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Gene Therapy

Gene Therapy for Tay-Sachs Disease

Phase 1 & 2
Waitlist Available
Led By Anupam Sehgal, MBBS
Research Sponsored by Dr. Anupam Sehgal
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female with age less than or equal to 15 months
Diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

Study Summary

This trial is testing a new drug to treat a group of rare degenerative diseases by injecting it into the brain.

Who is the study for?
This trial is for babies up to 15 months old diagnosed with infantile GM2 gangliosidosis, a neurodegenerative disease. They must have genetic proof of the condition and not be on invasive breathing support or have other major illnesses, allergies to immunosuppressants, another brain development disorder, or issues with sedation.Check my eligibility
What is being tested?
The study tests TSHA-101 gene therapy given through an injection into the spinal canal (intrathecal) to see if it's safe and can be tolerated in treating GM2 gangliosidosis which affects nerve cells due to missing enzyme activity.See study design
What are the potential side effects?
Potential side effects are not detailed but may include reactions related to immune suppression needed for treatment and risks associated with intrathecal injections such as discomfort at the injection site or nervous system complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 15 months old or younger.
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I have been diagnosed with infantile GM2 gangliosidosis, confirmed by genetic and enzyme tests.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years 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 and Tolerability: Electrocardiogram (ECG)
Safety and Tolerability: Number of participants with abnormal Laboratory assessments
Safety and tolerability: Treatment-emergent Adverse Events (TEAEs)
Secondary outcome measures
Assessment of Immunogenicity: Biomarkers in peripheral blood mononuclear cells (PBMCs
Assessment of Immunogenicity: Biomarkers in serum
Change from Baseline in Motor Function: Modified Ashworth Scale
+7 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: TSHA-101Experimental Treatment1 Intervention
Subjects who will receive one-time intrathecal TSHA-101, brain volume based sliding scale for dosage

Find a Location

Who is running the clinical trial?

Dr. Anupam SehgalLead Sponsor
GlycoNetUNKNOWN
Taysha Gene Therapies, Inc.Industry Sponsor
4 Previous Clinical Trials
45 Total Patients Enrolled

Media Library

TSHA-101 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04798235 — Phase 1 & 2
Tay-Sachs Disease Research Study Groups: TSHA-101
Tay-Sachs Disease Clinical Trial 2023: TSHA-101 Highlights & Side Effects. Trial Name: NCT04798235 — Phase 1 & 2
TSHA-101 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04798235 — Phase 1 & 2
Tay-Sachs Disease Patient Testimony for trial: Trial Name: NCT04798235 — 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 there any remaining vacancies in this clinical experiment?

"According to clinicaltrials.gov, the designation for this trial has changed - it is no longer recruiting subject participation. Initially posted on March 12th 2021 and last edited on September 12th 2022, there are currently 11 other trials that require volunteers at present."

Answered by AI

What primary outcomes are investigators aiming to achieve with this experiment?

"According to Taysha Gene Therapies, Inc., the key metric being monitored across this one-year trial is Treatment Emergent Adverse Events (TEAEs). Secondary outcomes include Progression of Hypotonia as assessed through neurological examinations, summary markers for ELISpot assays in Peripheral Blood Mononuclear Cells (PBMCs) against AAV9 and HexA, and positive presence of viral DNA from whole blood, urine saliva or stool - all measured during Safety & Viral Shedding Analysis."

Answered by AI

Who else is applying?

What state do they live in?
Georgia
What site did they apply to?
Queen's University/Kingston Health Sciences Centre
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0

Why did patients apply to this trial?

How long does the trial last, and how long would I need to be in Canada for?
PatientReceived 2+ prior treatments

What questions have other patients asked about this trial?

How long is the screening process to get in?
PatientReceived no prior treatments
Recent research and studies
clinicaltrials.govStudy
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Dr. Anupam Sehgal 2021. "First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04798235.
All > Tay Sachs
~1 spots leftby Mar 2027
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