AzTrials
AzTrials

Generalized Myasthenia Gravis (gMG)Phase 3NCT07215949

Zilucoplan for Severe gMG Exacerbations

A Phase 3 study testing Zilucoplan® in people with Generalized Myasthenia Gravis (gMG), run by Miriam Freimer at 3 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Generalized Myasthenia Gravis (gMG)
What is being tested?
Zilucoplan®
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
3 sitesUnited States
Study dates
20 Jan 2026to 1 Apr 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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patient determined to have severe MG exacerbation (e.g. bulbar and/or respiratory symptoms requiring hospitalization, neck extension weakness)
  • MGFA class II - IVb
  • Male or female aged ≥18
  • MG-ADL ≥6 in non-ocular domains
  • Serology - AChR antibody positive (or historically available data)
  • If female of child-bearing potential (i.e., not surgically sterile or post-menopausal defined as age > 51 years without menses for ≥ 2 years), negative serum pregnancy test at screening
  • Women of child-bearing potential or men with sexual partners of childbearing potential must be willing to use an acceptable method of birth control for the duration of the study and for 40 days after the last dose of study drug therapy. Acceptable methods of birth control include abstinence, oral contraceptives, the contraceptive patch, intra-uterine device, the contraceptive ring, and or barrier contraception such as condoms with spermicide.
  • Completed or updated meningococcal vaccination or initiated meningococcal vaccination with appropriate antibiotic prophylaxis according to current USPI and ACIP guidelines

Who cannot join

  • History of meningococcal disease
  • Participants requiring intubation prior to study start.
  • Recent significant infections which could have caused exacerbation e.g. sepsis and wound infections
  • Pregnancy or lactating
  • Recent surgery (<4 weeks). Minor procedures/surgeries allowed at the discretion of the site principal investigator
  • Current use or known failure of C5 inhibitors in the previous 3 months
Show all 10Show fewer
  • Initiation of plasma exchange or IVIG in the past 4 weeks
  • Participation in concurrent clinical trial with a therapeutic medication
  • Rituximab use in the previous 9 months
  • Any clinically significant condition or illness, which, in the opinion of the PI, would pose a risk to the subject or might confound the study

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Julie Agriesti, MACPR, CCRC contact

614-293-4098

Julie.Agriesti@osumc.edu

United States3 sites, 3 recruiting

  • The Ohio State University

    Columbus, Ohio, 43210

    Karim Kheniser, CCRP, 614-293-6953, Karim.Kheniser@osumc.edu

    Julie Agriesti, MACPR, CCRC, 614-293-4098, Julie.Agriesti@osumc.edu

    Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

    Andrew Heim, MS-CR, CCRP, 913-945-9926, aheim2@kumc.edu

    Mazen Dimachkie, MD

    Not yet recruiting

  • University of South Florida/Tampa General Hospital

    Tampa, Florida, 33620

    Jessica Shaw, MPH, CCRC, 813-974-9413, jessshaw@usf.edu

    Shawn Nguyen, 813-974-0284, nguyen466@usf.edu

    Not yet recruiting

Other trials in United States

About this study

Official title: Zilucoplan Treatment of Severe MG Exacerbations Leading to Hospitalization of Participants With Acetylcholine Receptor Antibody Positive gMG

This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.

Read the detailed description

The primary objective of the study is to evaluate the efficacy of subcutaneous zilucoplan in participants with AChR antibody positive gMG who experience severe exacerbations requiring hospitalization. A total of 15 patients will be enrolled in the study with treatment lasting 12 weeks. Dosing will be weight based.

Patients will be presented with the option of undergoing standard of care plasma exchange or IVIG or to participate in the research study to determine if the use of zilucoplan would rapidly alleviate the most severe symptoms of gMG (respiratory dysfunction and/or bulbar dysfunction). Assessments will include medical history, physical exam, vital signs and bloodwork. MG assessments will also be conducted including the QMG and MG-ADL and patient reported outcome measures like the Myasthenia Gravis Quality of Life 15 item Revised (MGQoL15r) scale. Treatment will start in the hospital and once the patient is discharged, treatment will continue as an outpatient. Current medications and adverse events will also be reviewed and documented. Patients who undergo treatment with zilucoplan will be vaccinated against meningitis prior to starting the study drug and will continue with the recommended set of vaccines for 6 months. Antibiotics will be taken concurrently until patients are fully vaccinated.

The study aims to find a less invasive method to treat MG patients who are experiencing an exacerbation. Utilization of a subcutaneous medication with relatively rapid onset of action may provide a viable alternative to the current therapeutic approaches. If rapid complement inhibition were to prove efficacious in the treatment of acute exacerbations of gMG, this therapy would potentially obviate the need for transfer to tertiary care centers, avoid the potentially hazardous placement of a large bore central venous catheter for plasmapheresis, and potentially reduce hospital length of stay.

Being tested
Zilucoplan®
Keywords
MG, exacerbation, zilucoplan
Medical subject headings
Myasthenia Gravis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Zilucoplan®Drug

    Subcutaneous injections of zilucoplan will be administered daily. The dose is dependent on the body weight of the patient. Each patient will receive a kit with prefilled syringes.

Study groups in NCT07215949
Study groupRoleWhat they receive
ZilucoplanExperimentalSubcutaneous zilucoplan
Receives: Drug: Zilucoplan®

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score

    The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).

    Measured: From baseline to Week 2

Secondary outcomes (1)

  1. Change from baseline to week 12 in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score

    The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).

    Measured: From baseline to week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Oct 2025
  2. Study start20 Jan 2026
  3. Primary completion1 Dec 2027 (estimated)
  4. Study completion1 Apr 2028 (estimated)

Resources

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
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07215949
Sponsor's protocol ID
MG0036-OSU
Status last verified
Feb 2026
First submitted
6 Oct 2025
Last updated
28 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Vu T, Meisel A, Mantegazza R, Annane D, Katsuno M, Aguzzi R, Enayetallah A, Beasley KN, Rampal N, Howard JF. Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. NEJM Evid. 2022 May;1(5):EVIDoa2100066. doi: 10.1056/EVIDoa2100066. Epub 2022 Apr 26.

    PubMed 38319212

  • Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7.

    PubMed 37059508

  • Kusner LL, Kaminski HJ. The role of complement in experimental autoimmune myasthenia gravis. Ann N Y Acad Sci. 2012 Dec;1274(1):127-32. doi: 10.1111/j.1749-6632.2012.06783.x.

    PubMed 23252907

  • Kapoor M, Spillane J, Englezou C, Sarri-Gonzalez S, Bell R, Rossor A, Manji H, Reilly MM, Lunn MP, Carr A. Thromboembolic risk with IVIg: Incidence and risk factors in patients with inflammatory neuropathy. Neurology. 2020 Feb 11;94(6):e635-e638. doi: 10.1212/WNL.0000000000008742. Epub 2019 Dec 18.

    PubMed 31852814

  • Mandawat A, Kaminski HJ, Cutter G, Katirji B, Alshekhlee A. Comparative analysis of therapeutic options used for myasthenia gravis. Ann Neurol. 2010 Dec;68(6):797-805. doi: 10.1002/ana.22139.

    PubMed 21061395

  • Chang CC, Yeh JH, Chiu HC, Liu TC, Chen YM, Jhou MJ, Lu CJ. Assessing the length of hospital stay for patients with myasthenia gravis based on the data mining MARS approach. Front Neurol. 2023 Dec 14;14:1283214. doi: 10.3389/fneur.2023.1283214. eCollection 2023.

    PubMed 38156090

  • Sanders DB, Wolfe GI, Benatar M, Evoli A, Gilhus NE, Illa I, Kuntz N, Massey JM, Melms A, Murai H, Nicolle M, Palace J, Richman DP, Verschuuren J, Narayanaswami P. International consensus guidance for management of myasthenia gravis: Executive summary. Neurology. 2016 Jul 26;87(4):419-25. doi: 10.1212/WNL.0000000000002790. Epub 2016 Jun 29.

    PubMed 27358333

  • Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Strobel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BR, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJ, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Randomized Trial of Thymectomy in Myasthenia Gravis. N Engl J Med. 2016 Aug 11;375(6):511-22. doi: 10.1056/NEJMoa1602489.

    PubMed 27509100

  • Ramizuddin MK. Review on: Myasthenia gravis and telithromycin-myasthenia crisis. IOSR J Dent Med Sci. 2014;13(7):67-97.

  • Gilhus NE. Myasthenia Gravis. N Engl J Med. 2016 Dec 29;375(26):2570-2581. doi: 10.1056/NEJMra1602678. No abstract available.

    PubMed 28029925

  • Howard JF Jr, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R; REGAIN Study Group. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017 Dec;16(12):976-986. doi: 10.1016/S1474-4422(17)30369-1. Epub 2017 Oct 20.

    PubMed 29066163

  • Rodrigues E, Umeh E, Aishwarya, Navaratnarajah N, Cole A, Moy K. Incidence and prevalence of myasthenia gravis in the United States: A claims-based analysis. Muscle Nerve. 2024 Feb;69(2):166-171. doi: 10.1002/mus.28006. Epub 2023 Dec 1.

    PubMed 38040629

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Zilucoplan® in people with Generalized Myasthenia Gravis (gMG), run by Miriam Freimer at 3 sites in United States.

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 8 inclusion and 10 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 28 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. 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 Julie Agriesti, MACPR, CCRC (614-293-4098, Julie.Agriesti@osumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score (time frame: From baseline to Week 2). 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 NCT07215949, which also lists 12 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 28 Aug 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in