A Randomized, Double-Blind, Placebo-Controlled Study of AUR200 in Generalized Myasthenia Gravis
- Trial ID
- 2025-525127-27-00
- Protocol
- AUR-200-2024-02
- Sponsor
- Aurinia Pharmaceuticals Inc.
Trial statistics
Diseases & Conditions
Objectives
The primary objective is to assess the efficacy of generalized myasthenia gravis treatment with aritinercept in phase 2, with phase 1 focused on its safety and tolerability. This is clinically relevant because it determines both the therapeutic effect and the acceptability of the intervention in a patient population with an immune-mediated neuromuscular disorder. The secondary objectives are to investigate the pharmacokinetics of aritinercept, the pharmacodynamics of aritinercept, and the immunogenicity profile of aritinercept.
Participants
The trial population consisted of 32 patients with Generalized Myasthenia Gravis, including males and females 18 to 70 years of age. Participants were generally required to have a body mass index of 18.0 to 36.0 kg/m2 and a body weight greater than 45 kg, as well as a documented antibody-positive disease history and symptomatic generalized myasthenia gravis of MGFA Class II-IV with a minimum MG-ADL score of 6. Enrollment was limited to individuals with stable background treatment when applicable, including specified doses of acetylcholinesterase inhibitors, steroids, or non-steroidal immunosuppressive therapy. Participants were also required to have received age-appropriate vaccinations for immunocompromised individuals, and women of childbearing potential and men with female partners of childbearing potential were subject to contraception requirements and related restrictions. The trial population was selected from patients able and willing to provide informed consent and comply with protocol requirements.
Plans and Procedures
This is a double-blind, randomized, placebo-controlled Phase 1/2 study in patients with generalized myasthenia gravis. The investigational product is administered by subcutaneous injection, with placebo as the comparator. The trial is designed to assess safety and tolerability in Phase 1 and efficacy in Phase 2. The overall trial duration is estimated from 2026-05-12 to 2028-10-31. Study participation begins with a screening visit to confirm eligibility criteria, including diagnosis, disease severity, treatment stability, and other protocol requirements. This is followed by study treatment visits and scheduled follow-up assessments to evaluate safety, pharmacokinetics, immunogenicity, and clinical response. An end-of-study visit is performed at the end of the protocol-defined observation period to complete final assessments. Expected participant involvement extends from screening through the last follow-up and end-of-study evaluation. Early termination may occur if eligibility criteria are not met, if protocol requirements are not followed, if the participant withdraws consent, or if discontinuation is required for safety or other study-defined reasons.
Treatment
The investigational treatment was aritinercept, identified in the source data as AUR200, presented as a solution for injection and administered by subcutaneous injection. The source data do not specify the dose or dosing frequency. In the trial, aritinercept was evaluated in a double-blind and randomized design for safety, tolerability, and efficacy.
The comparator treatment was placebo, identified as isotonic sodium chloride solution 0.9% Braun injection solution. It was also supplied as a solution for injection and administered by subcutaneous injection. The source data do not provide a dose or administration frequency for the placebo. Participant compliance monitoring is not described in the source data.
Efficacy
Efficacy will be assessed in phase 2 by the change from baseline in the Myasthenia Gravis Activities of Daily Living (MG-ADL) total score. This endpoint will be compared between aritinercept and placebo in patients with generalized myasthenia gravis. No additional efficacy parameters, assessment tools, or evaluation timepoints are specified.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Able and willing to sign ICF after receiving information about the Study
- Documented history for anti-AChR, anti-MuSK or anti-LRP4 antibodies
- A total Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of ≥6 at Screening, with >50% of the total score due to non-ocular symptoms
- If receiving ≥1 of the following gMG treatments, on a stable dose of: • Acetylcholinesterase inhibitors (no dose change for 2 weeks prior to Screening) • Steroids (at least 3 months of treatment, no dose change for 1 month prior to Screening) • Non-steroidal immunosuppressive therapy (NSIST) including mycophenolate mofetil (MMF), methotrexate, cyclosporine, tacrolimus or cyclophosphamide (at least 6 months of treatment, no dose change for 3 months prior to Screening) • Azathioprine (AZA) or cladribine (at least 6 months of treatment, no dose change for at least 2 months prior to Screening)
- Patients have received all age-appropriate vaccinations per local or professional guidelines for immunocompromised individuals, per Investigator judgement
- Women of childbearing potential who are heterosexually active must use an acceptable form of contraception from Screening through 60 days after the last dose of Study drug • Acceptable highly effective methods include: combined (estrogen- and progestogen-containing) oral, intravaginal or transdermal hormonal contraception (associated with inhibition of ovulation); progestogen-only oral, injectable or implantable hormonal contraception (associated with inhibition of ovulation); intrauterine device; intrauterine hormone-releasing system; bilateral tubal ligation or occlusion; abstinence; or intercourse with a male partner who has had a vasectomy with medical confirmation of surgical success. • In regions where highly effective contraception is not required per regulatory mandate or local standard practice, additional acceptable methods include: progestogen-only oral, injectable or implantable hormonal contraception (where inhibition of ovulation is not the primary mode of action); male or female condom with or without spermicide; cap, diaphragm or sponge with spermicide; or a combination of male condom with either cap, diaphragm or sponge with spermicide (double barrier methods).
- Men with a female partner of childbearing potential must use an acceptable form of contraception from Screening through 150 days after the last dose of Study drug • Acceptable methods include: male condoms; abstinence; or vasectomy with medical confirmation of surgical success. • Additionally, it is recommended that female partners of childbearing potential utilize an acceptable method of contraception (as described above).
- Men must agree to not donate sperm during the Study and for 150 days after receiving the last dose of Study drug
- Able and willing to comply with the requirements and restrictions of the Study protocol
- Male or female patients, 18-70 years of age on the day ICF is signed
- Body mass index (BMI) 18.0-36.0 kg/m2 and body weight >45 kg
- Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG as confirmed by any 1 of the following: • History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation; • History of positive edrophonium chloride test; • Improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician
Exclusion Criteria
- MGFA Class I and Class V patients
- Clinically significant liver and/or renal impairment, defined as any of the following: • Alanine aminotransferase (ALT) >2 × upper limit of normal (ULN) • Aspartate aminotransferase (AST) >2 × ULN • Total bilirubin >1.5 × ULN; does not apply to patients with Gilbert’s syndrome • Serum creatinine >ULN
- History of alcohol and/or other substance (except caffeine or nicotine) abuse within 1 year prior to Screening
- Current or medical history of any of the following: • Congenital or acquired immunodeficiency (eg, IgA deficiency); • Demyelinating disease such as, but not restricted to, multiple sclerosis, optic neuritis, transverse myelitis or acute or chronic demyelinating polyneuropathy; • Malignancy within 5 years prior to Screening, with the exception of treated patients who are considered cured with at least a 2-year period of remission prior to Screening and minimal risk of recurrence; • Lymphoproliferative disease or previous total lymphoid irradiation; • Active, chronic or severe viral infections (eg, cytomegalovirus, hepatitis B virus, hepatitis C virus) within 3 months prior to Screening. Severe viral infection is defined as active disease requiring antiviral therapy. Patients who test positive for hepatitis B surface antigen and/or hepatitis C at Screening will be excluded; • Active TB or known history of TB. Patients should have a negative TB test with the result reported prior to Day 1. Indeterminate results may be repeated. • Antiphospholipid syndrome or antiphospholipid antibodies at Screening; • History of human immunodeficiency virus (HIV) infection or demonstration of HIV antibodies at Screening
- Have active central nervous system (CNS) lupus (including seizures, psychosis, organic brain syndrome, cerebrovascular accident [CVA], cerebritis or CNS vasculitis) requiring therapeutic intervention within 60 days prior to Screening
- Patients with a history or current evidence of any other cardiac, hepatic, renal, pulmonary, endocrine, neurologic, gastrointestinal, hematologic, oncologic or psychiatric disease as determined by medical/disease history, physical examination, laboratory reports, 12-lead ECG or any findings that, in the view of the Investigator or the Study Medical Team, would compromise the patient’s safety or affect Study conduct
- Uncontrolled hypotension (systolic blood pressure <100 mmHg or diastolic blood pressure <60 mmHg)
- Any condition or circumstances that, in the opinion of the Investigator, may make a patient unlikely or unable to complete the Study or comply with the Study procedures and requirements
- Patient is an employee of the Investigator or Study site, with direct involvement in the Study or other studies under the direction of that Investigator or Study site, as well as family members of the employees or the Investigator
- Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoroquinolones, beta-blockers, etc.)
- Autoimmune disease other than MG (eg, autoimmune thyroiditis, rheumatoid arthritis) that would interfere with an accurate assessment of clinical symptoms
- Have received any B cell-targeted therapy including: • blisibimod, belimumab, inebilizumab or rituximab within 12 months prior to Screening; or • ocrelizumab within 18 months prior to Screening
- Have received treatment with complement inhibitor (eg, eculizumab, ravulizumab, zilucoplan) within 3 months prior to Screening
- Have received FcRn blockers (eg, efgartigimod alfa, rozanolixizumab-noli, nipocalimab) within 3 months prior to Screening
- Have received immunoglobulins given by IV (IVIg), subcutaneous or intramuscular route, or plasma exchange (PLEX), within 1 month prior to Screening
- Thymectomy performed within 3 months prior to Screening or planned to be performed during the Study
- Known or suspected allergy or hypersensitivity, intolerance or contraindication to any component of aritinercept or history of severe hypersensitivity reaction to any monoclonal antibody
- Pregnant, breastfeeding or intending to become pregnant during the Study
- Clinically significant electrocardiogram (ECG) abnormalities at Screening or on Day 1, defined as any of the following: • Average QT interval corrected according to Fridericia’s formula (QTcF) of 3 ECGs ≥450 msec for males and ≥470 msec for females; • Evidence of second- and third-degree atrioventricular (AV) block, complete left bundle branch block (LBBB) or complete right bundle branch block (RBBB); • Features of new ischemia; • Arrhythmia (except premature atrial contractions [PACs] and premature ventricular contractions [PVCs])
- History of hypogammaglobulinemia or serum IgG, IgM or IgA concentrations below the lower limit of normal at Screening
- Have uncontrolled diabetes defined as hemoglobin-A1c value >7.5%
- Blood drawn (>300 mL) within 30 days prior to Screening or receipt of blood products within 30 days prior to Day 1
- Have required recent management of acute or chronic infection as follows: • Currently on any suppressive therapy for a chronic infection (eg, tuberculosis [TB], pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical mycobacteria); • Hospitalization for treatment of infection within 60 days prior to Screening; • Use of parenteral (intravenous or intramuscular) antibiotics (antibacterials, antivirals, anti-fungals or anti-parasitic agents) within 60 days prior to Screening
- Recent live vaccination (within 28 days prior to first dose of Study drug) or planned live vaccination during the Study or within 6 weeks after the last dose of Study drug
- Known positive COVID-19 test result within 7 days prior to Day 1
- Prior treatment with aritinercept
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 Poland | Recruiting | 12 May 2026 | 28 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
AUR200 | Test | SOLUTION FOR INJECTION | SUBCUTANEOUS INJECTION | — | — | PRD13279069 |
Isotone Natriumchloridlösung 0,9 % Braun Injektionslösung | Placebo | INJEKTIONSLÖSUNG | SUBCUTANEOUS INJECTION | — | — | PRD11904224 |