EfgartigimodPhase 3NCT06587867
Seronegative Myasthenia Gravis - Efgartigimod IV
A Phase 3 study testing efgartigimod in people with Efgartigimod and Generalized Myasthenia Gravis, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
This is to study the efficacy, safety and tolerability of efgartigimod in patients with seronegative generalized myasthenia gravis. This is an open label study. There will be 30 participants to enroll at University Health Network Toronto General Hospital. Study duration is 43 weeks from screening to end of study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Efgartigimod, Generalized Myasthenia Gravis
- What is being tested?
- efgartigimod
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteCanada
- Study dates
- 1 Jun 2023to May 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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
- Evidence of signed and dated informed consent document(s) indicating that the subject has been informed of all pertinent aspects of the trial. Subjects must be willing and able to comply with the protocol, complete study assessments, and return for follow-up visits.
- Male or female subjects ≥ 18 years old.
- Diagnosis of SN MG defined as: (a) clinical syndrome consistent with a diagnosis of MG, and not otherwise explained by another condition, (b) abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; and (c) negative serologic test for anti-AChR and anti- MuSK antibodies as confirmed at screening, (d) limited, if any, response to therapy with immunotherapy and/or antiacetylcholinesterase (AChE) treatment. Further testing for low affinity antibodies to rapsyn-clustered AChR by cell-based assays will be done at baseline and the results included as part of subgroup analysis. All patients will have a negative genetic test for congenital myasthenic syndromes by history or at baseline to exclude the possibility of congenital myasthenic syndrome mimicking SN MG.
- MGFA Clinical Classification Class II, III, or IV at the time of screening and baseline.
- Moderate to severe myasthenia gravis as defined by a generalized myasthenia gravis impairment index score > 11 or MG-ADL score of at least 5 (with >50% of the score due to non-ocular symptoms) and a PASS response of "No" and a SSQ of < 70% with at least 6 months of historical data as the baseline.
- Stable or worsening MG as defined by MGII remaining stable or increasing in the 4 week run-in interval.
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- Patients are required to be on a stable dose of their MG treatment (Standard of care-SoC) for at least one month prior to screening. The SoC is limited to AChE inhibitors, steroids and NSISTs (e.g., azathioprine, methotrexate, cyclosporine, tacrolimus, and mycophenolate mofetil. There is no requirement for specific generalised myasthenia gravis therapies.
- Patients who discontinued early from previous trials of efgartigimod for reasons other than pregnancy, rescue therapy or a SAE can be included.
- Females of childbearing potential who are sexually active with a non-sterilized male partner must be willing to use at least one highly effective contraception method from the time of screening and for 3 months after the final dose of efgartigimod.
- Non-sterilized males who are sexually active with a female partner of childbearing potential must be willing to use a condom for the duration of the study and for 3 months after the last dose of efgartigimod. Because male condom is not a highly effective contraception method, it is strongly recommended that female partners of a male study subject also use a highly effective method of contraception throughout this period.
- Vital signs, electrocardiogram (ECG), and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the Investigator.
- Patient has documented IgG >6 g/L within one month of screening
- Vaccinated for COVID-19 at least 2 weeks prior to screening visit.
Who cannot join
- Patients who discontinued early from trials of efgartigimod for pregnancy or rescue reasons or an SAE that was likely to result in a life-threatening situation or pose a serious safety risk.
- Pregnant and lactating women, and those intending to become pregnant during the trial or within 3 months after the last dosing. Women of childbearing potential should have a negative urine pregnancy test at screening and baseline.
- Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 3 months after the last dosing or male patients who plan to donate sperm during the trial or within 3 months after the last dosing.
- Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.
- Patients with known autoimmune disease other than MG (e.g., rheumatoid arthritis) which in the investigator opinion would interfere with an accurate assessment of clinical symptoms.
- Patients with clinical evidence of other significant disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk.
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- Patients with renal/hepatic function impairment as defined by (Cr>1.5 x elevated) and/or (transaminases > 2.5 x elevation) at screening.
- Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
- Patients who have received rituximab or eculizumab in the 6 months before screening.
- Patients who have undergone thymectomy within 3 months of screening.
- Patients who had intravenous immunoglobulin or plasma exchange within 4 weeks of screening.
- Patient who has clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- Patient has received a live or a live-attenuated vaccination during the month before screening
Trial locations and contacts
1 site in 1 country.
Canada1 site, 1 recruiting
- Recruiting
University Health Network, Division of Neurology, Toronto General Hospital
Toronto, Ontario, M5G 2C4
Eduardo Ng, 416-340-3898, Eduardo.Ng@uhn.ca
Vera Bril, MD
About this study
Official title: Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis: an Open-Label Study
This is to study the efficacy, safety and tolerability of efgartigimod in patients with seronegative generalized myasthenia gravis. This is an open label study. There will be 30 participants to enroll at University Health Network Toronto General Hospital. Study duration is 43 weeks from screening to end of study.
Read the detailed description
There will be screening period that includes ECG, safety blood collection, collection of demographics, vital signs, questionnaires, weight, pregnancy test (if applicable), genetic test. If eligible, participants will have run-in period from week 2 to week 5. Baseline is at week 6. Induction phase will be weekly from week 7, 8, and 9. Maintenance phase will be once every 2 weeks thereafter. Observation period will have 4 visits, End of study is at week 43.
- Conditions
- Efgartigimod, Generalized Myasthenia Gravis
- Being tested
- efgartigimod
- Keywords
- seronegative, generalized myasthenia gravis
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efgartigimodBiological
active drug efgartigimod to be administer
| Study group | Role | What they receive |
|---|---|---|
| efgartigimod | Other | active treatment with efgartigimod. Receives: Biological: efgartigimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Myasthenia Gravis Impairment Index (MGII) score
Myasthenia Gravis Impairment Index (MGII) is a composite score based on a patient self-reported multidomain questionnaire as well as physician-documented clinical measures. MGII has 22 patient-reported and 6 physician-assessed items with a final composite score of 84, with higher score signifying greater disability.
Measured: through study completion for 42 weeks
Secondary outcomes (6)
MG-ADL (Myasthenia Gravis-Activities of Daily Living)
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity.
Measured: through study completion for 42 weeks
QMG (Quantitative Myasthenia Gravis) score
The QMG scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity.
Measured: through study completion for 42 weeks
MG-QOL (Myasthenia Gravis Quality of Life)
MG-QOL revised consists of 15 items/ question. These include mobility (9 items), symptoms (3 items), and contentment and emotional well-being (3 items). Items are scored in a Likert scale from 0 to 4 and the total sum score ranges from 0 to 60, where higher scores indicate worse HRQoL.
Measured: through study completion for 42 weeks
PASS (Patient Acceptable Symptom State) response
The PASS response is based on a dichotomous 'Yes' or 'No' response, asking whether a patient is satisfied overall with their current status and thus measures holistic satisfaction with their MG state. The PASS response of "Yes" has been shown to correlate with a favourable MGII and the simple single question (SSQ, percentage of normal).
Measured: through study completion for 42 weeks
Single Simple Question (SSQ)
The SSQ is a question that asks what percentage of normal that the patient feels with respect to their MG. A SSQ threshold ≥72.5% out of normal at 100% predicts an acceptable MG state.
Measured: through study completion for 42 weeks
Myasthenia Gravis Foundation of America (MGFA)
The MGFA classes are pure ocular (class I), mild generalized (class II), moderate generalized (class III), severe generalized (class IV), and intubation/myasthenic crisis (class V). Within the generalized categories II, III, and IV, patients are subclassified as class A if their symptoms are predominantly generalized or class B if their symptoms are predominantly bulbar. The MGFA also has a system to classify patients based on postintervention outcomes and includes remission, defined as 1 year or longer without signs or symptoms and without any symptomatic (pyridostigmine) treatment, and which can be divided in complete (no pharmacologic treatment at all) or pharmacologic remission. Minimal manifestation status is defined as minimal signs or symptoms (no specific timeframe was defined) and pyridostigmine use may be accepted.
Measured: through study completion for 42 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 Jun 2023
- Primary completionMay 2025 (estimated)
- Study completionMay 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for EfgartigimodStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe 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
- Model description
- open label administration of efgartigimod
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Vera Bril, University Health Network, Toronto
- Investigators
- Vera Bril, MD, Principal investigator, Toronto General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06587867
- Sponsor's protocol ID
- UHN 001
- Status last verified
- Aug 2024
- First submitted
- 27 Aug 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Khateb M, Sivadasan A, Barnett-Tapia C, Daniyal L, Fernando L, Chen S, Lovblom LE, Katzberg H, Bril V. Open-label study of efgartigimod in seronegative myasthenia gravis. Ther Adv Neurol Disord. 2025 Oct 31;18:17562864251388019. doi: 10.1177/17562864251388019. eCollection 2025.
PubMed 41180124
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis: an Open-Label Study
- ClinicalTrials.gov ID
- NCT06587867
- Acronym / sponsor ID
- UHN 001
- Conditions
- Efgartigimod, Generalized Myasthenia Gravis
- Keywords
- seronegative, generalized myasthenia gravis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 1 Jun 2023 (actual)
- Primary completion
- May 2025 (estimated)
- Study completion
- May 2025 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is to study the efficacy, safety and tolerability of efgartigimod in patients with seronegative generalized myasthenia gravis. This is an open label study. There will be 30 participants to enroll at University Health Network Toronto General Hospital. Study duration is 43 weeks from screening to end of study.
Detailed description
There will be screening period that includes ECG, safety blood collection, collection of demographics, vital signs, questionnaires, weight, pregnancy test (if applicable), genetic test. If eligible, participants will have run-in period from week 2 to week 5. Baseline is at week 6. Induction phase will be weekly from week 7, 8, and 9. Maintenance phase will be once every 2 weeks thereafter. Observation period will have 4 visits, End of study is at week 43.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- open label administration of efgartigimod
| Arm | Type | Receives |
|---|---|---|
| efgartigimod | Other | Biological: efgartigimodactive treatment with efgartigimod. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Evidence of signed and dated informed consent document(s) indicating that the subject has been informed of all pertinent aspects of the trial. Subjects must be willing and able to comply with the protocol, complete study assessments, and return for follow-up visits.
- Male or female subjects ≥ 18 years old.
- Diagnosis of SN MG defined as: (a) clinical syndrome consistent with a diagnosis of MG, and not otherwise explained by another condition, (b) abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; and (c) negative serologic test for anti-AChR and anti- MuSK antibodies as confirmed at screening, (d) limited, if any, response to therapy with immunotherapy and/or antiacetylcholinesterase (AChE) treatment. Further testing for low affinity antibodies to rapsyn-clustered AChR by cell-based assays will be done at baseline and the results included as part of subgroup analysis. All patients will have a negative genetic test for congenital myasthenic syndromes by history or at baseline to exclude the possibility of congenital myasthenic syndrome mimicking SN MG.
- MGFA Clinical Classification Class II, III, or IV at the time of screening and baseline.
- Moderate to severe myasthenia gravis as defined by a generalized myasthenia gravis impairment index score > 11 or MG-ADL score of at least 5 (with >50% of the score due to non-ocular symptoms) and a PASS response of "No" and a SSQ of < 70% with at least 6 months of historical data as the baseline.
Key exclusion criteria (13 listed)
- Patients who discontinued early from trials of efgartigimod for pregnancy or rescue reasons or an SAE that was likely to result in a life-threatening situation or pose a serious safety risk.
- Pregnant and lactating women, and those intending to become pregnant during the trial or within 3 months after the last dosing. Women of childbearing potential should have a negative urine pregnancy test at screening and baseline.
- Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 3 months after the last dosing or male patients who plan to donate sperm during the trial or within 3 months after the last dosing.
- Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.
- Patients with known autoimmune disease other than MG (e.g., rheumatoid arthritis) which in the investigator opinion would interfere with an accurate assessment of clinical symptoms.
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 |
|---|---|---|
| efgartigimodactive drug efgartigimod to be administer | Biological | efgartigimod |
Primary outcome
Total Myasthenia Gravis Impairment Index (MGII) score
Time frame: through study completion for 42 weeks
Myasthenia Gravis Impairment Index (MGII) is a composite score based on a patient self-reported multidomain questionnaire as well as physician-documented clinical measures. MGII has 22 patient-reported and 6 physician-assessed items with a final composite score of 84, with higher score signifying greater disability.
6 secondary outcomes
MG-ADL (Myasthenia Gravis-Activities of Daily Living)
Time frame: through study completion for 42 weeks
QMG (Quantitative Myasthenia Gravis) score
Time frame: through study completion for 42 weeks
MG-QOL (Myasthenia Gravis Quality of Life)
Time frame: through study completion for 42 weeks
PASS (Patient Acceptable Symptom State) response
Time frame: through study completion for 42 weeks
Single Simple Question (SSQ)
Time frame: through study completion for 42 weeks
Myasthenia Gravis Foundation of America (MGFA)
Time frame: through study completion for 42 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Derived from this study: PubMed 41180124Khateb M, Sivadasan A, Barnett-Tapia C, Daniyal L, Fernando L, Chen S, Lovblom LE, Katzberg H, Bril V. Open-label study of efgartigimod in seronegative myasthenia gravis. Ther Adv Neurol Disord. 2025 Oct 31;18:17562864251388019. doi: 10.1177/17562864251388019. eCollection 2025.
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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.
Vera Bril, MD principal investigator
Toronto General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing efgartigimod in people with Efgartigimod and Generalized Myasthenia Gravis, run by University Health Network, Toronto at 1 site in Canada.
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 13 inclusion and 13 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: status not confirmed, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Eduardo Ng (4163403898, eduardo.ng@uhn.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Myasthenia Gravis Impairment Index (MGII) score (time frame: through study completion for 42 weeks). 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 NCT06587867, which also lists 1 linked publication.
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 19 Sep 2024.