Clinical trial NCT04777539: Comparing the Safety and Benefit of Natalizumab (Tysabri®) At-home Infusion vs At-hospital Infusion in Multiple Sclerosis
Official title: Assessing the Safety and Benefit of Home-hospitalization Program in the Management of Natalizumab (Tysabri®) Medication in Multiple Sclerosis
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Multiple Sclerosis (MS)
Keywords: At-home hospitalization (HAD)
Natalizumab
Safety
Medico-economic data
Multiple sclerosis
Lead sponsor: Nantes University Hospital
Sponsor class: OTHER
Collaborators: name: Rennes University Hospital
First posted: 2021-03-02
Enrollment: 279
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with relapsing-remitting MS and treated with natalizumab at hospital will be assessed for eligibility.

Brief summary: At-home use of Natalizumab in multiple sclerosis (MS) patients has been temporarily granted by French security agency of medicines and Health products (ANSM). The main objective of the study is to compare the safety of natalizumab administration at home vs at hospital based on retrospective and prospective data collection. Quality of life, patient perception of at-home natalizumab administration are also evaluated as secondary objectives as well as medico-economic assessment of the method.

Data will be collected for a 12-month retrospective period and a 12-month prospective period.

Detailed description: As part of the Covid-19 pandemic, some neurologists have alerted the French authorities (ANSM: National Medicines Safety Agency) to the interruption of natalizumab treatment by some MS patients. The ANSM has granted temporary home use of natalizumab within the framework of a "at-home hospitalization" (HAD) program. The study purpose is to assess at-home natalizumab administration regarding safety, disease activity, quality of life, patient's perception and costs by comparing outcomes before and after "At Home" natalizumab treatment strategy.

The study consists of two periods of observation: a retrospective one corresponding to the year prior the study entry and a prospective one corresponding to patient follow-up for one year from the date of the patient inclusion. The date of the inclusion corresponds to the natalizumab-infusion conducted at hospital just before infusions hospital at-home started. Retrospective data are collected from medical record and a minimal dataset is needed to enroll the patient regarding MRI activity and safety data (i.e., nature and number of SAE and selected AE of grade 2 the year prior study entry).

The prospective period consists of 10 at-home natalizumab infusions (months 1-5 and 7-11) and 3 at-hospital natalizumab infusions (inclusion, months 6 and 12) according to standard care. In addition to clinical, MRI and safety (SAE and selected AE of grade 2) data collected as part of standard medical care from medical record over all the study period, Quality of life (EQ-5D-5L, MusiQol) and patient's preference (Musicare) questionnaires will be administered at inclusion and months 6 and 12 and clinical global impression of change will be recorded at month 6 and 12. Patients will be asked to notify any adverse events that may occur between each at-home infusion and to record each month his/her healthcare resource consumption in a diary. Patients will be contacted each month after at-home infusion to record safety and healthcare resource consumption data.

For a sub-group of 15 patients qualitative interview will be performed between month 3 and 8 as part of patient preference assessment.

Micro-costing dedicated forms and experts' statements will be used to assess resource utilization associated to natalizumab infusion during each period for medico-analyze purpose.

Eligibility criteria: Inclusion Criteria :

1. Male or female over 18 years old;
2. Patients with Relapsing-remitting MS followed in the neurology service
3. Treated for more than 24 months with natalizumab;
4. Anti-JCV negative status at inclusion;
5. Ability to understand the purpose of the study and provide opposition to use protected health information in accordance with national and local subject privacy regulations.
6. Had available medical records to meet study objectives (i.e., SAE and selected AE of grade 2 from the past 12 infusions of natalizumab performed at the hospital before inclusion)
7. Had a cerebral MRI within the previous 12 months (+/- 6 months) which results are available in the medical record.

Exclusion Criteria :

1. Patient having expressed their opposition to the use of their data;
2. Women who are pregnant or breastfeeding or intending to become pregnant during the study;
3. History of any clinically significant (as determined by the Investigator) cardiac, endocrinologic, hematologic, hepatic, immunologic, metabolic (including diabetes), urologic, pulmonary, neurologic (except for RRMS), dermatologic, psychiatric, renal, or other major disease that would preclude participation in a clinical study, in the opinion of the Investigator.
4. Patient under guardianship or under security measure

Primary outcomes:
measure: Number of serious Adverse events (SAE) and selected adverse events (AEs) of grade 2
timeFrame: One year after enrollment
Secondary outcomes:
measure: Annualized Relapse Rate
timeFrame: Two time points per patient one year prior to and one year after enrollment
measure: Change from baseline in T2 lesion load as assessed by MRI
timeFrame: 12-month (+/-6 month) prior enrollment, Baseline (enrollment+/-3 months), 12 months after enrollment
measure: Change from baseline in gadolinium positive lesion as assessed by MRI
timeFrame: Baseline and 12 month before and 12 month after enrollment
measure: Multiple Sclerosis International quality of Life (MusiQoL)
timeFrame: Baseline and months 6 and 12
measure: EuroQol-5Dimension 5Level (EQ-5D-5L)
timeFrame: Baseline and months 6 and 12
measure: Musicare
timeFrame: Baseline and months 6 and 12
measure: Patient experience
timeFrame: One point per patient between months 3 and 8
measure: Clinical Global impressions of improvement (CGI-I)
timeFrame: Months 6 and 12
measure: The average cost per patient
timeFrame: During study, it will be evaluated the average cost per patient for one year of treatment (comparison HDJ vs HAD)
Other outcomes:
Locations:
city: Strasbourg
country: France
facility: Strasbourg University Hospital
state: Bas-Rhin
city: Marseille
country: France
facility: Marseille University Hospital
state: Bouches-du-Rhône
city: Brest
country: France
facility: Brest University Hospital
state: Finistère
city: Quimper
country: France
facility: Quimper Hospital
state: Finistère
city: Bordeaux
country: France
facility: Bordeaux University Hospital
state: Gironde
city: Libourne
country: France
facility: Libourne Hospital
state: Gironde
city: Toulouse
country: France
facility: Toulouse University Hospital
state: Haute-Garonne
city: Limoges
country: France
facility: Limoges University Hospital
state: Haute-Vienne
city: Clamart
country: France
facility: Percy Army Training Hospital
state: Hauts-de-Seine
city: Montpellier
country: France
facility: Montpellier University Hospital
state: Hérault
city: Rennes
country: France
facility: Rennes University Hospital
state: Ille-et-Vilaine
city: Tours
country: France
facility: Tours University Hospital
state: Indre-et-Loire
city: Dax
country: France
facility: Dax Hospital
state: Landes
city: Nantes
country: France
facility: Nantes University Hospital
state: Loire-Atlantique
city: Orléans
country: France
facility: Orléans Hospital
state: Loiret
city: Lille
country: France
facility: Lille University Hospital
state: Nord
city: Rouen
country: France
facility: Rouen University Hospital
state: Seine-Maritime
city: Poitiers
country: France
facility: Poitiers University Hospital
state: Vienne
city: Ajaccio
country: France
facility: CH Ajaccio
city: Chartres
country: France
facility: Les Hôpitaux de Chartres
city: La Tronche
country: France
facility: CHU Grenoble Alpes
city: Lille
country: France
facility: Hôpital St Vincent de Paul
city: Paris
country: France
facility: Adolphe de Rothschild Ophthalmological Foundation
city: Paris
country: France
facility: AP-HP La Pitié-Salpêtrière Hospital
Study officials:
References: