Generalized Myasthenia GravisPhase 3NCT06607627
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
A Phase 3 study testing Gefurulimab in people with Generalized Myasthenia Gravis and gMG, run by Alexion Pharmaceuticals, Inc. at 13 sites in 5 countries.
Trial summary
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Generalized Myasthenia Gravis, gMG
- What is being tested?
- Gefurulimab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 13 sitesin 5 countries
- Study dates
- 13 Nov 2024to 23 Jan 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- United States of America (USA) specific inclusion criterion:
- Participant must be 12 to < 18 years of age at the time of signing the informed consent/assent.
- All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.
- Rest of World (ROW) specific inclusion criteria:
- Participant must be 6 to < 18 years of age at the time of signing the informed consent/assent.
- All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs < 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)
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- Global inclusion criteria:
- Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
- Positive serological test for autoantibodies against AChR
Who cannot join
- History of thymectomy, or any other thymic surgery within 12 months prior to Screening
- Untreated thymic malignancy, carcinoma, or thymoma
- History of Neisseria meningitidis infection
- Pregnancy, breastfeeding, or intention to conceive during the course of the study
Trial locations and contacts
13 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
13 sites in 5 countries
Brazil5 sites, 5 recruiting
- Recruiting
Research Site
Joinville, 89202-451
- Recruiting
Research Site
Salvador, 41253-190
- Recruiting
Research Site
São José do Rio Preto, 15090-000
- Recruiting
Research Site
São Paulo, 0438-002
- Recruiting
Research Site
São Paulo, 05403-000
Poland3 sites, 1 recruiting
- Withdrawn
Research Site
Bydgoszcz, 85-065
- Withdrawn
Research Site
Lodz, 93-338
- Recruiting
Research Site
Warsaw, 02-097
United States2 sites, 2 recruiting
- Recruiting
Research Site
Norfolk, Virginia, 23507
- Recruiting
Research Site
Washington D.C., District of Columbia, 20010
Taiwan2 sites, 2 recruiting
- Recruiting
Research Site
New Taipei City, 23561
- Recruiting
Research Site
Taipei, 11101
About this study
Official title: An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
- Conditions
- Generalized Myasthenia Gravis, gMG
- Being tested
- Gefurulimab
- Keywords
- Acetylcholine Receptor antibodies, AChR+, Generalized Myasthenia Gravis, gMG, ALXN1720, anti-acetylcholine receptor antibody-positive, acetylcholine receptor, AChR, myasthenia gravis, MG, complement component 5, C5, VHH antibody, pediatric
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GefurulimabCombination product
Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
| Study group | Role | What they receive |
|---|---|---|
| Gefurulimab | Experimental | On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks. Receives: Combination Product: Gefurulimab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Observed Serum Concentration (Cmax) of Gefurulimab
Measured: Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration
Measured: Day 1 predose through Week 18 predose
Secondary outcomes (8)
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Measured: Day 1 through Week 134
Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18
Measured: Baseline through Week 18
Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18
Measured: Baseline through Week 18
Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18
Measured: Baseline through Week 18
Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18
Measured: Baseline through Week 18
Number of Participants With Anti-drug Antibodies (ADAs)
Measured: Baseline through Week 134
Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses
Measured: Baseline through Week 134
Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status
Measured: Baseline through Week 134
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2024
- Study start13 Nov 2024
- Primary completion15 Dec 2026 (estimated)
- Study completion23 Jan 2029 (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 Generalized Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06607627
- Sponsor's protocol ID
- D6780C00004
- Other IDs
- 2024-515157-17-00, ALXN1720-MG-302, U1111-1327-6615
- Status last verified
- Mar 2026
- First submitted
- 19 Sep 2024
- Last updated
- 1 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Publications
Beltran J, Bolton S, Patel P, Sharma R. Patient engagement in protocol development for rare disease clinical trials: a framework. Res Involv Engagem. 2026 Oct 9;12(1):167. doi: 10.1186/s40900-026-00985-3.
PubMed 42855714
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)
- ClinicalTrials.gov ID
- NCT06607627
- Acronym / sponsor ID
- D6780C00004
- Conditions
- Generalized Myasthenia Gravis, gMG
- Keywords
- Acetylcholine Receptor antibodies, AChR+, Generalized Myasthenia Gravis, gMG, ALXN1720, anti-acetylcholine receptor antibody-positive, acetylcholine receptor, AChR
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Nov 2024 (actual)
- Primary completion
- 15 Dec 2026 (estimated)
- Study completion
- 23 Jan 2029 (estimated)
- First posted
- 23 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.
The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gefurulimab | Experimental | Combination Product: GefurulimabOn Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- Participant must be 12 to < 18 years of age at the time of signing the informed consent/assent.
- All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.
- Participant must be 6 to < 18 years of age at the time of signing the informed consent/assent.
- All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs < 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)
- Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV
Key exclusion criteria (4 listed)
- History of thymectomy, or any other thymic surgery within 12 months prior to Screening
- Untreated thymic malignancy, carcinoma, or thymoma
- History of Neisseria meningitidis infection
- Pregnancy, breastfeeding, or intention to conceive during the course of the study
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 |
|---|---|---|
| GefurulimabCombination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection. | Combination product | Gefurulimab |
Primary outcome
Maximum Observed Serum Concentration (Cmax) of Gefurulimab
Time frame: Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration
Time frame: Day 1 predose through Week 18 predose
8 secondary outcomes
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: Day 1 through Week 134
Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18
Time frame: Baseline through Week 18
Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18
Time frame: Baseline through Week 18
Change in Myasthenia Gravis Activities of Daily Living Profile (MG-ADL) Total Score From Baseline Through Week 18
Time frame: Baseline through Week 18
Number of Participants With ≥ 3-point Reduction From Baseline in the MG-ADL Total Score Through Week 18
Time frame: Baseline through Week 18
Number of Participants With Anti-drug Antibodies (ADAs)
Time frame: Baseline through Week 134
Number of ADA Positive Participants With Pre-existing Immunoreactivity,Treatment-emergent or Treatment-boosted ADA Responses
Time frame: Baseline through Week 134
Number of ADA Positive Participants With Neutralizing Antibody (NAb) Positive or Negative Status
Time frame: Baseline through Week 134
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2026.
- Derived from this study: PubMed 42855714Beltran J, Bolton S, Patel P, Sharma R. Patient engagement in protocol development for rare disease clinical trials: a framework. Res Involv Engagem. 2026 Oct 9;12(1):167. doi: 10.1186/s40900-026-00985-3.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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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.
13 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Gefurulimab in people with Generalized Myasthenia Gravis and gMG, run by Alexion Pharmaceuticals, Inc. at 13 sites in 5 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 9 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Brazil, Poland, United States, Taiwan, Japan. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alexion Pharmaceuticals, Inc. (Sponsor) (1-855-752-2356, clinicaltrials@alexion.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Observed Serum Concentration (Cmax) of Gefurulimab (time frame: Day 1 predose through Week 18 predose). The study lists 2 primary outcomes in all. 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 NCT06607627, 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 1 Apr 2026.