CIRACIGENE GOLPARVOVEC for Canavan disease

Inclusion criteria

• {"criterion_text":"- Definitive diagnosis of typical CD by a neurologist (see Section 3.2 of protocol for detailed description).\n- Age between 3 and 60 months at the time of neurosurgical administration of rAAV- Olig001-ASPA or over the age of 60 months for untreated patients in Cohort 4.\n- Written informed consent from parent(s)/guardian(s). Consent to enroll into the study will include a written agreement to comply with all the conditions of the study, including attendance at follow-up visits.\n- MRI or CT within the past 3 months, for preliminary neurosurgical planning; if no MRI or CT within 3 months is available, MRI can be done during Screening assuming that the results of all other Inclusion/Exclusion Criteria are acceptable."}

Exclusion criteria

• {"criterion_text":"- At the discretion of the PI, any significant chronic medical condition, including, but not limited to neurological, cardiac, hepatic, renal, hematological, gastrointestinal, endocrine, pulmonary, or infectious disease, which would put the subject at increased risk during surgery or which would interfere with participation in the study, interpretation of safety monitoring, or the integrity of the study data.\n- History of severe allergic reaction or anaphylaxis.\n- Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.\n- Any absolute contraindication to immunosuppression.\n- Any absolute contraindication to MRI.\n- Any vaccination less than 1 month prior to gene therapy.\n- Anticipated life expectancy of less than 12 months for any reason.\n- GMFM-88 total raw score >35%.\n- Clinically significant out-of-range lab values as indicated in Appendix G, at the discretion of clinical PI."}

Primary endpoints

• {"endpoint_text":"- Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.","definition_or_measurement_approach":"Change from baseline to Month 6 in cerebrospinal fluid (CSF) N-acetylaspartate (NAA) concentrations measured by biochemical assay."}

Secondary endpoints

• {"endpoint_text":"- Rate of change in myelin volume in the brain as measured by SyMRI through Month 12.","definition_or_measurement_approach":"Measured by SyMRI (rate of change in myelin volume through Month 12)."}
• {"endpoint_text":"- Rate of change from baseline in Gross Motor Function Measure-88 total score, comparing FIH gene therapy–treated cohort to an external natural history cohort.","definition_or_measurement_approach":"Change from baseline in GMFM-88 total score compared between first-in-human (FIH) gene therapy–treated cohort and an external natural history (external control) cohort."}
• {"endpoint_text":"- Rate of change from baseline in Gross Motor Function Measure-88 specific domains, comparing FIH gene therapy–treated cohort to an external natural history cohort: - Lying/rolling domain - Sitting domain - Crawling domain - Standing domain - Walking domain","definition_or_measurement_approach":"Domain-specific GMFM-88 rates of change from baseline (lying/rolling, sitting, crawling, standing, walking) compared to an external natural history cohort."}
• {"endpoint_text":"- Rate of change from baseline in multivariate MSEL domains age-equivalent scores compared with an external natural history cohort. MSEL domains include: Visual Reception, Fine Motor, Receptive Language, Expressive Language, Gross Motor","definition_or_measurement_approach":"Multivariate MSEL age-equivalent domain scores (visual reception, fine motor, receptive language, expressive language, gross motor) rate of change from baseline compared with external natural history cohort."}
• {"endpoint_text":"- Rate of change from baseline in univariate MSEL domains age-equivalent scores compared with an external natural history cohort: - MSEL Receptive Language domain - MSEL Expressive Language domain - MSEL Visual Reception domain - MSEL Fine Motor domain - MSEL - Gross Motor domain","definition_or_measurement_approach":"Univariate MSEL age-equivalent domain scores rate of change from baseline for each listed domain compared with external natural history cohort."}
• {"endpoint_text":"- Change from baseline to Month 6 in urine NAA concentrations, as measured by biochemical assays","definition_or_measurement_approach":"Change from baseline to Month 6 in urine NAA concentrations measured by biochemical assay."}

France

Earliest CTIS Part Ii Submission Date

03-02-2026

Latest Decision Or Authorization Date

07-04-2026

Processing Time Days

63

Number Of Sites

1

Number Of Participants

7

Primary sponsor

Full Name

Myrtelle Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

Sponsor duties codes: 10

Name

Voisin Consulting CH SARL

Responsibilities

Sponsor duties include Trial Master File management (code 15) and other listed responsibilities (codes 1,11,12,5,6,7,8)

Third parties

• {"country":"United States","full_name":"Mural Health Technologies Inc.","duties_or_roles":"Patient reimbursement and patient level","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Kennedy Krieger Institute","duties_or_roles":"NAA Analysis in CSF and Urine; other duties (code 4) listed","organisation_type":"Health care"}
• {"country":"United States","full_name":"Kcas LLC","duties_or_roles":"Duties listed (code 4)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Perceptive Informatics Inc.","duties_or_roles":"SyMRI","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Voisin Consulting CH SARL","duties_or_roles":"Multiple duties including Trial Master File management (code 15 value provided) and other listed responsibilities (codes 1,11,12,5,6,7,8)","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Duties listed (code 10)","organisation_type":"Pharmaceutical company"}