Canavan-Single Patient IND

Status:

NO_LONGER_AVAILABLE

Canavan-Single Patient IND

Lead Sponsor:

University of Florida

Collaborating Sponsors:

University of Miami

University of Massachusetts, Worcester

Conditions:

Canavan Disease

Eligibility:

MALE

18-24 years

Brief Summary

A recombinant virus vector constructed from adeno-associated virus (AAV) has been engineered to carry the human aspartoacylase (ASPA) gene expressed from a modified CMV-enhancer chicken β-actin (CB6) ...

Eligibility Criteria

Inclusion

Male
18-24 months of age at time of study enrollment
Have a diagnosis of Canavan disease, as defined by biochemical criteria AND/OR genetic mutation analysis, AND demonstrate clinical findings such as macrocephaly, developmental delay, seizures or other positive findings
Elevated brain NAA levels, which is correlated with NAA acidemia and aciduria
Willing to discontinue aspirin, aspirin-containing products and other drugs that may alter platelet function 7 days prior to dosing, resuming 24 hours after the gene transfer agent has been administered

Exclusion

Have required acute (as distinguished from long-term, maintenance or chronic suppressive) oral or intravenous antibiotic therapy for a respiratory infection within 15 days prior to screening
Have required oral or systemic corticosteroids within the last 15 days prior to baseline screening
Have a platelet count less than 75,000/mm3
Have history of platelet dysfunction, evidence of abnormal platelet function at screening, or history of recent use of drugs that may alter platelet function, which the subject is unable/unwilling to discontinue for study agent administration
Have an INR greater than 1.3
Have transaminases and alkaline phosphatase more than ten times the upper limit of normal at screening or Day-1; or an abnormal chemistry profile
Have bilirubin and gamma-glutamyl transpeptidase greater than 2 times the upper limit of normal at screening or Day -1
Be currently or within the past 30 days participating in any other research protocol involving investigational agents or therapies (Other than approved therapy)
Have received gene transfer agents within the past 6 months
Have any other concurrent condition that, in the opinion of the investigator, would make the subject unsuitable for the study.

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT05317780

Last Update

August 23 2023

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