SATRALIZUMAB for Neuromyelitis optica spectrum disorder (AQP4 antibody-positive)

Inclusion criteria

• {"criterion_text":"- 1. Age at screening 2-11 years, inclusive"}
• {"criterion_text":"- 2. Body weight at screening ≥10 kg"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 4. Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria"}
• {"criterion_text":"- 5. Neurological stability for ≥30 days prior to both screening and baseline"}
• {"criterion_text":"- 6. Normal or abnormal neurologic status as described by EDSS (0 to 6.5)"}

Exclusion criteria

• {"criterion_text":"- 1. Pregnancy or lactation"}
• {"criterion_text":"- 2. Evidence of other demyelinating disease mimicking NMOSD including but not limited to MS, myelin oligodendrocyte glycoprotein-IgG associated disease, or progressive multifocal leukoencephalopathy"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 4. Evidence of chronic active hepatitis B or C"}
• {"criterion_text":"- 5. Evidence of untreated latent or active tuberculosis (TB)"}
• {"criterion_text":"- 6. Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline"}

Primary endpoints

• {"endpoint_text":"- 1. Summary of observed serum concentration of satralizumab at specified trough timepoints (mean and standard deviation of trough concentration [Ctrough] at each sampling timepoint)","definition_or_measurement_approach":"Observed serum concentration of satralizumab measured at specified trough timepoints; summarized as mean and standard deviation of trough concentration (Ctrough) at each sampling timepoint."}
• {"endpoint_text":"- 2. Population and individual estimates of PK parameters using a population-PK modeling approach","definition_or_measurement_approach":"Population and individual pharmacokinetic parameter estimates derived using a population-PK modelling approach."}

Secondary endpoints

• {"endpoint_text":"- 1. Proportion of relapse-free participants by Week 48","definition_or_measurement_approach":"Proportion of participants without clinical relapse assessed through Week 48."}
• {"endpoint_text":"- 2. Annualized relapse rate (ARR)","definition_or_measurement_approach":"ARR calculated from observed relapses over the follow-up period, annualized."}
• {"endpoint_text":"- 3. Time to first relapse (TFR)","definition_or_measurement_approach":"Time from baseline to first documented clinical relapse."}
• {"endpoint_text":"- 4. Time to relapse requiring rescue therapy","definition_or_measurement_approach":"Time from baseline to relapse event that necessitates rescue therapy."}
• {"endpoint_text":"- 5. Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48","definition_or_measurement_approach":"Change in EDSS scores from baseline measured at Weeks 24 and 48."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Change in visual acuity from baseline measured at Weeks 24 and 48 using standardized logMAR charts (e.g., Sloan, LEA Symbols®, HOTV)."}
• {"endpoint_text":"- 7. Change from baseline in FACES® Pain Rating Scale at Weeks 24 and 48","definition_or_measurement_approach":"Change from baseline on the FACES® Pain Rating Scale at Weeks 24 and 48."}
• {"endpoint_text":"- 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","definition_or_measurement_approach":"Change from baseline in EQ-5D-Y score and proxy-completed EQ-5D-Y for participants <8 years, assessed at Weeks 24 and 48."}
• {"endpoint_text":"- 9. Incidence and severity of adverse events, adverse events of special interest, serious adverse events","definition_or_measurement_approach":"Recording and classification of adverse events, AEs of special interest, and SAEs with incidence and severity assessments."}
• {"endpoint_text":"- 10. Change from baseline in targeted vital signs","definition_or_measurement_approach":"Change in specified vital sign measurements from baseline."}
• {"endpoint_text":"- 11. Change from baseline in weight and height","definition_or_measurement_approach":"Measured change in weight and height from baseline."}
• {"endpoint_text":"- 12. Change from baseline in targeted clinical laboratory test results","definition_or_measurement_approach":"Change in predefined clinical laboratory parameters from baseline."}
• {"endpoint_text":"- 13. Change from baseline in targeted ECG parameters","definition_or_measurement_approach":"Change in specified ECG parameters from baseline."}
• {"endpoint_text":"- 14. Incidence of anti-drug antibodies (ADAs) at baseline and incidence of ADAs during the study","definition_or_measurement_approach":"Incidence and timing of anti-drug antibodies measured at baseline and during the study."}

Methods

• Study brochure for parents/caregivers (country-specific; e.g. IT document K2_WN41733_Study-brochure-for-parents-caregivers_IT_Italian_Public)
• Recruitment infographic (Italy-specific; K2_WN41733_Pediatric-Clinical-Study-Infographic_IT_Italian_Public)
• Referral letter (Italy-specific; K2_WN41733_Referral-Letter_IT_Italian_Public)
• Recruitment and informed consent procedure documents (Poland and Italy; e.g. K1_WN41733_Recruitment-and-Informed-consent-procedure_PL_Polish_Public and K2_WN41733_Recruitment_Informed_Consent_Procedure_IT_Public)
• Parent/guardian informed consent forms and privacy ICFs (Italian and Polish versions listed among documents)
• Age-specific assent forms for children (Italian and Polish versions listed among documents)

France

Earliest CTIS Part Ii Submission Date

25-06-2024

Latest Decision Or Authorization Date

16-07-2024

Processing Time Days

21

Number Of Sites

1

Number Of Participants

1

Italy

Earliest CTIS Part Ii Submission Date

25-06-2024

Latest Decision Or Authorization Date

13-08-2024

Processing Time Days

49

Number Of Sites

2

Number Of Participants

4

Poland

Earliest CTIS Part Ii Submission Date

25-06-2024

Latest Decision Or Authorization Date

22-07-2024

Processing Time Days

27

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

F. Hoffmann-La Roche AG

Organisation Type

Pharmaceutical company

Country Of Registered Address

Switzerland

Contract research organisations

Name

PPD Development LP

Responsibilities

Global CRO, Monitoring, Country and Site management

Third parties

• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Q Squared Solutions Holdings LLC","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Global CRO, Monitoring, Country and Site management","organisation_type":"Pharmaceutical company"}
• {"country":"Japan","full_name":"SRL Inc.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Japan","full_name":"LSI Medience Corp.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}