A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Satralizumab in Patients with Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

F. Hoffmann-La Roche AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

20 Jun 2022 → ongoing

Decision date (initial)

2024-03-08

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

F. Hoffmann-La Roche AG

External identifiers

EU CT number

2023-507196-22-00

EudraCT number

2021-003192-34

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

1. To evaluate the efficacy of satralizumab compared with placebo based on time from randomization to the first occurrence of an adjudicated MOGAD relapse in the double-blind (DB) treatment period

Secondary objectives 2

1. 1. To evaluate the efficacy of satralizumab compared with placebo based on rate of adjudicated MOGAD relapses, presence of active lesions on magnetic resonance imaging (MRI) of the neuroaxis, rescue therapy use, inpatient hospitalizations, and proportion of relapse-free participants at 6-month intervals
2. 2. To evaluate the safety of satralizumab compared with placebo

Conditions and MedDRA coding

Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-001625-PIP03-21

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 1. Participants who are aged >=12 years at the time of signing Informed Consent Form and confirmed diagnosis of MOGAD with a history of >=1 MOGAD relapse in the 12 months prior to screening or >=2 attacks in the 24 months prior to screening
2. 2. Expanded Disability Status Scale (EDSS) score of 0-6.5 at screening
3. 3. High-contrast visual acuity (HCVA) better than 20/800 in each eye at screening
4. 4. Participants receiving either no ongoing chronic immunosuppressant treatment (IST) for MOGAD at the time of screening or receiving ongoing treatment with azathioprine (AZA), mycophenolate mofetil (MMF), oral corticosteroids (OCS) or a combination of OCS and AZA or MMF prior to and at the time of screening
5. 5. No contraindications to rescue treatments and no contraindications to MRI
6. 6. For women of childbearing potential: participants who agree to remain abstinent or use adequate contraception during the treatment period and for at least 3 months after the final dose of satralizumab

Exclusion criteria 6

1. 1. Presence of aquaporin-4-antibodies (AQP4-IgG) in the serum
2. 2. History of anti-N-methyl-d-aspartate receptor (NMDAR) encephalitis
3. 3. Any concomitant disease other than MOGAD that may require treatment with ISTs or OCS or intravenous (IV) corticosteroids at doses >20 mg prednisone equivalent per day for >21 days during the study
4. 4. Intravenous immunoglobulins (IVIg) or subcutaneous immunoglobulins ( ScIg) within 4 weeks prior to screening
5. 5. Plasma exchange (PLEX) within 4 weeks prior to screening
6. 6. Systemic corticosteroids, AZA or MMF within 4 weeks prior to screening (if not continued as concomitant IST in the study)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 1. Time from randomization to the first occurrence of a MOGAD relapse in the DB treatment period, as determined by an adjudication committee (CEC)

Secondary endpoints 12

1. 1. Rate of adjudicated MOGAD relapses
2. 2. Active lesions on MRI of the neuroaxis (Gd-enhancing or new/enlarging T2 hyperintense lesions measured across the optic nerve, the spinal cord and the brain, including brainstem and cerebellum)
3. 3. Proportion of participants receiving rescue therapy
4. 4. Rate of inpatient hospitalizations (defined as more than an overnight stay, excluding those for elective procedures)
5. 5. Proportion of relapse-free participants at 6-month intervals
6. 6. Change from baseline in total Montreal cognitive assessment (MoCA) score (adolescents only)
7. 7. Incidence, seriousness and severity of adverse events, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)
8. 8. Change from baseline in targeted vital signs
9. 9. Change from baseline in targeted electrocardiogram (ECG) parameters
10. 10. Change from baseline in targeted clinical laboratory test results
11. 11. Change from baseline in suicidality, as determined by Columbia-Suicide Severity Rating Scale
12. 12. Change from baseline in weight

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

F. Hoffmann-La Roche AG

Sponsor organisation

F. Hoffmann-La Roche AG

Address

Grenzacherstrasse 124

City

Basel

Postcode

4058

Country

Switzerland

Scientific contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information Support Line - TISL

Public contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information Support Line - TISL

Third parties 4

Locations

4 EU/EEA countries · 24 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 68 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

9 submissions — initial application plus substantial / non-substantial modifications