q4w	100	± 27.3

C3 (n = 33, 45, 11, 39, 18)

Group	Value	95% CI
NSCLC 400 mg/q4w	151	± 32.0
Melanoma 400 mg/q4w	141	± 33.4
TNBC 400 mg/q4w	163	± 34.7
NSCLC 300 mg/q3w	103	± 36.6
ATC 400 mg/q4w	146	± 22.6

Phase ll: Serum Pharmacokinetic (PK) Parameter Tmax Secondary · Predose, 1hour (h), 24h, 48h, 72h, 168h, 240h, 336h post dose (Cycle 1 & 3)

The time to reach maximum (peak) plasma, blood, serum, or other body fluid drug concentration after single dose administration (time)

C1 (n= 52, 58, 32, 55, 35)

Group	Value	95% CI
NSCLC 400 mg/q4w	1.58	0.55 – 2.52
Melanoma 400 mg/q4w	1.58	1.07 – 2.9
TNBC 400 mg/q4w	1.58	1.18 – 2.15
NSCLC 300 mg/q3w	1.65	1.00 – 3.08
ATC 400 mg/q4w	1.55	0.5 – 2.75

C3 (n = 33, 45, 11, 39, 18)

Group	Value	95% CI
NSCLC 400 mg/q4w	1.6	0.983 – 2.08
Melanoma 400 mg/q4w	1.55	1.07 – 2.22
TNBC 400 mg/q4w	1.53	1.42 – 1.62
NSCLC 300 mg/q3w	1.58	1.33 – 2.92
ATC 400 mg/q4w	1.57	0 – 4.63

Phase I: Presence and/or Concentration of Anti-PDR001 Secondary · 42 months

Patients with ADA-negative sample at baseline

Group	Value	95% CI
1mg/kg q2w	11
3mg/kg q2w	9
10mg/kg q2w	1
All Phase I q2w	21
3mg/kg q4w	5
5mg/kg q4w	9
All Phase I q4w	14
All Phase I Patients	35

Patients with ADA-positive sample at baseline

Group	Value	95% CI
1mg/kg q2w	5
3mg/kg q2w	2
10mg/kg q2w	2
All Phase I q2w	9
3mg/kg q4w	1
5mg/kg q4w	1
All Phase I q4w	2
All Phase I Patients	11

ADA-negative

Group	Value	95% CI
1mg/kg q2w	10
3mg/kg q2w	8
10mg/kg q2w	1
All Phase I q2w	19
3mg/kg q4w	4
5mg/kg q4w	8
All Phase I q4w	12
All Phase I Patients	31

ADA-positive (i.e., ADA incidence)

Group	Value	95% CI
1mg/kg q2w	4
3mg/kg q2w	2
10mg/kg q2w	2
All Phase I q2w	8
3mg/kg q4w	1
5mg/kg q4w	1
All Phase I q4w	2
All Phase I Patients	10

Treatment-induced ADA-positive

Group	Value	95% CI
1mg/kg q2w	1
3mg/kg q2w	1
10mg/kg q2w	0
All Phase I q2w	2
3mg/kg q4w	1
5mg/kg q4w	1
All Phase I q4w	2
All Phase I Patients	4

Treatment-boosted ADA-positive

Group	Value	95% CI
1mg/kg q2w	3
3mg/kg q2w	1
10mg/kg q2w	2
All Phase I q2w	6
3mg/kg q4w	0
5mg/kg q4w	0
All Phase I q4w	0
All Phase I Patients	6

Phase II: Presence and/or Concentration of Anti-PDR001 Secondary · 42 months

Assessed PDR001 anti-drug anti-body (ADA) incidence in Phase I patients - the emergence of anti-PDR001 antibodies following one or more intravenous (i.v.) infusions of PDR001. Each cycle = 28 days; End of treatment was expected to be on average 1 year after the start of study treatment. For Treatment -induced ADA-positive, Percentage was based on subjects ADA-negative at baseline. For Treatment-boosted ADA-positive, Percentage was based on subjects ADA-positive at baseline.

Patients with ADA-negative sample at baseline

Group	Value	95% CI
NSCLC 400 mg/q4w	43
Melanoma 400 mg/q4w	48
TNBC 400 mg/q4w	29
NSCLC 300 mg/q3w	41
ATC 400 mg/q4w	29
All Phase II Patients	190

Patients with ADA-positive sample at baseline

Group	Value	95% CI
NSCLC 400 mg/q4w	9
Melanoma 400 mg/q4w	6
TNBC 400 mg/q4w	4
NSCLC 300 mg/q3w	5
ATC 400 mg/q4w	2
All Phase II Patients	26

ADA-negative

Group	Value	95% CI
NSCLC 400 mg/q4w	34
Melanoma 400 mg/q4w	46
TNBC 400 mg/q4w	23
NSCLC 300 mg/q3w	31
ATC 400 mg/q4w	24
All Phase II Patients	158

ADA-positive (i.e., ADA incidence)

Group	Value	95% CI
NSCLC 400 mg/q4w	11
Melanoma 400 mg/q4w	4
TNBC 400 mg/q4w	7
NSCLC 300 mg/q3w	12
ATC 400 mg/q4w	6
All Phase II Patients	40

Treatment-induced ADA-positive

Group	Value	95% CI
NSCLC 400 mg/q4w	9
Melanoma 400 mg/q4w	2
TNBC 400 mg/q4w	6
NSCLC 300 mg/q3w	10
ATC 400 mg/q4w	5
All Phase II Patients	32

Treatment-boosted ADA-positive

Group	Value	95% CI
NSCLC 400 mg/q4w	2
Melanoma 400 mg/q4w	2
TNBC 400 mg/q4w	1
NSCLC 300 mg/q3w	2
ATC 400 mg/q4w	1
All Phase II Patients	8

Phase l: Overall Response Rate (ORR) as Per Investigator Based on RECIST v1.1 Secondary · 27 months

ORR is the percentage of participants with a best overall response of complete response CR or partial response PR as per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 CR = at least two determinations of CR at least 4 weeks apart before progression where confirmation required or one determination of CR prior to progression where confirmation not required. PR = at least two determinations of PR or better at least 4 weeks apart before progression (and not qualifying for a CR) where confirmation required or one determination of PR prior to progression where confirmation not required.

Group	Value	95% CI
1mg/kg q2w	0.00	0.0 – 17.1
3mg/kg q2w	6.7	0.3 – 27.9
10mg/kg q2w	9.1	0.5 – 36.4
All Phase I q2w	4.8	0.9 – 14.2
3mg/kg q4w	0.0	0.0 – 39.3
5mg/kg q4w	0.0	0.0 – 25.9
All Phase I q4w	0.0	0.0 – 17.1
All Phase I Patients	3.4	0.6 – 10.5

Adverse events — posted to ClinicalTrials.gov

Time frame: On treatment deaths were collected from the start of study treatment up to 30 days after last study treatment exposure, for a maximum duration of 114.3 weeks for the Part I phase (treatment duration ranged from 2 to 110.3 weeks) and for a maximum duration of 194.9 weeks for the Phase II part (treatment duration ranged from 0.6 to 190.9 weeks).. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

1mg/kg q2w

Serious: 9/16 (56%)

Deaths: 2/16

3mg/kg q2w

Serious: 7/15 (47%)

Deaths: 2/15

10mg/kg q2w

Serious: 4/11 (36%)

Deaths: 1/11

All Phase I q2w

Serious: 20/42 (48%)

Deaths: 5/42

3mg/kg q4w

Serious: 2/6 (33%)

Deaths: 1/6

5mg/kg q4w

Serious: 2/10 (20%)

Deaths: 2/10

All Phase I q4w

Serious: 4/16 (25%)

Deaths: 3/16

All Phase I Patients

Serious: 24/58 (41%)

Deaths: 8/58

NSCLC 400mg/q4w

Serious: 27/59 (46%)

Deaths: 4/59

Melanoma 400mg/q4w

Serious: 22/61 (36%)

Deaths: 4/61

TNBC 400mg/q4w

Serious: 18/40 (45%)

Deaths: 4/40

NSCLC 300mg/q3w

Serious: 37/59 (63%)

Deaths: 12/59

ATC 400 mg/q4w

Serious: 22/42 (52%)

Deaths: 11/42

All Phase II Patients

Serious: 126/261 (48%)

Deaths: 35/261

Serious adverse events (128 terms)

Reaction	System	1mg/kg q2w	3mg/kg q2w	10mg/kg q2w	All Phase I q2w	3mg/kg q4w	5mg/kg q4w	All Phase I q4w	All Phase I Patients	NSCLC 400mg/q4w	Melanoma 400mg/q4w	TNBC 400mg/q4w	NSCLC 300mg/q3w	ATC 400 mg/q4w	All Phase II Patients
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Atrial fibrillation	Cardiac disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Cellulitis	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pneumonitis	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Cardiac arrest	Cardiac disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pericardial effusion	Cardiac disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dysphagia	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dehydration	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypercalcaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Tumour pain	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Spinal cord compression	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Embolism	Vascular disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Tachycardia	Cardiac disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Gastrointestinal haemorrhage	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—

Other adverse events (204 terms — click to expand)

Reaction	System	1mg/kg q2w	3mg/kg q2w	10mg/kg q2w	All Phase I q2w	3mg/kg q4w	5mg/kg q4w	All Phase I q4w	All Phase I Patients	NSCLC 400mg/q4w	Melanoma 400mg/q4w	TNBC 400mg/q4w	NSCLC 300mg/q3w	ATC 400 mg/q4w	All Phase II Patients
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Asthenia	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Oedema peripheral	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Weight decreased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypothyroidism	Endocrine disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Non-cardiac chest pain	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Productive cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Blood alkaline phosphatase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypoalbuminaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hyponatraemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hyperthyroidism	Endocrine disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hyperglycaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Vitiligo	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dry mouth	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—

Most-reported serious reactions: Dyspnoea, Pneumonia, Pleural effusion, Back pain, Atrial fibrillation, Pyrexia, Cellulitis, Pneumonitis.

Data from ClinicalTrials.gov NCT02404441 adverse events section.

Sponsor's own description

The purpose of this "first-in-human" study of PDR001 was to characterize the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and antitumor activity of PDR001 administered i.v. as a single agent to adult patients with solid tumors. By blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2, PDR001 inhibits the PD-1 immune checkpoint, resulting in activation of an antitumor immune response by activating effector T-cells and inhibiting regulatory T-cells.

Publications & conference data

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

PD-1 and PD-L1 Checkpoint Signaling Inhibition for Cancer Immunotherapy: Mechanism, Combinations, and Clinical Outcome.
Alsaab HO, Sau S, Alzhrani R, Tatiparti K, et al · · 2017 · cited 1206× · PMID 28878676 · DOI 10.3389/fphar.2017.00561
The Society for Immunotherapy of Cancer consensus statement on immunotherapy for the treatment of squamous cell carcinoma of the head and neck (HNSCC).
Cohen EEW, Bell RB, Bifulco CB, Burtness B, et al · · 2019 · cited 528× · PMID 31307547 · DOI 10.1186/s40425-019-0662-5
2021 American Thyroid Association Guidelines for Management of Patients with Anaplastic Thyroid Cancer.
Bible KC, Kebebew E, Brierley J, Brito JP, et al · · 2021 · cited 464× · PMID 33728999 · DOI 10.1089/thy.2020.0944
Melanoma treatment in review.
Domingues B, Lopes JM, Soares P, Pópulo H. · · 2018 · cited 432× · PMID 29922629 · DOI 10.2147/itt.s134842
PD-1 Blockade in Anaplastic Thyroid Carcinoma.
Capdevila J, Wirth LJ, Ernst T, Ponce Aix S, et al · · 2020 · cited 246× · PMID 32364844 · DOI 10.1200/jco.19.02727
Triple-negative breast cancer: treatment challenges and solutions.
Collignon J, Lousberg L, Schroeder H, Jerusalem G. · · 2016 · cited 246× · PMID 27284266 · DOI 10.2147/bctt.s69488
Advances in the systemic treatment of triple-negative breast cancer.
Lebert JM, Lester R, Powell E, Seal M, et al · · 2018 · cited 212× · PMID 29910657 · DOI 10.3747/co.25.3954
Mechanisms of immune evasion in breast cancer.
Bates JP, Derakhshandeh R, Jones L, Webb TJ. · · 2018 · cited 206× · PMID 29751789 · DOI 10.1186/s12885-018-4441-3

Verify or expand the search:

Other trials of PDR001

Trials testing the same drug.

NCT07157345 — Testing if PDR-001 Can Safely and Effectively Remove Harmful Brain Protein in Parkinson's Disease · Phase 1 · recruiting
NCT04895748 — DFF332 as a Single Agent and in Combination With Everolimus & Immuno-Oncology Agents in Advanced/Relapsed Renal Cancer & · Phase 1 · terminated
NCT04544111 — PDR001 Combination Therapy for Radioiodine-Refractory Thyroid Cancer · Phase 2 · active not recruiting
NCT04237649 — KAZ954 Alone and With PDR001, NZV930 and NIR178 in Advanced Solid Tumors · EARLY_PHASE1 · terminated
NCT04058756 — Rollover Study for Continued Safety and Tolerability in Subjects Treated With Spartalizumab Alone or in Combination With · Phase 1 · active not recruiting

Other recruiting trials for Melanoma

Currently open trials in the same condition.

NCT07524114 — Study of High-Precision Evaluation of Molecular ResiduaL Disease Through a PlatfOrm for Cancer TracKing and Interception · recruiting
NCT07224425 — A Study in People With Advanced Cancer (Solid Tumours) to Test Different Doses of BI 3810944 and to Find Out Whether it · Phase 1 · recruiting
NCT07285044 — The Cancer Connected Access and Remote Expertise Beyond Walls Program to Provide In-Home Cancer Treatment and Improve Tr · Phase 2 · recruiting
NCT07225036 — Promoting Immunotherapy Efficacy With Low-Dose Liver RT · NA · recruiting
NCT07379138 — Studying Quality of Life Inclusive of Mental Health and Cognitive Behavioral Therapy for Cancer Distress for the Improve · NA · recruiting

Other Novartis Pharmaceuticals trials

Trials by the same sponsor.

NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary · Phase 3 · not yet recruiting
NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa · Phase 4 · not yet recruiting
NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan · not yet recruiting
NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy · not yet recruiting
NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+ · Phase 1, PHASE2 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02404441 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
Last refreshed: 3 August 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02404441.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing