A MULTICENTER, SINGLE ARM, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF SATRALIZUMAB IN PATIENTS WITH NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD)

• Register: EUCTR
• Last refreshed on: 8 July 2024
• Main ID: EUCTR2020-003413-35-DE
• Date of registration: 27/10/2020
• Prospective Registration: Yes
• Primary sponsor: F. Hoffmann-La Roche Ltd
• Public title: A MULTICENTER, SINGLE ARM, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF SATRALIZUMAB IN PATIENTS WITH NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD)
• Scientific title: A MULTICENTER, SINGLE ARM, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF SATRALIZUMAB IN PATIENTS WITH NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD)
• Date of first enrolment: 03/02/2021
• Target sample size: 127
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-003413-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Bulgaria, Canada, Croatia, Georgia, Germany, Hungary, Italy, Japan, Korea, Republic of, Malaysia, Poland, Romania, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Trial Information Support Line-TISL
• Affiliation: F. Hoffman-La Roche Ltd

• Name: Trial Information Support Line-TISL
• Affiliation: F. Hoffman-La Roche Ltd

Key inclusion & exclusion criteria

Inclusion criteria:
Patients must meet the following criteria for study entry:
- Participated in Study BN40898 or Study BN40900 with satralizumab in NMOSD, are on ongoing satralizumab treatment and were AQP4-IgG seropositive at screening in these studies. Patients with NMOSD who were AQP4-IgG seronegative at screening in Study BN40898 or Study BN40900 can be enrolled if the investigator considers the continued treatment with satralizumab to be beneficial for the patient.
- Signed Informed Consent Form (ICF).
- Ability to comply with the study protocol
- For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use adequate contraception during the treatment period and for 3 months after the final dose of satralizumab.

A woman is considered to be of childbearing potential if she is postmenarchal, has not reached a postmenopausal state (=12 continuous months of amenorrhea with no identified cause other than menopause), and is not permanently infertile due to surgery (i.e., removal of ovaries, fallopian tubes, and/or uterus) or another cause as determined by the investigator (e.g., Müllerian agenesis). The definition of childbearing potential may be adapted for alignment with local guidelines or regulations.

The following are examples of adequate contraceptive methods: bilateral tubal ligation; male sterilization; hormonal contraceptives; hormone-releasing intrauterine devices; copper intrauterine devices; male or female condom with or without spermicide; and cap, diaphragm, or sponge with spermicide.

The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not adequate methods of contraception. If required per local guidelines or regulations, locally recognized adequate methods of contraception and information about the reliability of abstinence will be described in the local ICF.
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 116
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion criteria:
Patients who meet any of the following criteria will be excluded from study entry:
- Use of prohibited medication
- Pregnant or breastfeeding, or intending to become pregnant during the study or within 3 months after the final dose of study drug. Women of childbearing potential must have a negative urine pregnancy test result on the baseline visit prior to initiation of study drug.
- Evidence of any serious uncontrolled concomitant diseases that may preclude patient participation, such as:
other nervous system disease, cardiovascular disease, hematologic/hematopoiesis disease, respiratory disease, muscular disease, endocrine disease, renal/urologic disease, digestive system disease, congenital or acquired severe immunodeficiency.
- Known active infection that requires delaying the next satralizumab dose at the time of enrollment a a In case of an active infection, the patient should remain in the parent study, as governed by that protocol, and may enroll in this study once the active infection is controlled.
- NMOSD relapse at the time of enrollment .In case of a relapse, the patient should remain in the parent study, as governed by that protocol, and may enroll in this study once the patient is stable.
- Laboratory abnormalities at the last assessment in Study BN40898 or Study BN40900 that preclude re-treatment with satralizumab
If a patient does not meet the criteria to restart treatment with satralizumab based on laboratory assessments, the patient should remain in the parent study and the baseline visit should be delayed. The last assessment before enrollment must meet the re-treatment criteria.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Neuromyelitis Optic Spectrum Disorder
MedDRA version: 21.1 Level: PT Classification code 10077875 Term: Neuromyelitis optica spectrum disorder System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: Satralizumab
Product Code: RO5333787
Pharmaceutical Form: Injection
INN or Proposed INN: SATRALIZUMAB
CAS Number: 1535963-91-7
Current Sponsor code: RO5333787
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-

Primary Outcome(s)

Secondary Objective: To further evaluate the risks of serious infections and hepatotoxicity in patients with NMOSD who are treated with satralizumab

Timepoint(s) of evaluation of this end point: Not applicable

Primary end point(s): - Incidence and severity of adverse events (AE), adverse events of special interest (AESI), serious AEs (SAE), and selected AEs
- Vital signs (temperature, blood pressure, and pulse rate), clinical laboratory tests (hematology, chemistry, and urinalysis), 12-lead electrocardiograms (ECG), and suicidality (Columbia-Suicide Severity Rating Scale [C-SSRS])

Main Objective: Safety Objective

- To evaluate the long-term safety of satralizumab in patients with NMOSD.

Efficacy Objective
The efficacy objective for this study is to evaluate long-term efficacy of satralizumab on the basis of the following endpoints:
- Time to first relapse (TFR) and proportion of patients who are relapsefree
- Annualized relapse rate (ARR)
- Change in Expanded Disability Status Scale (EDSS) score
- Time to EDSS worsening and proportion of patients without EDSS worsening
-Change in visual acuity

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: The time to first relapse (TFR) efficacy endpoint is based on several endpoint definitions to accommodate different estimand strategies. The most important intercurrent event is the use of therapy for the treatment of an acute NMOSD relapse (rescue therapy). For the treatment policy strategy, the use of rescue therapy is ignored. Therefore, in the analysis for the treatment policy estimand the TFR is defined as the time from randomization in ITT or first dose of satralizumab in ALLSA to the first protocol-defined relapse as assessed by the investigator (iPDR).

Secondary end point(s): All efficacy analyses will be based on the ITT population and the ALLSA population. For both populations, data from Study WN42349 will be combined with data from the Studies BN40898 and BN40900.
The definitions are in line with Studies BN408989 and BN40900 to investigate long-term efficacy by summarizing the data of the parental studies and this Study WN42349.

Secondary ID(s)

2020-003413-35-GB

WN42349

Source(s) of Monetary Support

F. Hoffmann-La Roche Ltd

Ethics review

Status: Approved
Approval date: 03/02/2021
Contact: