A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness. (ADAPT+)
Primary Purpose
Generalized Myasthenia Gravis
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ARGX-113
Sponsored by
About this trial
This is an interventional treatment trial for Generalized Myasthenia Gravis
Eligibility Criteria
Inclusion Criteria:
- Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
- Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.
Other more specific inclusion criteria are further defined in the protocol.
Exclusion Criteria:
- Patients who discontinued early from trial ARGX-113-1704 or patients who discontinued early from randomized treatment for pregnancy or rescue reasons or an (S)AE that might jeopardize the safety of the patient in that trial.
- Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women or childbearing potential should have a negative urine pregnancy test at SEB.
- Male patients who are sexually active and do not intend to use effective methods of contraception during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
- Patients with known Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or Human Immunodeficiency Virus (HIV) seropositivity.
Other, more specific exclusion criteria are further defined in the protocol.
Sites / Locations
- Investigator Site 42
- Investigator Site 8
- Investigator Site 33
- Investigator Site 21
- Investigator Site 41
- Investigator Site 4
- Investigator Site 14
- Investigator Site 32
- Investigator Site 2
- Investigator Site 18
- Investigator Site 12
- Investigator Site 1
- Investigator Site 3
- Investigator Site 37
- Investigator Site 11
- Investigator Site 7
- Investigator Site 20
- Investigator Site 25
- Investigator Site 16
- Investigator Site 19
- Investigator Site 30
- Investigator Site 49
- Investigator Site 17
- Investigator Site 50
- Investigator Site 51
- Investigator Site 45
- Investigator Site 31
- Investigator Site 46
- Investigator Site 28
- Investigator Site 35
- Investigator Site 52
- Investigator Site 10
- Investigator Site 5
- Investigator Site 38
- Investigator Site 24
- Investigator Site 13
- Investigator Site 27
- Investigator Site 22
- Investigator Site 23
- Investigator Site 40
- Investigator Site 43
- Investigator Site 47
- Investigator Site 44
- Investigator Site 48
- Investigator Site 36
- Investigator Site 9
- Investigator Site 29
- Investigator Site 6
- Investigator Site 15
- Investigator Site 34
- Investigator Site 39
- Investigator Site 26
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ARGX-113
Arm Description
Outcomes
Primary Outcome Measures
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
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.
Secondary Outcome Measures
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
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.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03770403
Brief Title
A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
Acronym
ADAPT+
Official Title
A Long-Term, Single-Arm, Open-Label, Multicenter, Phase 3 Follow-on Trial of ARGX-113-1704 to Evaluate the Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
March 1, 2019 (Actual)
Primary Completion Date
June 23, 2022 (Actual)
Study Completion Date
June 30, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
argenx
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Generalized Myasthenia Gravis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
151 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ARGX-113
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
ARGX-113
Other Intervention Name(s)
efgartigimod
Intervention Description
Intravenous administration of ARGX-113
Primary Outcome Measure Information:
Title
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
Description
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.
Time Frame
TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
Secondary Outcome Measure Information:
Title
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
Description
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.
Time Frame
TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.
Other more specific inclusion criteria are further defined in the protocol.
Exclusion Criteria:
Patients who discontinued early from trial ARGX-113-1704 or patients who discontinued early from randomized treatment for pregnancy or rescue reasons or an (S)AE that might jeopardize the safety of the patient in that trial.
Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women or childbearing potential should have a negative urine pregnancy test at SEB.
Male patients who are sexually active and do not intend to use effective methods of contraception during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
Patients with known Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or Human Immunodeficiency Virus (HIV) seropositivity.
Other, more specific exclusion criteria are further defined in the protocol.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sabine Coppieters, MD
Organizational Affiliation
argenx
Official's Role
Study Director
Facility Information:
Facility Name
Investigator Site 42
City
Carlsbad
State/Province
California
ZIP/Postal Code
92011
Country
United States
Facility Name
Investigator Site 8
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Investigator Site 33
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Investigator Site 21
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Investigator Site 41
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32209
Country
United States
Facility Name
Investigator Site 4
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Investigator Site 14
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Investigator Site 32
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Investigator Site 2
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Investigator Site 18
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Investigator Site 12
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Investigator Site 1
City
Cordova
State/Province
Tennessee
ZIP/Postal Code
38018
Country
United States
Facility Name
Investigator Site 3
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Investigator Site 37
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Investigator Site 11
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Investigator Site 7
City
Ghent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Investigator Site 20
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Facility Name
Investigator Site 25
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Investigator Site 16
City
Brno
ZIP/Postal Code
625 00
Country
Czechia
Facility Name
Investigator Site 19
City
Ostrava-Poruba
ZIP/Postal Code
70852
Country
Czechia
Facility Name
Investigator Site 30
City
Praha
ZIP/Postal Code
12800
Country
Czechia
Facility Name
Investigator Site 49
City
Aarhus
Country
Denmark
Facility Name
Investigator Site 17
City
Copenhagen
ZIP/Postal Code
DK-2100
Country
Denmark
Facility Name
Investigator Site 50
City
Bordeaux
Country
France
Facility Name
Investigator Site 51
City
Marseille
Country
France
Facility Name
Investigator Site 45
City
Tbilisi
ZIP/Postal Code
0112
Country
Georgia
Facility Name
Investigator Site 31
City
Tbilisi
ZIP/Postal Code
0114
Country
Georgia
Facility Name
Investigator Site 46
City
Tbilisi
ZIP/Postal Code
0114
Country
Georgia
Facility Name
Investigator Site 28
City
Berlin
ZIP/Postal Code
10117
Country
Germany
Facility Name
Investigator Site 35
City
Budapest
ZIP/Postal Code
1204
Country
Hungary
Facility Name
Investigator Site 52
City
Szeged
Country
Hungary
Facility Name
Investigator Site 10
City
Milano
State/Province
MI
ZIP/Postal Code
20133
Country
Italy
Facility Name
Investigator Site 5
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Investigator Site 38
City
Roma
ZIP/Postal Code
00189
Country
Italy
Facility Name
Investigator Site 24
City
Sapporo
State/Province
Hokkaido
ZIP/Postal Code
060-8543
Country
Japan
Facility Name
Investigator Site 13
City
Hanamaki-shi
State/Province
Iwate
ZIP/Postal Code
025-0075
Country
Japan
Facility Name
Investigator Site 27
City
Sendai
State/Province
Miyagi
ZIP/Postal Code
983-8520
Country
Japan
Facility Name
Investigator Site 22
City
Suita
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Investigator Site 23
City
Ōsaka-sayama
State/Province
Osaka
ZIP/Postal Code
589-8511
Country
Japan
Facility Name
Investigator Site 40
City
Meguro
State/Province
Tokyo
ZIP/Postal Code
153-8515
Country
Japan
Facility Name
Investigator Site 43
City
Shinjuku-Ku
State/Province
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Investigator Site 47
City
Chiba
Country
Japan
Facility Name
Investigator Site 44
City
Hiroshima
ZIP/Postal Code
730-8518
Country
Japan
Facility Name
Investigator Site 48
City
Minato
Country
Japan
Facility Name
Investigator Site 36
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
Investigator Site 9
City
Gdańsk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Investigator Site 29
City
Katowice
ZIP/Postal Code
40-123
Country
Poland
Facility Name
Investigator Site 6
City
Kraków
ZIP/Postal Code
31-505
Country
Poland
Facility Name
Investigator Site 15
City
Warszawa
ZIP/Postal Code
02-097
Country
Poland
Facility Name
Investigator Site 34
City
Novosibirsk
ZIP/Postal Code
630087
Country
Russian Federation
Facility Name
Investigator Site 39
City
Samara
ZIP/Postal Code
443095
Country
Russian Federation
Facility Name
Investigator Site 26
City
Belgrade
ZIP/Postal Code
11000
Country
Serbia
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
34146511
Citation
Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9. Erratum In: Lancet Neurol. 2021 Aug;20(8):e5.
Results Reference
derived
Learn more about this trial
A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
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