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Note: This record shows only 24 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	22 May 2023
Main ID:	NCT04798235
Date of registration:	22/02/2021
Prospective Registration:	Yes
Primary sponsor:	Dr. Anupam Sehgal
Public title:	First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Scientific title:	Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Date of first enrolment:	March 12, 2021
Target sample size:	3
Recruitment status:	Active, not recruiting
URL:	https://clinicaltrials.gov/show/NCT04798235
Study type:	Interventional
Study design:	Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Phase:	Phase 1/Phase 2

Countries of recruitment
Canada

Contacts

Contact type:
Name:	Anupam Sehgal, MBBS
Address:
Telephone:
Email:
Affiliation:	Queen's University

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- male or female with age less than or equal to 15 months

- diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile
disease

Key Exclusion Criteria:

- a second neurodevelopmental disorder independent of the HEXA or HEXB

- inability to tolerate sedation or intrathecal administration

- invasive ventilatory support

- concomitant illness, allergies or known hypersensitivity to the required
immunosuppression regimen

Age minimum: N/A
Age maximum: 15 Months
Gender: All

Health Condition(s) or Problem(s) studied

Infantile GM2 Gangliosidosis (Disorder)

Intervention(s)

Biological: TSHA-101

Primary Outcome(s)

Safety and tolerability: Treatment-emergent Adverse Events (TEAEs) [Time Frame: 1 year]

Safety and Tolerability: Electrocardiogram (ECG) [Time Frame: 1 year]

Safety and Tolerability: Number of participants with abnormal Laboratory assessments [Time Frame: 1 year]

Secondary Outcome(s)

Head Control: Number of events for abnormal head control [Time Frame: 1 year]

Hex A Enzyme Activity: Cerebrospinal fluid (CSF) and serum [Time Frame: 1 year]

Change from Baseline in Motor Function: Modified Ashworth Scale [Time Frame: 1 year]

Assessment of Immunogenicity: Biomarkers in serum [Time Frame: 1 year]

Safety and tolerability: Viral shedding analysis [Time Frame: 1 year]

Change from Baseline in motor function: Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) [Time Frame: 1 year]

Assessment of Immunogenicity: Biomarkers in peripheral blood mononuclear cells (PBMCs [Time Frame: 5 years]

Clinical Efficacy Assessment: Progression of Hypotonia [Time Frame: 1 year]

Clinical Efficacy Assessment: Dysphagia [Time Frame: From onset up to 3 years, if present]

Overall Survival [Time Frame: treatment to death from any cause, up to 5 years]

Secondary ID(s)

TSHA-101-IST-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

GlycoNet

Taysha Gene Therapies, Inc.

Ethics review

Results

Results available:
URL:
URL of the protocol:
Date Posted:
Date of completion:
Date of first publication:
Results summary:
Baseline characteristics:
Adverse events:
Outcome measures:
IPD sharing plan:
IPD sharing description:

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