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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04875975




Registration number
NCT04875975
Ethics application status
Date submitted
3/05/2021
Date registered
6/05/2021

Titles & IDs
Public title
A Study to Test the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Rozanolixizumab in Adult Study Participants With Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis
Secondary ID [1] 0 0
2019-004778-25
Secondary ID [2] 0 0
AIE001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Leucine-Rich Glioma Inactivated 1 Autoimmune Encephalitis 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Neurological 0 0 0 0
Epilepsy
Metabolic and Endocrine 0 0 0 0
Thyroid disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Rozanolixizumab
Treatment: Drugs - Placebo

Experimental: Rozanolixizumab - Participants will be randomized to receive a predefined dose of rozanolixizumab.

Placebo comparator: Placebo - Participants will be randomized to receive a dose of placebo.


Treatment: Drugs: Rozanolixizumab
* Pharmaceutical form: Solution for infusion
* Route of administration: Subcutaneous use

Subjects will receive rozanolixizumab in a pre-specified sequence during the Treatment Period.

Treatment: Drugs: Placebo
* Pharmaceutical form: Solution for infusion
* Route of administration: Subcutaneous use

Subjects will receive placebo in a pre-specified sequence during the Treatment Period.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of seizure free study participants at the end of the Treatment Period
Timepoint [1] 0 0
From Baseline until the end of the Treatment Period (Week 25)
Secondary outcome [1] 0 0
Change from Baseline in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score at the end of the Treatment Period
Timepoint [1] 0 0
From Baseline until the end of the Treatment Period (Week 25)
Secondary outcome [2] 0 0
Proportion of participants with a favorable outcome in the Modified Rankin Scale (mRS) during the Treatment Period
Timepoint [2] 0 0
From Baseline until the end of the Treatment Period (Week 25)
Secondary outcome [3] 0 0
Proportion of participants who required rescue medication due to an absence or loss of clinical benefit during the Treatment Period
Timepoint [3] 0 0
From Baseline until the end of the Treatment Period (Week 25)
Secondary outcome [4] 0 0
Time to first occurrence of seizure freedom during the Treatment Period
Timepoint [4] 0 0
From Baseline until the end of the Treatment Period (Week 25)
Secondary outcome [5] 0 0
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Timepoint [5] 0 0
From Baseline until the End of Study Visit (Week 32)

Eligibility
Key inclusion criteria
* Study participant must be =18 to =89 years of age
* Study participant must be seropositive for leucine-rich glioma inactivated 1 (LGI1) antibody
* Study participant must have =2 seizures/week during the Screening Period or have experienced such seizures that stopped following high dose corticosteroids (500 to 1000 milligram (mg) methylprednisolone (MP) equivalent/day):

* Either faciobrachial dystonic seizures (FBDS) with or without other focal (partial) seizures including focal to bilateral tonic clonic
* Or focal (partial) seizures including focal to bilateral tonic clonic and fulfil the following new-onset Autoimmune encephalitis (AIE) criteria
* Study participant has initiated or re-initiated corticosteroids at a dose of 500 to 1000 mg MP equivalent/day within 42 days prior to randomization. Participants re-initiating corticosteroids are eligible only if re-initiation is due to seizure rebound and within the timeframe outlined. If the study participant has initiated a steroid taper, the study participant cannot receive an oral steroid dose lower than 40mg/day when randomized
* Study participant with onset of disease symptom between 0 to 12 months prior to Screening, per investigator's assessment.
* Study participant weighs at least 35 kg at Screening
* A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:

i) Not a woman of childbearing potential (WOCBP) OR ii) A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 90 days after the final dose of study treatment
Minimum age
18 Years
Maximum age
89 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications.
* Study participant has a confirmed prior diagnosis of epilepsy or new onset seizures that are unrelated to LGI1 autoimmune encephalitis (AIE) or has any known or suspected medical cause for the onset of seizures other than possible AIE
* Study participant has a known active neoplastic disease or history of neoplastic disease within 5 years of study entry
* Study participant has renal impairment, defined as glomerular filtration rate (GFR) <30mL/min/1.73m2 at the Screening Visit
* Study participant has a clinically important active infection (including unresolved or not adequately treated infection) as assessed by investigator, including participants with a serious infection within 6 weeks prior to the first dose of investigational medicinal product (IMP)
* Study participant has a history of chronic ongoing infections
* Study participant has current unstable liver or biliary disease, per investigator assessment, defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis
* Study participant has positive tuberculosis (TB) test at the Screening Visit
* Study participant has a history of solid organ transplant or hematopoietic stem cell transplant
* Study participant has undergone a splenectomy
* Study participant has a current or medical history of primary immune deficiency
* Study participant has received a live vaccination within 4 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of investigational medicinal product (IMP)
* Study participant has previously received rozanolixizumab drug product
* Alanine transaminase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP) are >3x upper limit of normal (ULN)
* Study participant has a total IgG level =5.5 g/L at the Screening Visit
* Study participant has absolute neutrophil count <1500 cells/mm^3 at the Screening Visit

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/09/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/04/2024
Sample size
Target
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Aie001 30027 - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Colorado
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Belgium
State/province [9] 0 0
Bruxelles
Country [10] 0 0
France
State/province [10] 0 0
Bordeaux
Country [11] 0 0
France
State/province [11] 0 0
Bron
Country [12] 0 0
France
State/province [12] 0 0
Lille
Country [13] 0 0
France
State/province [13] 0 0
Nancy
Country [14] 0 0
France
State/province [14] 0 0
Nice
Country [15] 0 0
France
State/province [15] 0 0
Paris
Country [16] 0 0
France
State/province [16] 0 0
Toulouse Cedex
Country [17] 0 0
Germany
State/province [17] 0 0
Berlin
Country [18] 0 0
Germany
State/province [18] 0 0
Kiel
Country [19] 0 0
Italy
State/province [19] 0 0
Pavia
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
Netherlands
State/province [21] 0 0
Rotterdam
Country [22] 0 0
Spain
State/province [22] 0 0
Barcelona
Country [23] 0 0
Spain
State/province [23] 0 0
Málaga
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Nottingham
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
UCB Biopharma SRL
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
UCB Cares
Address 0 0
001 844 599 2273 (UCB)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
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.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
When will data be available (start and end dates)?
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.
Available to whom?
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, 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 pre-specified time, typically 12 months, on a password protected portal.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.Vivli.org


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04875975