Can-Gt

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Myrtelle Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Decision date (initial)

2026-02-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Myrtelle, Inc.

External identifiers

EU CT number

2024-519412-13-00

ClinicalTrials.gov

NCT04833907

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Pharmacodynamic, Efficacy

Assess the effect of rAAV-OLIG001-ASPA on cerebrospinal fluid (CSF) NAA concentrations in children with Canavan disease as measured by biochemical assay

Secondary objectives 6

1. Assess the effect of rAAV-OLIG001-ASPA on myelination in children with Cavanan disease as measured by volumetric MRI.
2. Assess the effect of rAAV-OLIG001-ASPA on gross motor development in children with Cavanan disease as measured by GMFM compared to an external control group
3. Assess the domain-specific effect of rAAV-OLIG001-ASPA on gross motor development in children with Cavanan disease as measured by GMFM compared to an external control group
4. Evaluate the overall effect of rAAV-OLIG001-ASPA on the developmental progress in children with Canavan disease compared to an external control group as measured by MSEL age-equivalent domain scores
5. Evaluate the domain-specific effects of rAAV-OLIG001-ASPA on the developmental progress in children with Canavan disease compared to an external control group as measured by MSEL age-equivalent domain scores
6. Assess the effect of rAAV-OLIG001-ASPA on urine NAA concentration as measured by biochemical assay

Conditions and MedDRA coding

Canavan disease

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003459-PIP01-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Definitive diagnosis of typical CD by a neurologist (see Section 3.2 of protocol for detailed description).
2. 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.
3. 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.
4. 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 9

1. 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.
2. History of severe allergic reaction or anaphylaxis.
3. Past participation in gene therapy trials or receipt of any other investigational product within 6 months prior to enrollment.
4. Any absolute contraindication to immunosuppression.
5. Any absolute contraindication to MRI.
6. Any vaccination less than 1 month prior to gene therapy.
7. Anticipated life expectancy of less than 12 months for any reason.
8. GMFM-88 total raw score >35%.
9. Clinically significant out-of-range lab values as indicated in Appendix G, at the discretion of clinical PI.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to Month 6 in CSF NAA concentrations, as measured by biochemical assay.

Secondary endpoints 6

1. Rate of change in myelin volume in the brain as measured by SyMRI through Month 12.
2. 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.
3. 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
4. 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
5. 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
6. Change from baseline to Month 6 in urine NAA concentrations, as measured by biochemical assays

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Myrtelle Inc.

Sponsor organisation

Myrtelle Inc.

Address

17 State Street

City

New York

Postcode

10004-1501

Country

United States

Scientific contact point

Organisation

Myrtelle Inc.

Contact name

Olga Flamini

Public contact point

Organisation

Myrtelle Inc.

Contact name

Olga Flamini

Third parties 6

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 7 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications