A study to learn if Rozanolixizumab works and is safe over a long period in adults with relapsing myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

2 Aug 2022 → ongoing

Decision date (initial)

2024-08-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

External identifiers

EU CT number

2023-509237-39-00

EudraCT number

2021-000352-19

WHO UTN

U1111-1301-0122

ClinicalTrials.gov

NCT05063162

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacogenetic, Efficacy, Pharmacogenomic, Therapy, Pharmacokinetic, Pharmacodynamic, Safety

Evaluate the efficacy of rozanolixizumab for treatment of participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD)

Secondary objectives 1

1. Assess the safety and tolerability of rozanolixizumab in participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD)

Conditions and MedDRA coding

Myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002681-PIP03-21

Plan to share IPD

Yes

IPD plan description

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Participants must be ≥18 to ≤89 years of age, at the time of signing the informed consent
2. Confirmed diagnosis of MOG-AD consistent with published diagnostic criteria for MOG-AD
3. Participant has history of relapsing MOG-AD with at least 1 documented relapse over the last 12 months and a documented positive serum MOG Ab test using a cellbased assay (CBA) within 6 months prior to randomization
4. Participant must be clinically stable at the time of the Screening Visit and during the Screening Period

Exclusion criteria 5

1. Participant has been diagnosed with a neurological autoimmune disease (including multiple sclerosis (MS) and aquaporin-4 positive neuromyelitis optica spectrum disorder (NMOSD)), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant
2. Participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by the investigator, including participants with a serious infection within 6 weeks prior to the first dose of the investigational medicinal product (IMP)
3. Participant has a current or medical history of primary immunodeficiency
4. Participant tests positive for aquaporin-4 antibodies at screening
5. Participant has a serum total IgG level ≤5.5g/L

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. For Part A: 1.Time from randomization to first independently centrally adjudicated relapse (TTFR) during the Double Blind (DB) Treatment Period
2. For Part B: 2. Incidence of treatment-emergent adverse events (TEAEs) during Open-Label Extension (OLE) Treatment Period 3. Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period

Secondary endpoints 5

1. For Part A 1. Change from Baseline in Low-Contrast Monocular Visual Acuity (Worst Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the End of Double-Blind/Early Withdrawal (EDB/EWD) Visit
2. Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the end of the EDB/EWD Visit (with confirmation at 3 months)
3. Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period
4. Incidence of treatment-emergent adverse events (TEAEs) during DB Treatment Period
5. For Part B 5. Independently centrally adjudicated annualized relapse rate (ARR) during the DB and OLE Treatment Period

Investigational products

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

Test 1

Placebo 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UCB Biopharma

Sponsor organisation

UCB Biopharma

Address

Researchdreef 60

City

Anderlecht

Postcode

1070

Country

Belgium

Scientific contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Public contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Third parties 19

Locations

10 EU/EEA countries · 34 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 159 files

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

Application history

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