ADENO-ASSOCIATED VIRAL VECTOR SEROTYPE S3 CONTAINING CODON-OPTIMISED EXPRESSION CASSETTE ENCODING HUMAN BETA-GLUCOCEREBROSIDASE VARIANT for Gaucher disease type 1

Inclusion criteria

• {"criterion_text":"- 1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).\n- 2. Participants able to give full informed consent and able to comply with all requirements of the trial."}

Exclusion criteria

• {"criterion_text":"- Not applicable."}

Primary endpoints

• {"endpoint_text":"- Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)","definition_or_measurement_approach":"Incidence (count/rate) of treatment-emergent adverse events, including dose-limiting toxicities (DLTs). No further definition provided in the available record."}

Secondary endpoints

• {"endpoint_text":"- 1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments.","definition_or_measurement_approach":"Incidence counts of AEs and SAEs; assessment of changes from baseline in vital signs, 12-lead ECG, physical exam, and labs."}
• {"endpoint_text":"- 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time.","definition_or_measurement_approach":"Change-from-baseline measurements of GCase activity in DBS, plasma (activity and relative concentration), and leukocyte GCase activity over time."}
• {"endpoint_text":"- 3. Change from baseline in lyso-Gb1 concentration.","definition_or_measurement_approach":"Change-from-baseline measurement of lyso-Gb1 concentration over time."}
• {"endpoint_text":"- 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI.","definition_or_measurement_approach":"Change-from-baseline measurements for hemoglobin, platelet count, spleen volume (MRI) and liver volume (MRI)."}
• {"endpoint_text":"- 5. Change from baseline in GCase antibody levels.","definition_or_measurement_approach":"Change-from-baseline measurement of anti-GCase antibody levels."}
• {"endpoint_text":"- 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)","definition_or_measurement_approach":"Time-to-event measurement: time until three consecutive negative samples for vector genomes in plasma and semen, where applicable."}

Spain

Earliest CTIS Part Ii Submission Date

09-04-2024

Latest Decision Or Authorization Date

15-10-2025

Processing Time Days

554

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Spur Therapeutics Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

United Kingdom

Contract research organisations

Name

Medpace Finland Oy

Responsibilities

sponsorDuties codes: [1,12,13,15,2,4,5,6,8]; includes 'Clinical Imaging Services'

Name

PPD International Holdings LLC

Responsibilities

sponsorDuties codes: [4]

Name

Everest Clinical Research Corporation

Responsibilities

sponsorDuties codes: [11]

Third parties

• {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"sponsorDuties codes: [4]; contact email: claudia.cozma@centogene.com","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"sponsorDuties codes: [1,12,13,15,2,4,5,6,8]; includes 'Clinical Imaging Services' for code 15","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"sponsorDuties codes: [15]; value: 'Patient travel reimbursement'","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Canada","full_name":"Everest Clinical Research Corporation","duties_or_roles":"sponsorDuties codes: [11]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: [7]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}