Generalized Myasthenia GravisPhase 3ADAPT+NCT03770403
A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
A Phase 3 study testing ARGX-113 in people with Generalized Myasthenia Gravis, run by argenx at 52 sites in 15 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- ARGX-113
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 151 participants
- Where
- 52 sitesin 15 countries
- Study dates
- 1 Mar 2019to 30 Jun 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
52 sites in 15 countries.
52 sites in 15 countries
United States14 sites
Investigator Site 42
Carlsbad, California, 92011
Investigator Site 2
Chapel Hill, North Carolina, 27599
Investigator Site 37
Charlottesville, Virginia, 22908
Investigator Site 18
Cleveland, Ohio, 44195
Investigator Site 1
Cordova, Tennessee, 38018
Investigator Site 32
Detroit, Michigan, 48201
Investigator Site 41
Jacksonville, Florida, 32209
Investigator Site 14
Kansas City, Kansas, 66160
Investigator Site 8
Los Angeles, California, 90033
Investigator Site 33
Orange, California, 92868
Investigator Site 21
Palo Alto, California, 94304
Investigator Site 12
Portland, Oregon, 97239
Investigator Site 3
San Antonio, Texas, 78229
Investigator Site 4
Tampa, Florida, 33612
Japan10 sites
Investigator Site 47
Chiba
Investigator Site 13
Hanamaki-shi, Iwate, 025-0075
Investigator Site 44
Hiroshima, 730-8518
Investigator Site 40
Meguro City, Tokyo, 153-8515
Investigator Site 48
Minato
Investigator Site 23
Ōsaka-sayama, Osaka, 589-8511
Investigator Site 24
Sapporo, Hokkaido, 060-8543
Investigator Site 27
Sendai, Miyagi, 983-8520
Investigator Site 43
Shinjuku-Ku, Tokyo, 160-0023
Investigator Site 22
Suita, Osaka, 565-0871
Poland4 sites
Investigator Site 9
Gdansk, 80-952
Investigator Site 29
Katowice, 40-123
Investigator Site 6
Krakow, 31-505
Investigator Site 15
Warsaw, 02-097
Czechia3 sites
Investigator Site 16
Brno, 625 00
Investigator Site 19
Ostrava-Poruba, 70852
Investigator Site 30
Prague, 12800
Georgia3 sites
Investigator Site 45
Tbilisi, 0112
Investigator Site 31
Tbilisi, 0114
Investigator Site 46
Tbilisi, 0114
Italy3 sites
Investigator Site 10
Milan, MI, 20133
Investigator Site 5
Naples, 80131
Investigator Site 38
Roma, 00189
Belgium2 sites
Investigator Site 11
Edegem, 2650
Investigator Site 7
Ghent, 9000
Canada2 sites
Investigator Site 25
Montreal, Quebec, H3A 2B4
Investigator Site 20
Toronto, Ontario, M5G 2C4
Denmark2 sites
Investigator Site 49
Aarhus
Investigator Site 17
Copenhagen, DK-2100
Russia2 sites
Investigator Site 34
Novosibirsk, 630087
Investigator Site 39
Samara, 443095
About this study
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
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.
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- ARGX-113
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARGX-113Biological
Also called efgartigimod
Intravenous administration of ARGX-113
| Study group | Role | What they receive |
|---|---|---|
| ARGX-113 | Experimental | Not described Receives: Biological: ARGX-113 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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 outcomes (1)
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.
Measured: TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2018
- Study start1 Mar 2019
- Primary completion23 Jun 2022
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 151 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sabine Coppieters, MD, Study director, argenx
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03770403
- Sponsor's protocol ID
- ARGX-113-1705
- Other IDs
- 2018-002133-37, 2018
- Acronym
- ADAPT+
- Status last verified
- Jun 2023
- First submitted
- 16 Nov 2018
- Last updated
- 14 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Publications
Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front Neurol. 2024 Jan 17;14:1284444. doi: 10.3389/fneur.2023.1284444. eCollection 2023.
PubMed 38318236Howard 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.
PubMed 34146511
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03770403
- Acronym / sponsor ID
- ADAPT+ / ARGX-113-1705
- Conditions
- Generalized Myasthenia Gravis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 151 participants (actual)
- Lead sponsor
- argenxIndustry (pharma, biotech, devices)
- Study start
- 1 Mar 2019 (actual)
- Primary completion
- 23 Jun 2022 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 10 Dec 2018 (actual)
- Results
- Posted 14 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ARGX-113 | Experimental | Biological: ARGX-113 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARGX-113Also: efgartigimodIntravenous administration of ARGX-113 | Biological | ARGX-113 |
Primary outcome
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
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
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.
1 secondary outcome
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38318236Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front Neurol. 2024 Jan 17;14:1284444. doi: 10.3389/fneur.2023.1284444. eCollection 2023.
- Derived from this study: PubMed 34146511Howard 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sabine Coppieters, MD study director
argenx
52 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ARGX-113 in people with Generalized Myasthenia Gravis, run by argenx at 52 sites in 15 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States, Japan, Poland, Czechia, Georgia and 10 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jul 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03770403, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2023.