Generalized Myasthenia GravisPhase 3NCT05644561
Evaluation of PK, PD, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants With Generalized Myasthenia Gravis
A Phase 3 study testing Ravulizumab in people with Generalized Myasthenia Gravis and gMG, run by Alexion Pharmaceuticals, Inc. at 11 sites in 5 countries.
Trial summary
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Generalized Myasthenia Gravis, gMG
- What is being tested?
- Ravulizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 24 Jun 2023to 30 Jun 2028
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
- Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
- Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.
- Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.
- All participants must be vaccinated against meningococcal infection
Who cannot join
- Medical Conditions
- Any untreated thymic malignancy, carcinoma, or thymoma.
- Participants with a history of treated benign thymoma
- History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening
- History of N meningitidis infection
- Known to be human immunodeficiency virus (HIV) positive
- History of unexplained infections
- Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to the start of the Screening Period
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
United States4 sites
Research Site
Boston, Massachusetts, 02115
Research Site
Chapel Hill, North Carolina, 27514
Research Site
Los Angeles, California, 90078
Research Site
Philadelphia, Pennsylvania, 19104
Italy3 sites
Research Site
Milan, 20133
Research Site
Roma, 00165
Research Site
Torino, 10126
About this study
Official title: Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)
The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
- Conditions
- Generalized Myasthenia Gravis, gMG
- Being tested
- Ravulizumab
- Keywords
- Generalized Myasthenia Gravis, gMG
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Ravulizumab will be administered by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Ravulizumab Intravenous (IV) Infusion | Experimental | All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment. Receives: Drug: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum Concentration of Ravulizumab
Measured: Day 1 predose through Week 18 predose
Serum Free C5 Concentration
Measured: Day 1 predose through Week 18 predose
Secondary outcomes (12)
Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
Measured: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18
Measured: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18
Measured: Baseline, Up to Week 18
Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18
Measured: Week 10, Up to Week 18
Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18
Participants ≥8 years of age will be evaluated.
Measured: Baseline, Up to Week 18
Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18
Participants <8 years of age will be evaluated.
Measured: Baseline, Up to Week 18
Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18
Measured: Baseline through Week 18
Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18
Measured: Baseline through Week 18
Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
Stable is defined as a ±5-point change from Baseline.
Measured: Baseline through Week 18
Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline
Stable is defined as a ±3-point change from baseline.
Measured: Baseline through Week 18
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Measured: Baseline up to Week 126 (8 weeks after last dose of study drug)
Number of Participants With Anti-Drug Antibody (ADA) at Week 18
Measured: Baseline through Week 18
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2022
- Study start24 Jun 2023
- Primary completion15 Jul 2026 (estimated)
- Study completion30 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05644561
- Sponsor's protocol ID
- ALXN1210-MG-319
- Status last verified
- Apr 2026
- First submitted
- 1 Dec 2022
- Last updated
- 17 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3, Open-label, Single-arm, Multicenter Study Evaluating Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of IV Ravulizumab in Pediatric Participants (6 to <18 Years) With Generalized Myasthenia Gravis (gMG)
- ClinicalTrials.gov ID
- NCT05644561
- Acronym / sponsor ID
- ALXN1210-MG-319
- Conditions
- Generalized Myasthenia Gravis, gMG
- Keywords
- Generalized Myasthenia Gravis, gMG
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jun 2023 (actual)
- Primary completion
- 15 Jul 2026 (estimated)
- Study completion
- 30 Jun 2028 (estimated)
- First posted
- 9 Dec 2022 (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 purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ravulizumab Intravenous (IV) Infusion | Experimental | Drug: RavulizumabAll participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
- Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
- Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.
- Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.
- All participants must be vaccinated against meningococcal infection
Key exclusion criteria (8 listed)
- Medical Conditions
- Any untreated thymic malignancy, carcinoma, or thymoma.
- Participants with a history of treated benign thymoma
- History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to Screening
- History of N meningitidis infection
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 |
|---|---|---|
| RavulizumabRavulizumab will be administered by intravenous (IV) infusion. | Drug | Ravulizumab Intravenous (IV) Infusion |
Primary outcome
Serum Concentration of Ravulizumab
Time frame: Day 1 predose through Week 18 predose
Serum Free C5 Concentration
Time frame: Day 1 predose through Week 18 predose
12 secondary outcomes
Change From Baseline in The Quantitative Myasthenia Gravis (QMG) Total Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change From Baseline in Myasthenia Gravis Composite (MGC) Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change in Status from Week 10 in Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS) as Assessed by the Investigator or Neurologist at Up to Week 18
Time frame: Week 10, Up to Week 18
Change from Baseline in Neurology Quality of Life (Neuro QoL) Pediatric Fatigue Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Change from Baseline in Patient-reported Outcomes Measurement Information System (PROMIS) Parent Proxy - Fatigue Score at Up to Week 18
Time frame: Baseline, Up to Week 18
Number of Participants With ≥5-point Reduction Compared to Baseline in the QMG Total Score Over Time Through Week 18
Time frame: Baseline through Week 18
Number of Participants With ≥3 point Reduction Compared to Baseline in the MG-ADL Total Score Over Time Through Week 18
Time frame: Baseline through Week 18
Number of Participants That Improve or Remain Stable in QMG Total Score at Week 18 Compared to Baseline
Time frame: Baseline through Week 18
Number of Participants That Improve or Remain Stable in MG ADL Total Score at Week 18 Compared to Baseline
Time frame: Baseline through Week 18
Number of Participants With Treatment Emergent Adverse Events and Serious Adverse Events
Time frame: Baseline up to Week 126 (8 weeks after last dose of study drug)
Number of Participants With Anti-Drug Antibody (ADA) at Week 18
Time frame: Baseline through Week 18
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 17 Apr 2026.
- 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 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 Ravulizumab in people with Generalized Myasthenia Gravis and gMG, run by Alexion Pharmaceuticals, Inc. at 11 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 5 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 17 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 11 sites in United States, Italy, Serbia, Japan, Switzerland. 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?
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?
Serum Concentration of Ravulizumab (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?
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 17 Apr 2026.