REBISUFLIGENE ETISPARVOVEC for Mucopolysaccharidosis type IIIA

Inclusion criteria

• {"criterion_text":"- Participants that have participated in a prior clinical trial in which they received UX111\n- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule."}

Exclusion criteria

• {"criterion_text":"- Planned or current participation in another clinical trial that may confound the safety or efficacy evaluation of UX111 during this study\n- Any other situation or medical condition that precludes the participant from undergoing procedures required in this study."}

Primary endpoints

• {"endpoint_text":"- Safety: Incidence of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs)","definition_or_measurement_approach":"Incidence will be measured by recording and reporting treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (SAEs) as counted/reported during follow-up."}
• {"endpoint_text":"- Efficacy: Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive raw score","definition_or_measurement_approach":"Measured using the Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Cognitive raw score."}

Secondary endpoints

• {"endpoint_text":"- Cerebrospinal fluid (CSF) heparan sulfate (HS) (disaccharide) exposure","definition_or_measurement_approach":"Measured as CSF heparan sulfate (HS) (disaccharide) exposure in cerebrospinal fluid samples."}
• {"endpoint_text":"- CSF HS percentage change from baseline in prior trial","definition_or_measurement_approach":"Calculated as percentage change in CSF heparan sulfate (HS) from baseline values recorded in the prior trial."}
• {"endpoint_text":"- BSITD-III Receptive Communication raw score","definition_or_measurement_approach":"Measured using the Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Receptive Communication raw score."}
• {"endpoint_text":"- BSITD-III Expressive Communication raw score","definition_or_measurement_approach":"Measured using the Bayley Scales of Infant and Toddler Development - Third Edition (BSITD-III) Expressive Communication raw score."}
• {"endpoint_text":"- Total cortical volume (cm 3) annualized percentage change from baseline in prior trial","definition_or_measurement_approach":"Measured as total cortical volume (cm3) and reported as the annualized percentage change from baseline values in the prior trial."}
• {"endpoint_text":"- Survival","definition_or_measurement_approach":"Measured as survival status/time during the follow-up period."}

Spain

Earliest CTIS Part Ii Submission Date

31-01-2024

Latest Decision Or Authorization Date

20-10-2025

Processing Time Days

629

Number Of Sites

3

Number Of Participants

24

Primary sponsor

Full Name

Ultragenyx Pharmaceutical Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Pharmapace Inc.

Name

DF/Net Research

Name

Fortrea Inc.

Responsibilities

Home Health

Name

Primevigilance Limited

Responsibilities

Pharmacovigilance Services

Third parties

• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Anti-AAV9 ELISA, Anti-SGSH ELISA, NABs","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Patient Primary","duties_or_roles":"Patient reimburment","organisation_type":"Industry"}
• {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"Collection/shipping of lab kits, shipment samples","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmapace Inc.","duties_or_roles":"","organisation_type":"Pharmapace Inc."}
• {"country":"Australia","full_name":"Sa Pathology","duties_or_roles":"","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Marken LLP","duties_or_roles":"Lab Sample Courier","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmapace Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Home Health","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Trulab","duties_or_roles":"Lab Sample Management","organisation_type":"Industry"}
• {"country":"United States","full_name":"Quantims, LLC (Igor Nestrasil)","duties_or_roles":"Central MRI reader","organisation_type":"Industry"}
• {"country":"United States","full_name":"EPL Archives, LLC","duties_or_roles":"Samples storage","organisation_type":"Health care"}
• {"country":"United States","full_name":"Lumanity Patient Centered Outcomes LLC","duties_or_roles":"Qualitative Interviews","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"Pharmacovigilance Services","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Life Courier (fka Optimize Courier)","duties_or_roles":"Lab Sample Courier","organisation_type":"Industry"}
• {"country":"United States","full_name":"DF/Net Research","duties_or_roles":"","organisation_type":"Industry"}