Clinical trial NCT05311904: Efficacy and Safety of Vaccination Against COVID-19 in Neuromuscular Patients
Official title: Observatory of the Efficacy and Safety of Vaccination Against SARS-CoV-2 in Neuromuscular Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Neuromuscular Diseases
Keywords: COVID-19
vaccination
safety
Lead sponsor: University Hospital, Bordeaux
Sponsor class: OTHER
First posted: 2022-04-05
Enrollment: 1388
Sex: ALL
Minimum age (years): 18
Maximum age (years): 100
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient with a neuromuscular disease

Brief summary: The frequency of severe forms of COVID-19 is higher in people with neuromuscular disease and in severe cases and long hospital stays, the disability of some neuromuscular patients may worsen due to prolonged bed rest . Finally, the symptoms of certain diseases such as myasthenia gravis can worsen after an infection such as COVID-19.

Thanks to an unprecedented research effort, vaccines are now available and others still in development. The first studies published in medical journals are reassuring about the efficacy and safety of these vaccines. However, they have been studied in the general population and we do not yet have specific information in neuromuscular patients.

This is the reason why the Va-C-NEMUS observatory was launched.

Detailed description: Prevention of COVID-19 in neuromuscular patients is therefore fundamental and vaccination is eagerly awaited by patients and their healthcare teams. An unprecedented research effort has made it possible to create vaccines in a very short time when no vaccine directed against a coronavirus has existed until now. Some of these vaccines are based on proven technologies while others such as RNA vaccines are very innovative. The results of the phase III studies of 3 of them have just been published (Pfizer/BioNTech, Moderna and Astra-Zeneca). Among them, the Pfizer-BioNTech and Moderna vaccines have received authorization for use from the European Medicines Agency. The data on the individuals enrolled in these trials are still not very detailed. However, we already know that in the Pfizer/BioNTech trial, patients on immunosuppressants were excluded. For the Astra-Zeneca vaccine, the publication combines data from 4 different trials. In two of them the patients had to be in "good health". In any case, we do not know the pathologies presented by patients outside the usual risk factors for the severe form of COVID-19 (age, obesity, cardiovascular diseases, respiratory diseases). No data on neuromuscular patients are currently known. The vaccination campaign began in France in January 2021. The first neuromuscular patients should be vaccinated soon depending on their comorbidities, but many questions arise in these patients both in terms of safety and efficacy.

Eligibility criteria: Inclusion Criteria:

1. Patient with one of the following neuromuscular diseases:

   * Autoimmune myasthenia gravis, Lambert-Eaton syndrome
   * Congenital myasthenic syndromes
   * Myositis
   * Inflammatory neuropathies
   * Hereditary neuropathies (Charcot-Marie-Tooth disease, etc.)
   * Amyloid neuropathies
   * Spinal atrophies
   * Myotonic dystrophies type 1 (Steinert disease) and 2 (PROMM)
   * Duchenne and Becker muscular dystrophies
   * Muscular dystrophies of the girdles
   * Pump disease
   * Congenital myopathies
   * Congenital muscular dystrophies
   * Metabolic myopathies
   * Other myopathies with confirmed genetic diagnosis
2. Patient over 18 years old
3. Patient having understood that the follow-up of a possible side effect of the vaccination or of a COVID-19 must be carried out by his usual doctor (s) and not by the observatory team Va-C-NEMUS
4. Patients having been informed of the study and having expressed their agreement

Exclusion Criteria:

1. Patient under legal protection, curatorship or tutorship

Primary outcomes:
measure: the frequency of serious adverse reactions (SAEs)
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexpected adverse reactions (SUSARs)
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary outcomes:
measure: vaccination's global safety : Frequency of Adverse Effects (AEs)
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: Risk of worsening autoimmune myasthenia gravis after vaccination
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: Frequency of worsening of the autonomy of the patients evaluated with the MG-ADL
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: Frequency of hospitalizations for myasthenic crisis
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at least 7 days after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: effectiveness of the vaccination
timeFrame: at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of serious adverse reactions (SAEs) at 2 months
timeFrame: at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of serious adverse reactions (SAEs) at 3 months
timeFrame: at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of serious adverse reactions (SAEs) at 4 months
timeFrame: at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of serious adverse reactions (SAEs) at 5 months
timeFrame: at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of serious adverse reactions (SAEs) at 6 months
timeFrame: at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexpected adverse reactions (SUSARs) at 2 months
timeFrame: at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexpected adverse reactions (SUSARs) at 3 months
timeFrame: at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexpected adverse reactions (SUSARs) at 4 months
timeFrame: at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexepected adverse reactions (SUSARs) at 5 months
timeFrame: at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
measure: the frequency of unexpected adverse reactions (SUSARs) at 6 months
timeFrame: at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Other outcomes:
Locations:
city: Bordeaux
country: France
facility: CHU de Bordeaux
Study officials:
affiliation: Université Hospital, Bordeaux
name: Guilhem SOLE, MD
role: PRINCIPAL_INVESTIGATOR
References: