AXIS MG Patient Registry (AXIS)

August 14, 2026 updated by: Autoimmune Neurology Alliance

AXIS Autoimmune Neurology Registry

The goal of the AXIS Autoimmune Neurology Registry is to learn about the experiences of adults living with autoimmune neurological disorders, starting with myasthenia gravis (MG), over time.

The main questions the registry aims to answer are:

  • How do symptoms, daily functioning, and quality of life change over time?
  • What treatments do participants use, and how are these treatments related to their symptoms and health?
  • What challenges do participants experience with treatment, including side effects, treatment burden, missed doses, and access to care?

Participants will:

  • Join the registry and complete an enrollment survey (online or through a mobile app)
  • Complete a brief online survey about their MG approximately once a month
  • Provide information that can help confirm their MG diagnosis
  • Optionally, share their medical records with the research team

This is an observational study. The registry will not assign treatments or ask participants to change their medical care. Information collected through the registry may help researchers better understand MG and improve future research and care.

Study Overview

Status

Recruiting

Conditions

Detailed Description

The AXIS Autoimmune Neurology Registry is a prospective, longitudinal, observational registry of adults in the United States with autoimmune neurological disorders. The registry is beginning with myasthenia gravis (MG), and participants with MG are currently being enrolled. The registry may expand to include other autoimmune neurological disorders in the future.

The registry is decentralized. Participants complete study activities remotely through a secure website or mobile application. The registry does not assign treatments and does not ask participants to change their usual medical care.

The registry collects information directly from participants to support research on the natural history and real-world management of MG. At enrollment, participants provide information about their MG diagnosis, whether they have ocular or generalized MG, age at diagnosis, demographic characteristics, education, employment, other medical conditions, prior testing, and treatments they have used.

Participants are also asked about MG-related antibody testing, including acetylcholine receptor (AChR), muscle-specific kinase (MuSK), low-density lipoprotein receptor-related protein 4 (LRP4), agrin, and other antibody tests. The registry collects participant-reported information about chest imaging, thymus findings, and thymectomy when applicable.

Participants identify MG treatments they have used and treatments taken during the prior month. These may include symptomatic therapies, corticosteroids, other immunosuppressive therapies, biologic or targeted therapies, intravenous or subcutaneous immunoglobulin, and plasma exchange. Additional questions may collect information about current and prior prednisone doses.

At enrollment and during monthly follow-up, participants complete standardized measures of MG symptoms, functioning, and quality of life. These include:

  • The Myasthenia Gravis Activities of Daily Living profile (MG-ADL), which asks about talking, chewing, swallowing, breathing, brushing teeth or combing hair, rising from a chair, double vision, and eyelid droop during the previous week
  • The revised 15-item Myasthenia Gravis Quality of Life questionnaire (MG-QOL15r), which asks about the effect of MG on areas such as social activities, work, family responsibilities, mobility, driving, personal care, emotional well-being, and enjoyment of activities during the previous four weeks

Participants are also asked to describe the severity of their MG symptoms at their worst during the prior month as a patient-reported proxy of MGFA Clinical Classification. Monthly questionnaires collect information about MG exacerbations and healthcare use, including emergency department visits, intensive care unit stays, and the use of mechanical ventilation. Questions may change or use branching logic based on a participant's earlier responses.

Participants complete an enrollment questionnaire and an initial monthly questionnaire when they join. After enrollment, they are asked to complete a follow-up questionnaire approximately once per month. Participants may pause a questionnaire, save their progress, and return later.

Participants provide information indicating that a healthcare provider has diagnosed them with MG. They are also asked to provide information that can help the research team confirm the diagnosis. Participants may confirm their diagnosis through one or more of the following methods:

  • Uploading a laboratory report showing a positive MG-related antibody test
  • Uploading another medical document supporting the MG diagnosis
  • Authorizing the registry to contact their healthcare provider
  • Authorizing the registry to request relevant medical records
  • Connecting electronic medical records through an available secure process

Sharing medical records is optional. Participants may continue in the registry if their diagnosis has not yet been confirmed, although diagnosis confirmation may be required for certain participant incentives. Authorized registry staff review submitted documentation or information received from healthcare providers to determine whether the diagnosis has been confirmed.

The registry uses structured electronic questionnaires and data-quality procedures to support accurate and consistent data collection. Required fields, response options, date and age validations, branching rules, and other predefined checks are built into the questionnaires. Responses may also be reviewed for completeness, ambiguity, values outside expected ranges, and inconsistencies with other information in the registry. Participants may be contacted when clarification is needed.

Source data verification may be conducted by comparing participant-reported information with uploaded diagnostic documents, laboratory reports, healthcare-provider confirmation, connected electronic health information, or medical records authorized by the participant. Because medical-record sharing is optional, the amount of externally verified information may differ among participants.

Study data are associated with a unique participant code. Information that directly identifies participants is stored separately, and access is limited to authorized personnel who need the information for registry operations, participant support, diagnosis verification, monitoring, or other approved purposes. Data used for research analyses are de-identified.

The Principal Investigator and the Scientific Committee are responsible for monitoring the quality and integrity of the registry. The registry may also be reviewed, audited, or inspected by the institutional review board, regulatory authorities, sponsor representatives, and authorized quality or compliance personnel. The electronic system maintains records of informed consent and participant activity that can be provided for monitoring, audit, or inspection.

Registry procedures are governed by the study protocol and supporting study documents. These procedures address informed consent, participant recruitment, questionnaire administration, diagnosis confirmation, medical-record authorization, participant communications and reminders, data management, confidentiality, data sharing, withdrawal, monitoring, and changes to registry procedures. Questionnaire specifications document the questions, response options, required fields, and branching logic used for data collection.

There is no fixed maximum sample size. Broad recruitment is planned to support analyses of the overall MG population and clinically relevant subgroups, including groups based on MG type, symptom severity, antibody status, treatment use, age, and other participant characteristics.

Statistical methods will depend on the research question and may include descriptive statistics, comparisons among participant subgroups, regression modeling, longitudinal analyses, and survival analyses. Analyses may evaluate changes in symptoms, functioning, quality of life, treatment use, and healthcare use over time.

Missing data are expected in this long-term observational registry. Electronic and email reminders are used to encourage participants to complete enrollment, diagnosis-confirmation, and monthly questionnaires. Other follow-up methods may include telephone calls or mail when appropriate. Data-quality reviews will identify missing or inconsistent responses. The methods used to address missing data in an analysis will depend on the research question, the variables involved, and the amount and pattern of missingness.

The registry is designed to continue over many years. Research questions, questionnaires, variables, and analyses may be updated as clinical practice changes, additional treatments become available, other autoimmune neurological disorders are added, and new research needs emerge. Any material changes to the protocol or participant-facing study materials will undergo required review and approval before implementation.

Study Type

Observational

Enrollment (Estimated)

1000

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Massachusetts
      • Framingham, Massachusetts, United States, 01702
        • Recruiting
        • Alira Health - Virtual/Remote Site - Can Be Completed 100% Online - All US Residents Accepted
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jared R Adams, MD, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Adults in the United States with a healthcare provider diagnosis of myasthenia gravis will be recruited from the community. Participants may be identified through healthcare providers, patient advocacy and research organizations, existing patient communities or registries, digital and social media outreach, and direct communications. The registry is decentralized, so participants may enroll and complete study activities remotely from locations across the United States.

Description

Inclusion Criteria:

  • Physician-confirmed diagnosis of myasthenia gravis (MG)
  • Willing and legally able to provide consent
  • Lives in the United States

Exclusion Criteria:

  • Unwilling or unable to provide consent or comprehend and complete questionnaires
  • Cognitively impaired adults/unable to comprehend or understand the consent or overall study information

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Myasthenia Gravis (MG)
Adults with confirmed diagnosis of MG

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
MG-ADL
Time Frame: through study completion, up to 10 years
The Myasthenia Gravis Activities of Daily Living (MG-ADL) profile assesses the severity of myasthenia gravis symptoms during the previous week. Participants rate 8 activities: talking, chewing, swallowing, breathing, brushing teeth or combing hair, rising from a chair, double vision, and eyelid droop. Each item is scored from 0 to 3. Item scores are summed for a total score ranging from 0 to 24. A score of 0 indicates no reported impairment, and higher scores indicate greater symptom severity and functional impairment.
through study completion, up to 10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
MG-QOL15r
Time Frame: through study completion, up to 10 years
The revised 15-item Myasthenia Gravis Quality of Life (MG-QOL15r) questionnaire assesses how myasthenia gravis affected the participant's life during the previous 4 weeks. Participants rate 15 items covering physical functioning, social activities, work, family responsibilities, mobility, personal care, emotional well-being, and enjoyment of activities. Each item is scored from 0 to 2. Item scores are summed for a total score ranging from 0 to 30. A score of 0 indicates no reported effect on quality of life, and higher scores indicate a greater negative effect of myasthenia gravis on quality of life.
through study completion, up to 10 years
MGFA Clinical Classification (patient-reported proxy)
Time Frame: through study completion, up to 10 years
A patient-reported proxy for the Myasthenia Gravis Foundation of America Clinical Classification, based on the participant's MG symptoms at their worst during the past month. Participants select 1 of 5 ordered categories: Class I, weakness limited to the eye or eyelid muscles; Class II, mild weakness affecting other muscles; Class III, moderate weakness affecting other muscles; Class IV, severe weakness affecting other muscles; or Class V, use of a breathing tube, except when planned for surgery. Eye or eyelid weakness may also occur in Classes II-IV. Higher classes indicate more severe MG. This proxy is not a clinician-assessed classification.
through study completion, up to 10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Autoimmune Neurology Alliance

Collaborators

Alira Health

Investigators

  • Study Chair: Richard J Nowak, MD, MS, Yale University
  • Study Director: Gary R Cutter, PhD, University of Alabama at Birmingham
  • Study Director: Neelam Goyal, MD, Stanford University
  • Study Director: Srikanth Muppidi, MD, Stanford University
  • Principal Investigator: Jared R Adams, MD, PhD, Alira Health

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 19, 2026

Primary Completion (Estimated)

March 1, 2036

Study Completion (Estimated)

March 1, 2036

Study Registration Dates

First Submitted

August 4, 2026

First Submitted That Met QC Criteria

August 7, 2026

First Posted (Actual)

August 12, 2026

Study Record Updates

Last Update Posted (Actual)

August 17, 2026

Last Update Submitted That Met QC Criteria

August 14, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AXIS (Alias Study Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

De-identified individual participant data may be made available to qualified researchers for approved research projects. Requests will be reviewed by the AXIS Registry Scientific Committee and/or other applicable scientific or governance bodies. Access will be subject to an approved research proposal, applicable data-use agreements, participant consent, privacy protections, and any legal, ethical, or contractual requirements. A data dictionary describing the shared variables will be provided with approved datasets. Direct identifiers will not be shared.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • ICF

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe