Trial Outcomes & Findings for A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness. (NCT NCT03770403)
NCT ID: NCT03770403
Last Updated: 2023-07-14
Results Overview
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram \[ECG\], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
151 participants
Primary outcome timeframe
TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
Results posted on
2023-07-14
Participant Flow
This Phase III, open-label study was a follow-on study of ARGX-113-1704 (NCT03669588) and was conducted in participants with myasthenia gravis having generalized muscle weakness at 51 investigational sites in 14 countries between 01 Mar 2019 and 30 Jun 2022. This study was conducted in 2 sequential parts: Part A (1 year) and Part B (\<=2 years).
Participants from ARGX-113-1704 who either completed that study or required retreatment that could not be completed during a treatment cycle in that study were included in this study to receive efgartigimod. A total of 151 participants rolled over to this study and 145 of them received at least 1 dose of study treatment. Participants starting part B of this study were eligible to roll over in ARGX-113-2002 study.
Participant milestones
| Measure |
Efgartigimod
Participants were administered efgartigimod intravenous (IV) 10 milligrams/kilograms (mg/kg) over 1 hour every 7 days for 4 administrations per treatment period (TP) for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an intertreatment period (ITP) of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Overall Study
STARTED
|
145
|
|
Overall Study
COMPLETED
|
28
|
|
Overall Study
NOT COMPLETED
|
117
|
Reasons for withdrawal
| Measure |
Efgartigimod
Participants were administered efgartigimod intravenous (IV) 10 milligrams/kilograms (mg/kg) over 1 hour every 7 days for 4 administrations per treatment period (TP) for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an intertreatment period (ITP) of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Death
|
5
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Sponsor decision
|
1
|
|
Overall Study
Study terminated by Sponsor
|
1
|
|
Overall Study
Withdrawal by Subject
|
13
|
|
Overall Study
Other
|
88
|
Baseline Characteristics
A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
Baseline characteristics by cohort
| Measure |
Efgartigimod
n=145 Participants
Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Age, Continuous
|
47.0 years
STANDARD_DEVIATION 14.76 • n=93 Participants
|
|
Sex: Female, Male
Female
|
103 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
42 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
2 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Asian
|
11 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
5 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
White
|
126 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Multiple
|
1 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Japanese
|
10 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
9 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic or Latino
|
126 Participants
n=93 Participants
|
|
Anti-acetylcholine receptor antibodies (AChR-Ab) status
AChR-Ab seropositive
|
111 Participants
n=93 Participants
|
|
Anti-acetylcholine receptor antibodies (AChR-Ab) status
AChR-Ab seronegative
|
34 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 yearsPopulation: The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study. Only those participants with AChR-positive status are included in this analysis.
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram \[ECG\], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.
Outcome measures
| Measure |
Efgartigimod
n=111 Participants
Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants
TEAE
|
92 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants
Treatment-emergent SAE
|
28 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants
TEAEs leading to study drug discontinuation
|
10 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants
Fatal TEAE
|
4 Participants
|
SECONDARY outcome
Timeframe: TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 yearsPopulation: The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
Overall population included both AChR-Ab seropositive and AChR-Ab seronegative participants. An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (ECG, radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. An SAE was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.
Outcome measures
| Measure |
Efgartigimod
n=145 Participants
Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
TEAE
|
124 Participants
|
|
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
Treatment-emergent SAE
|
36 Participants
|
|
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
TEAEs leading to study drug discontinuation
|
12 Participants
|
|
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
Fatal TEAE
|
5 Participants
|
Adverse Events
Efgartigimod
Serious events: 36 serious events
Other events: 83 other events
Deaths: 5 deaths
Serious adverse events
| Measure |
Efgartigimod
n=145 participants at risk
Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Cardiac disorders
Acute myocardial infarction
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Cardiac disorders
Arrhythmia
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Cardiac disorders
Atrial fibrillation
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Cardiac disorders
Cardiac failure congestive
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Cardiac disorders
Defect conduction intraventricular
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Eye disorders
Retinal detachment
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
General disorders
Death
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
COVID-19
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
COVID-19 pneumonia
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Dysentery
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Pneumonia
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Pneumonia escherichia
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Pseudomonal sepsis
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Septic shock
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Urinary tract infection
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Investigations
SARS-CoV-2 test positive
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Metabolism and nutrition disorders
Type 1 diabetes mellitus
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma of colon
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatocellular carcinoma
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung neoplasm malignant
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pancreatic carcinoma metastatic
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma of the vulva
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Uterine leiomyoma
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Nervous system disorders
Cerebral venous sinus thrombosis
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Nervous system disorders
Myasthenia gravis
|
4.8%
7/145 • Number of events 7 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Nervous system disorders
Myasthenia gravis crisis
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Nervous system disorders
Stupor
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Renal and urinary disorders
Acute kidney injury
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Renal and urinary disorders
Bladder neck obstruction
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.4%
2/145 • Number of events 2 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis aspiration
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary mass
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Surgical and medical procedures
Shoulder arthroplasty
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Surgical and medical procedures
Spinal decompression
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Surgical and medical procedures
Spinal operation
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Vascular disorders
Shock
|
0.69%
1/145 • Number of events 1 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
Other adverse events
| Measure |
Efgartigimod
n=145 participants at risk
Participants were administered efgartigimod IV 10 mg/kg over 1 hour every 7 days for 4 administrations per TP for 3 weeks in this study irrespective of whether they received efgartigimod or placebo in ARGX-113-1704. After the fourth infusion, participants entered an ITP of variable duration (minimum 4 weeks between treatment periods). Subsequent TPs were implemented according to clinical response.
|
|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
9.0%
13/145 • Number of events 18 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Gastrointestinal disorders
Nausea
|
6.2%
9/145 • Number of events 13 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
General disorders
Pyrexia
|
7.6%
11/145 • Number of events 11 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
COVID-19
|
11.0%
16/145 • Number of events 16 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Nasopharyngitis
|
13.8%
20/145 • Number of events 24 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Infections and infestations
Urinary tract infection
|
8.3%
12/145 • Number of events 18 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.3%
12/145 • Number of events 15 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Nervous system disorders
Headache
|
24.8%
36/145 • Number of events 103 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.5%
8/145 • Number of events 8 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
|
Vascular disorders
Hypertension
|
5.5%
8/145 • Number of events 10 • TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
The Safety Analysis set consisted of all participants who rolled over from ARGX-113-1704 and received \>=1 dose or part of a dose of efgartigimod in this study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place