A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients with Aquaporin-4 (AQP4) Antibody Positive Neuromyelitis Optica Spectrum Disorder

Overview

Sponsor-declared trial summary

Key facts

Sponsor

F. Hoffmann-La Roche AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

21 Apr 2022 → ongoing

Decision date (initial)

2024-07-16

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

F. Hoffmann-La Roche AG

External identifiers

EU CT number

2023-507817-85-00

EudraCT number

2019-004092-39

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Pharmacodynamic, Safety, Pharmacokinetic

1. To investigate the pharmacokinetics (PK) of satralizumab by evaluating serum exposure over 24 weeks in participants aged 2-11 years, inclusive

Secondary objectives 3

1. 1. To evaluate the efficacy of subcutaneously administered satralizumab for 48 weeks in pediatric patients (three weight-based cohorts).
2. 2. To evaluate the safety of subcutaneously administered satralizumab for 48 weeks in pediatric patients (three weight-based cohorts)
3. 3. To evaluate the immune response to satralizumab

Conditions and MedDRA coding

Neuromyelitis Optica Spectrum Disorder (NMOSD)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 1. Age at screening 2-11 years, inclusive
2. 2. Body weight at screening ≥10 kg
3. 3. For female participants of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception
4. 4. Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria
5. 5. Neurological stability for ≥30 days prior to both screening and baseline
6. 6. Normal or abnormal neurologic status as described by EDSS (0 to 6.5)

Exclusion criteria 6

1. 1. Pregnancy or lactation
2. 2. Evidence of other demyelinating disease mimicking NMOSD including but not limited to MS, myelin oligodendrocyte glycoprotein-IgG associated disease, or progressive multifocal leukoencephalopathy
3. 3. Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection (excluding fungal infection of nail beds or dental caries) at baseline
4. 4. Evidence of chronic active hepatitis B or C
5. 5. Evidence of untreated latent or active tuberculosis (TB)
6. 6. Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. 1. Summary of observed serum concentration of satralizumab at specified trough timepoints (mean and standard deviation of trough concentration [Ctrough] at each sampling timepoint)
2. 2. Population and individual estimates of PK parameters using a population-PK modeling approach

Secondary endpoints 14

1. 1. Proportion of relapse-free participants by Week 48
2. 2. Annualized relapse rate (ARR)
3. 3. Time to first relapse (TFR)
4. 4. Time to relapse requiring rescue therapy
5. 5. Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48
6. 6. Change from baseline in visual acuity (evaluated using standardized logMAR visual acuity charts, such as Sloan charts, LEA Symbols® chart, and HOTV chart) at Weeks 24 and 48
7. 7. Change from baseline in FACES® Pain Rating Scale at Weeks 24 and 48
8. 8. Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy (for participants younger than 8 years of age) at Weeks 24 and 48
9. 9. Incidence and severity of adverse events, adverse events of special interest, serious adverse events
10. 10. Change from baseline in targeted vital signs
11. 11. Change from baseline in weight and height
12. 12. Change from baseline in targeted clinical laboratory test results
13. 13. Change from baseline in targeted ECG parameters
14. 14. Incidence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study

Investigational products

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

Test 2

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 System - TISL

Public contact point

Organisation

F. Hoffmann-La Roche AG

Contact name

Trial Information System - TISL

Third parties 5

Locations

3 EU/EEA countries · 4 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 11 files

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

Application history

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