Panorama: Neuromuscular Organoids to understand refractory AChR+ myasthenia gravis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

Panorama uses human neuromuscular organoids to model the autoimmune attack on the neuromuscular junction in adults with generalized AChR-positive myasthenia gravis. The organoids are generated from induced pluripotent stem cells and form functional motor units, allowing testing of autoantibody effects in a human-relevant system. Researchers analyze patient IgG–mediated damage and search for circulating biomarkers that predict treatment response. The study includes both retrospective biomarker work and prospective characterization in adults, with stratification by disease duration and prior therapy. Interventions are not specified in this observational framework, focusing on mechanistic insights and potential predictive markers.

Why it matters

Who can join: the full rules

Inclusion Criteria:

AChR-positive MG participants (all of the following):

* Age \>= 18 years
* Established diagnosis of myasthenia gravis according to international criteria (fluctuating muscle weakness plus at least one of: positive anticholinesterase test; \>10% decrement on repetitive nerve stimulation \[SR-ENG\]; or increased jitter on single-fiber EMG \[SFEMG\])
* Anti-AChR antibody positivity confirmed by serological testing
* Generalized MG stratified by disease duration and response to conventional immunosuppressive therapy into: treatment-naive MG (nMG); treatment-sensitive MG with stable disease and at least 12 months of follow-up (sMG); or treatment-refractory MG with at least 12 months of follow-up (rMG)
* Signed informed consent (prospective component)
* For the retrospective component, availability of biobanked biological samples with a signed specific informed consent

Control participants (all of the following):

* Age \>= 18 years
* Absence of neuromuscular disease
* Absence of known autoimmune disease
* No ongoing immunosuppressive or immunomodulatory therapy
* Negative for anti-AChR and anti-MuSK antibodies
* Age- and sex-matched to the MG groups
* Signed informed consent

Exclusion Criteria:

* Seronegative (AChR-negative) generalized MG, or MG with anti-MuSK or other non-AChR antibodies
* Purely ocular myasthenia gravis
* Other concomitant neuromuscular disease (e.g., Lambert-Eaton myasthenic syndrome, polymyositis, muscular dystrophies)
* Pregnancy or breastfeeding
* Inability to provide informed consent

Where it's running

• Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  Milan, Italy Recruiting

Study contacts

• Study coordinator: Delia Gagliardi, MD, PhD
• Email: delia.gagliardi@policlinico.mi.it
• Phone: 02 5503 3802

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.