BridgeMD
CAN-GT
EU CTIS ID: 2024-519412-13-00
What this study is testing
Assess the effect of rAAV-OLIG001-ASPA on cerebrospinal fluid (CSF) NAA concentrations in children with Canavan disease as measured by biochemical assay
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Definitive diagnosis of typical CD by a neurologist (see Section 3.2 of protocol for detailed description).
- 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.
- 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.
- 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.
You likely can't join if
- 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.
- History of severe allergic reaction or anaphylaxis.
- Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.
- Any absolute contraindication to immunosuppression.
- Any absolute contraindication to MRI.
- Any vaccination less than 1 month prior to gene therapy.
See the full eligibility criteria
- Definitive diagnosis of typical CD by a neurologist (see Section 3.2 of protocol for detailed description).
- 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.
- 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.
- 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.
- 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.
- History of severe allergic reaction or anaphylaxis.
- Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.
- Any absolute contraindication to immunosuppression.
- Any absolute contraindication to MRI.
- Any vaccination less than 1 month prior to gene therapy.
- Anticipated life expectancy of less than 12 months for any reason.
- GMFM-88 total raw score >35%.
- Clinically significant out-of-range lab values as indicated in Appendix G, at the discretion of clinical PI.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.