Efficacy and Safety of Vaccination Against COVID-19 in Neuromuscular Patients

Neuromuscular Diseases Clinical Trial

— VA-C-NEMUS  

Official title:

Observatory of the Efficacy and Safety of Vaccination Against SARS-CoV-2 in Neuromuscular Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05311904
• Other study ID #: CHUBX 2020/68
• Status: Completed
• Start date: March 11, 2021
• Est. completion date: February 3, 2023

Study information

• Verified date: October 2023
• Source: University Hospital, Bordeaux
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1388
• Est. completion date: February 3, 2023
• Est. primary completion date: February 3, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 100 Years

Eligibility

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

Related Conditions & MeSH terms

• Neuromuscular Diseases

Intervention

Other:
• Survey
1 initial questionnaire and 1 monthly follow-up questionnaire for 11 months

Locations

Country	Name	City	State
France	CHU de Bordeaux	Bordeaux

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Bordeaux

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	the frequency of serious adverse reactions (SAEs)	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Primary	the frequency of unexpected adverse reactions (SUSARs)	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	vaccination's global safety : Frequency of Adverse Effects (AEs)	Frequency of Adverse Effects (AEs) in the population of neuromuscular patients vaccinated against COVID19	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	Risk of worsening autoimmune myasthenia gravis after vaccination	Myasthenia Gravis Foundation of America classification	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	Frequency of worsening of the autonomy of the patients evaluated with the MG-ADL	Frequency of worsening of the MG-ADL activity score by more than 2 points between the week before vaccination and the week following vaccination will be evaluated The Myasthenia Gravis-specific Activities of Daily Living scale consists of the assessment of 8 parameters: speaking, chewing, swallowing, breathing, self-care activities (brushing the teeth or combing the hair), simple physical activities (getting up from a chair), double vision and eye lid dropping. Each parameter is subjected to assessment depending on the degree of symptoms intensification, awarding points from 0 to 3 points. The maximum number a patient may receive is 24 points. The higher the score of points, the bigger limitations of the patient in everyday life activities caused by intensification of myasthenia gravis (Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.)	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	Frequency of hospitalizations for myasthenic crisis	Frequency of hospitalizations for myasthenic crisis in the month following vaccination	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test	at least 7 days after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 1 month after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	effectiveness of the vaccination	Frequency of COVID-19 cases confirmed by a PCR test or an antigen test occurring	at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of serious adverse reactions (SAEs) at 2 months	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of serious adverse reactions (SAEs) at 3 months	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of serious adverse reactions (SAEs) at 4 months	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of serious adverse reactions (SAEs) at 5 months	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of serious adverse reactions (SAEs) at 6 months	the frequency of serious adverse reactions (SAEs) in the population of neuromuscular patients vaccinated against COVID19 Serious adverse reaction or event (Article R1123-46 of the Public Health Code and ICH-E2B guideline); Any adverse reaction or event that: results in death,; endangers the life of the participant,; requires hospitalisation or prolongation of hospitalisation,; causes an inability or significant or long-term disability,; results in an abnormality or congenital malformation,; or any event considered to be medically serious, and with respect to the drug, regardless of the dose administered. The expression "life-threatening" is reserved for an immediate life threat at the time of the adverse event.	at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of unexpected adverse reactions (SUSARs) at 2 months	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 2 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of unexpected adverse reactions (SUSARs) at 3 months	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 3 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of unexpected adverse reactions (SUSARs) at 4 months	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 4 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of unexepected adverse reactions (SUSARs) at 5 months	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 5 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).
Secondary	the frequency of unexpected adverse reactions (SUSARs) at 6 months	Unexpected adverse event or suspected adverse reaction refers to an event or reaction that is not listed in the investigator's brochure or is not listed at the specificity or severity that has been observed; or, if an investigator's brochure is not required or available, is not consistent with the risk information	at 6 months after the end of the vaccination against SARS-CoV-2 (after the 2nd injection for the majority of vaccines).