Trial Outcomes & Findings for A Retrospective Observational Study of Patients With Early-stage HER2-positive Breast Cancer, Treated With Neratinib (NCT NCT05599334)

NCT ID: NCT05599334

Last Updated: 2025-10-06

Results Overview

Age will be assessed in years = neratinib initiation date - date of birth

• Recruitment status: COMPLETED
• Target enrollment: 111 participants
• Primary outcome timeframe: At baseline
• Results posted on: 2025-10-06

Participant Flow

Participant milestones

Measure	Neratinib Arm Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Belgium, Croatia, France, Italy and Spain between 01 August 2017 and 31 December 2020 (the patient identification period).
Overall Study STARTED	111
Overall Study COMPLETED	108
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

111 in the number of enrolled patients and 108 is the number of analysed patients in the FAS

Baseline characteristics by cohort

Measure	Neratinib Arm n=111 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Age, Continuous	49.2 years STANDARD_DEVIATION 8.83 • n=108 Participants • 111 in the number of enrolled patients and 108 is the number of analysed patients in the FAS
Sex: Female, Male Female	107 Participants n=108 Participants • 111 in the number of enrolled patients and 108 is the number of analysed patients
Sex: Female, Male Male	1 Participants n=108 Participants • 111 in the number of enrolled patients and 108 is the number of analysed patients
Race (NIH/OMB) American Indian or Alaska Native	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) Asian	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) Black or African American	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) White	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) More than one race	0 Participants The details of participants by ethnicity was not collected
Race (NIH/OMB) Unknown or Not Reported	0 Participants The details of participants by ethnicity was not collected
Region of Enrollment Croatia	35 participants n=111 Participants • The total number of participants of participants is 111 corresponding to the enrolled number of participants on which the disposition by country was analysed, as this was not analysed in the 108 patients corresponding to the FAS. Therefore, the distribution by region has been done out of 111 patients, and not out of 108 patients.
Region of Enrollment Spain	24 participants n=111 Participants • The total number of participants of participants is 111 corresponding to the enrolled number of participants on which the disposition by country was analysed, as this was not analysed in the 108 patients corresponding to the FAS. Therefore, the distribution by region has been done out of 111 patients, and not out of 108 patients.
Region of Enrollment Italy	21 participants n=111 Participants • The total number of participants of participants is 111 corresponding to the enrolled number of participants on which the disposition by country was analysed, as this was not analysed in the 108 patients corresponding to the FAS. Therefore, the distribution by region has been done out of 111 patients, and not out of 108 patients.
Region of Enrollment Belgium	16 participants n=111 Participants • The total number of participants of participants is 111 corresponding to the enrolled number of participants on which the disposition by country was analysed, as this was not analysed in the 108 patients corresponding to the FAS. Therefore, the distribution by region has been done out of 111 patients, and not out of 108 patients.
Region of Enrollment France	15 participants n=111 Participants • The total number of participants of participants is 111 corresponding to the enrolled number of participants on which the disposition by country was analysed, as this was not analysed in the 108 patients corresponding to the FAS. Therefore, the distribution by region has been done out of 111 patients, and not out of 108 patients.

PRIMARY outcome

Timeframe: At baseline

Population: FAS

Age will be assessed in years = neratinib initiation date - date of birth

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Description of Age	49.2 years Standard Deviation 8.83

PRIMARY outcome

Timeframe: At baseline

Population: FAS

Gender will be described in percentage of Male and Female among patients

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Description of Gender Female	107 Participants
Description of Gender male	1 Participants

PRIMARY outcome

Timeframe: At baseline

Population: FAS

BMI will be assessed in kg/m2

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Description of BMI	26.7 Kg/m² Standard Deviation 5.36

PRIMARY outcome

Timeframe: At baseline

Population: FAS

Menopausal status will be described in percentage of Premenopausal, Perimenopausal, Postmenopausal, Surgical/other reason for amenorrhea

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Menopausal Status premenopausal	40 participants
Menopausal Status perimenopausal	5 participants
Menopausal Status postmenopausal	34 participants
Menopausal Status surgical/other reason for amenorrhea	11 participants
Menopausal Status unavailable	17 participants
Menopausal Status Not applicable (Male)	1 participants

PRIMARY outcome

Timeframe: At baseline

Population: FAS

Comorbidities will be described in number and percentage of patients with at least one relevant comorbidity. Comorbidities were including but were not limited to: renal disease, liver disease, gastro-intestinal disorders, cardiovascular conditions, skin and subcutaneous disorders, diabetes (type I or II)

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Comorbidities	26 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

HER2 overexpression/amplification testing will be assessed by Immunohistochemistry (IHC), Fluorescence in situ hybridization (FISH), Chromogenic in situ hybridization (CISH)

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
HER2 Overexpression/Amplification Testing IHC 3+ (positive)	81 Participants
HER2 Overexpression/Amplification Testing IHC 2+ (equivocal)	24 Participants
HER2 Overexpression/Amplification Testing IHC no	1 Participants
HER2 Overexpression/Amplification Testing Unknown	2 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

Hormone receptor status will be described in percentage of patients with Estrogen receptor (ER) positive, Progesterone receptor (PR) positive, ER and PR negative

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Hormone Receptor Status Estrogen receptor (ER) positive	91 Participants
Hormone Receptor Status ER negative	17 Participants
Hormone Receptor Status progesterone receptor (PR) positive	74 Participants
Hormone Receptor Status PR negative	34 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

Primary tumor location will be described in percentage of patients with Left Breast cancer, Right Breast cancer

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Primary Tumor Location Left breast	47 Participants
Primary Tumor Location Right breast	62 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

Primary tumor histology will be described in percentage of patients with Infiltrating ductal carcinoma, Infiltrating lobular carcinoma, Invasive carcinoma, Tubular, Other

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Primary Tumor Histology Infiltrating ductal carcinoma	56 Participants
Primary Tumor Histology Infiltrating lobular carcinoma	3 Participants
Primary Tumor Histology Invasive carcinoma	43 Participants
Primary Tumor Histology Other	6 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

Histological grade will be described in percentage of patients with G1 Well differentiated, G2 Moderately differentiated, G3 Poorly differentiated, G4 Undifferentiated

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Histological Grade G1 Well differentiated	5 Participants
Histological Grade G2 Moderately differentiated	34 Participants
Histological Grade G3 Poorly differentiated	54 Participants
Histological Grade Unavailable	15 Participants

PRIMARY outcome

Timeframe: At initial diagnosis

Population: FAS

Higher is the stage worse is the cancer.

Stage is defined as:

1A: tumor (T) up to 2 cm + not spread outside the breast; no lymph nodes (LN).1B: Small clusters of cancer cells (CC) (0.2-2 mm) are found in LN, or T in the breast is < 2 cm and small clusters of CC are found in LN. 2A: No T in the breast, but cancer is in 1-3 axillary LN or in LN near the breastbone or T is 2 cm or smaller + has spread to 1-3 axillary LN or LN near the breastbone, or T is > 2 cm and < 5 cm and not spread to LN.2B: T is > 2 cm and < 5 cm and has spread to 1-3 axillary LN or LN near the breastbone, or T is > 5 cm and not spread to the LN. 3A: no T in the breast or the T is any size; cancer is found in 4-9 axillary LN or LN near the breastbone, or T is larger than 5 cm and small clusters of CC found in LN. 3B: T has spread to chest wall or breast skin + may have spread to up to 9 axillary LN. 3C: Cancer has spread to ≥10 axillary LN, LN near the collarbone, or LN near breastbone

Outcome measures

Measure	Neratinib Arm n=108 Participants Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Pathologic Stage (AJCC Classification) of Breast Cancer I	21 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer IIA	17 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer IIB	29 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer IIIA	25 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer IIIB	5 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer IIIC	6 Participants
Pathologic Stage (AJCC Classification) of Breast Cancer Missing	5 Participants

Adverse Events

Neratinib Arm

Serious events: 12 serious events

Other events: 10 other events

Deaths: 1 deaths

Serious adverse events

Measure	Neratinib Arm n=108 participants at risk Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Nervous system disorders Subarachnoid haemorrhage	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Infections and infestations Infected seroma	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Diarrhea	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Death	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Pyrexia	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Chest pain	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Investigations Hepatic enzymze increased	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Metabolism and nutrition disorders Hypercalcaemia	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Oedema peripheral	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Cardiac disorders Palpitations	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Metabolism and nutrition disorders Dehydration	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Metabolism and nutrition disorders Hypokalaemia	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Metabolism and nutrition disorders Hyperkalaemia	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Cardiac disorders Atrial fibrillation	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date

Other adverse events

Measure	Neratinib Arm n=108 participants at risk Eligible patients were selected among those who had received at least one dose of neratinib in the context of the EAP in Europe between 01 August 2017 and 31 December 2020 (the patient identification period) and residing in one of the five target following countries: Belgium, Croatia, France, Italy and Spain, which were part of the EAP.
Gastrointestinal disorders abdominal pain	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Abdominal pain upper	2.8% 3/108 • Number of events 3 • From the baseline to the study entry date
Musculoskeletal and connective tissue disorders arthralgia	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Constipation	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Metabolism and nutrition disorders Decreased apetite	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Nervous system disorders Depression	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Diarrhea	9.3% 10/108 • Number of events 10 • From the baseline to the study entry date
Nervous system disorders Dizziness	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders dry skin	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Infections and infestations Enterovirus infection	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Respiratory, thoracic and mediastinal disorders Epistaxis	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Fatigue	4.6% 5/108 • Number of events 5 • From the baseline to the study entry date
Gastrointestinal disorders Flatulence	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Nervous system disorders headache	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders Hyperhydrosis	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Metabolism and nutrition disorders Hyperlipidaemia	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Metabolism and nutrition disorders Hyperuricaemia	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Nervous system disorders Memory impairment	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Musculoskeletal and connective tissue disorders Muscle spasm	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Musculoskeletal and connective tissue disorders Musculoskeletal pain	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders Nail disorder	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Respiratory, thoracic and mediastinal disorders Nasal disorder	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Nausea	3.7% 4/108 • Number of events 4 • From the baseline to the study entry date
General disorders Oedema peripheral	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Musculoskeletal and connective tissue disorders Pain in extremity	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
General disorders Pyrexia	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders rash	1.9% 2/108 • Number of events 2 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders Skin disorders	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders Skin fissures	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Eye disorders Strabismus	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Gastrointestinal disorders Vomiting	2.8% 3/108 • Number of events 3 • From the baseline to the study entry date
Reproductive system and breast disorders Vulvovaginal dryness	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date
Skin and subcutaneous tissue disorders Pain of skin	0.93% 1/108 • Number of events 1 • From the baseline to the study entry date

Additional Information

Medical Director (Jeanne Suissa)

Pierre Fabre Médicament

Phone: +33149108269

Email: jeanne.suissa@pierre-fabre.com

Results disclosure agreements

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