Tys at Home: Comparing the Safety and Benefit of Natalizumab (Tysabri®) At-home Infusion vs At-hospital Infusion in Multiple Sclerosis

Sponsor

Nantes University Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT04777539

Collaborator

Rennes University Hospital (Other)

315

Enrollment

Locations

35.5

Anticipated Duration (Months)

15.8

Patients Per Site

0.4

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Multiple Sclerosis (MS)	Other: At-home natalizumab treated MS patient

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.

Study Design

Study Type:

Observational

Anticipated Enrollment :

315 participants

Observational Model:

Other

Time Perspective:

Prospective

Official Title:

Assessing the Safety and Benefit of Home-hospitalization Program in the Management of Natalizumab (Tysabri®) Medication in Multiple Sclerosis

Actual Study Start Date :

Jun 17, 2021

Anticipated Primary Completion Date :

Mar 1, 2024

Anticipated Study Completion Date :

Jun 1, 2024

Outcome Measures

Primary Outcome Measures

Number of serious Adverse events (SAE) and selected adverse events (AEs) of grade 2 [One year after enrollment]
Grades are based on CTCAE V5. Selected AEs of grade 2: myocarditis, pericarditis, diarrhea, enterocolitis, esophagitis, mucositis, fever/hypothermia folicullitis, papular and/or pustular rash, herpes zoster.

Secondary Outcome Measures

Annualized Relapse Rate [Two time points per patient one year prior to and one year after enrollment]
Annualized Relapse Rate
Change from baseline in T2 lesion load as assessed by MRI [12-month (+/-6 month) prior enrollment, Baseline (enrollment+/-3 months), 12 months after enrollment]
Modification is based on percentage of patient with at least one new T2 lesion
Change from baseline in gadolinium positive lesion as assessed by MRI [Baseline and 12 month before and 12 month after enrollment]
Modification is based percentage of patient with at least one Gd-enhancing lesion
Multiple Sclerosis International quality of Life (MusiQoL) [Baseline and months 6 and 12]
MusiQoL is a self-administered disease-specific quality of life instrument that comprises 31 items encompassing 9 dimensions: activities of daily living (8), psychological well-being (4), symptoms (3), relationships with friend (4), relationships with family (3), relationship with healthcare system (3), sentimental and sexual life (2), coping (2) and rejection (2). Each item is answered using a 6-point Likert scale (Never/Not at all, Rarely/A little, Sometimes/Somewhat, Often/A lot, Always/Very much and Not applicable)
EuroQol-5Dimension 5Level (EQ-5D-5L) [Baseline and months 6 and 12]
The EQ-5D-5L is a self-administered instrument consisting in 2 components that describe patient's health state: A five dimensional questionnaire describing health in terms of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Patient is asked to indicate for each one the level of functioning: no problems, slight problems, moderate problems, severe problems and extreme problems. A visual analogue scale (VAS) that measures health state (from 0 to 100).
Musicare [Baseline and months 6 and 12]
Musicare is a self-administered disease-specific instrument investigating experience of the quality of care for MS. It comprises 35 items encompassing 5 domains: Information about the disease (11), Information about the treatments/medical investigation (8), Relationships with health care teams (8) Health care access (5) Reception in care structures (3).
Patient experience [One point per patient between months 3 and 8]
Semi-structured interview based on " patient tracer " model will be conducted in a sub-group of 15 patients.
Clinical Global impressions of improvement (CGI-I) [Months 6 and 12]
The CGI is a generic 3-component scale used over the years in a broad range of diseases. The CGI-I (Improvement) is one of the components (one item) measuring change in health state since the entry in the study and can be rated independently. CGI-I is rated on a 7-point scale: from 1 (very much improved) to 7 (very much worse).
The average cost per patient [During study, it will be evaluated the average cost per patient for one year of treatment (comparison HDJ vs HAD)]
Compare the cost of the "At home" and "Hospital" management strategies in a before-after design from collective perspective

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Inclusion Criteria :

Male or female over 18 years old;
Patients with Relapsing-remitting MS followed in the neurology service
Treated for more than 24 months with natalizumab;
Anti-JCV negative status at inclusion;
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.
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)
Had a cerebral MRI within the previous 12 months (+/- 6 months) which results are available in the medical record.

Exclusion Criteria :

Patient having expressed their opposition to the use of their data;
Women who are pregnant or breastfeeding or intending to become pregnant during the study;
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.
Patient under guardianship or under security measure

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Strasbourg University Hospital	Strasbourg	Bas-Rhin	France	67000
2	Marseille University Hospital	Marseille	Bouches-du-Rhône	France	13000
3	Brest University Hospital	Brest	Finistère	France	29200
4	Quimper Hospital	Quimper	Finistère	France	29000
5	Bordeaux University Hospital	Bordeaux	Gironde	France	33000
6	Libourne Hospital	Libourne	Gironde	France	33500
7	Toulouse University Hospital	Toulouse	Haute-Garonne	France	31000
8	Limoges University Hospital	Limoges	Haute-Vienne	France	87000
9	Percy Army Training Hospital	Clamart	Hauts-de-Seine	France	92140
10	Montpellier University Hospital	Montpellier	Hérault	France	34000
11	Rennes University Hospital	Rennes	Ille-et-Vilaine	France	35000
12	Tours University Hospital	Tours	Indre-et-Loire	France	37000
13	Dax Hospital	Dax	Landes	France	40100
14	Nantes University Hospital	Nantes	Loire-Atlantique	France	44000
15	Orléans Hospital	Orléans	Loiret	France	45000
16	Lille University Hospital	Lille	Nord	France	59000
17	Rouen University Hospital	Rouen	Seine-Maritime	France	76000
18	Poitiers University Hospital	Poitiers	Vienne	France	86000
19	Adolphe de Rothschild Ophthalmological Foundation	Paris		France	75000
20	AP-HP La Pitié-Salpêtrière Hospital	Paris		France	75000