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Gene Therapy

Gene Therapy for Canavan Disease (CAN-GT Trial)

Phase 1 & 2

Recruiting

Led By Christopher G Janson, MD

Research Sponsored by Myrtelle LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

For cohort 3: age less than 15 months

For cohort 1: age more than 36 months and up to 60 months

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months post dose

Awards & highlights

CAN-GT Trial Summary

This trial is testing a new technology targeted at the cells most affected by Canavan Disease in the safest way possible.

Who is the study for?

This trial is for children with typical Canavan Disease, a brain disorder. Eligible participants are aged 3-60 months and diagnosed by a neurologist. They can't have had gene therapy or intracranial surgery before, severe allergies, MRI contraindications, immune system issues, or other serious health problems.Check my eligibility

What is being tested?

The trial tests rAAV-Olig001-ASPA gene therapy delivered directly to the brain to treat Canavan Disease. It aims to restore enzyme function in cells responsible for producing white matter. The study includes clinical assessments and brain imaging over time.See study design

What are the potential side effects?

Potential side effects may include reactions from the neurosurgical procedure used to administer the gene therapy and complications related to immunosuppression required for treatment.

CAN-GT Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My child is younger than 15 months.

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I am between 3 and 5 years old.

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I am between 15 and 36 months old.

CAN-GT Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months post dose

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months post dose

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months post dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Safety evaluation

Secondary outcome measures

Myelination

N-Acetyl-Aspartate (NAA) concentrations in the Brain

NAA concentration measured in Cerebrospinal Fluid (CSF)

+4 more

CAN-GT Trial Design

1Treatment groups

Experimental Treatment

Group I: 3.7 x 10^13 v.g. rAAV-Olig001-ASPAExperimental Treatment3 Interventions

3.7 x 10^13 v.g. of rAAV-Olig001-ASPA administered as a single dose neurosurgically to the brain via 2 pre-defined intracerebroventricular sites

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Levetiracetam

2017

Completed Phase 4

~3990

Prednisone

2014

Completed Phase 4

~2370

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Myrtelle LLCLead Sponsor

Myrtelle Inc.Lead Sponsor

1 Previous Clinical Trials

12,000 Total Patients Enrolled

1 Trials studying Canavan Disease

12,000 Patients Enrolled for Canavan Disease

Christopher G Janson, MDPrincipal InvestigatorDayton Children's Hospital

Media Library

rAAV-Olig001-ASPA (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04833907 — Phase 1 & 2

Canavan Disease Research Study Groups: 3.7 x 10^13 v.g. rAAV-Olig001-ASPA

Canavan Disease Clinical Trial 2023: rAAV-Olig001-ASPA Highlights & Side Effects. Trial Name: NCT04833907 — Phase 1 & 2

rAAV-Olig001-ASPA (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04833907 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are elderly patients eligible for this research?

"This trial is for patients that are 3 to 60 months old, which falls in between the 46 studies for children and the 77 trials concerning adults over 65 years old."

Answered by AI

What do we know about rAAV-Olig001-ASPA from other research?

"Presently, 112 different clinical trials are underway for rAAV-Olig001-ASPA. Of those active studies, 36 are in Phase 3 testing. Though many of the trials take place in Brisbane, Queensland, there are 3020 total locations running these sorts of tests."

Answered by AI

For what reasons is rAAV-Olig001-ASPA commonly given to patients?

"RAAV-Olig001-ASPA can be used to treat a variety of conditions, such as ulcerative colitis, varicella-zoster virus acute retinal necrosis, and brain cancer."

Answered by AI

If I enroll, am I able to complete the whole clinical trial?

"This study is for children aged 3 months to 5 years old that have canavan disease. A total of 24 participants are needed."

Answered by AI

How many patients are being accepted into this clinical trial?

"At the moment, this clinical trial is not open for enrollment. The first posting was on April 1st 2021 with the most recent update being on June 23rd, 2022. If you are interested in participating in other studies, there are 3 trials involving canavan disease and 112 rAAV-Olig001-ASPA studies that have active participant recruitment."

Answered by AI

Are there any vacancies in this clinical trial for new participants?

"Unfortunately, this particular clinical trial is not currently looking for new patients. Although the study was first posted on 4/1/2021 and was last edited on 6/23/2022, it has since finished recruiting participants. That said, there are 115 other studies that are still recruiting patients."

Answered by AI

Recent research and studies

Molecular Therapy - Methods & Clinical DevelopmentJournal

Preclinical Biodistribution, Tropism, and Efficacy of Oligotropic Aav/olig001 in a Mouse Model of Congenital White Matter Disease

Francis, Jeremy S., Vladimir Markov, Irenuez D. Wojtas, Steve Gray, Thomas McCown, R. Jude Samulski, Marciano Figueroa, and Paola Leone. 2021. “Preclinical Biodistribution, Tropism, and Efficacy of Oligotropic Aav/olig001 in a Mouse Model of Congenital White Matter Disease”. Molecular Therapy - Methods & Clinical Development. Elsevier BV. doi:10.1016/j.omtm.2021.01.009.

Neurobiology of DiseaseJournal

N-acetylaspartate Supports the Energetic Demands of Developmental Myelination via Oligodendroglial Aspartoacylase

Francis, Jeremy S., Ireneusz Wojtas, Vladimir Markov, Steven J. Gray, Thomas J. McCown, R. Jude Samulski, Larissa T. Bilaniuk, et al.. 2016. “N-acetylaspartate Supports the Energetic Demands of Developmental Myelination via Oligodendroglial Aspartoacylase”. Neurobiology of Disease. Elsevier BV. doi:10.1016/j.nbd.2016.10.001.

clinicaltrials.govStudy

rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

2021. "rAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04833907.