Safety and Tolerability Study of CTx1000 In Participants... | NCT07401121

Studies
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis

NCT07401121

Sponsor

Celosia Therapeutics Pty Ltd

Status

Recruiting

Last Update Posted

Feb 10, 2026Actual

Enrollment

15Estimated

Phase

Phase 1

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Interventions

AAV9 Gene therapy

Countries

Australia

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 12, 2026Actual

Primary Completion Date

Dec 2027Estimated

Completion Date

Dec 2030Estimated

First Submitted Date

Dec 10, 2025

First Submission Date that Met QC Criteria

Feb 6, 2026

First Posted Date

Feb 10, 2026Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 6, 2026

Last Update Posted Date

Feb 10, 2026Actual

Name	Class
Novotech (Australia) Pty Limited	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.

Detailed Description

CTx1000 is an investigational gene therapy that encodes a degron for targeted degradation of TDP-43 following a single dose intra cisterna magna (ICM) delivery in participants diagnosed with Amyotrophic Lateral Sclerosis (ALS).

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

15Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Active drug-CTx1000	Experimental	All participants will receive only one dose of the study drug	Drug: AAV9 Gene therapy

Interventions

Name	Type	Description	Arm Group Labels	Other Names
AAV9 Gene therapy	Drug	Single dose gene therapy	Active drug-CTx1000

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants	Incidence of Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)	52 weeks

Secondary Outcomes

Measure	Description	Time Frame
To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants	Incidence of Treatment Emergent Adverse Events (TEAEs), Severe Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)	3 years
To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score
An overall disease duration of ≤ 2 years after the participant's first symptoms
No or low circulating anti-AAV9 antibodies (titre ≤ 1:50
Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening
Not pregnant or breastfeeding, or willing to cease breastfeeding
All participants must use a barrier method of contraception

Exclusion Criteria:

Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing
Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C > 9%)
Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.
Inadequate organ function
Any participant with a current open tracheostomy

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

80 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Facility	Status	City	State	ZIP	Country	Contacts
Macquarie University Hospital	Recruiting	Sydney	New South Wales	2109	Australia

Label	URL
Related Info	View source

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D000690	Amyotrophic Lateral Sclerosis
D016472	Motor Neuron Disease

Ancestor Terms

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D019636	Neurodegenerative Diseases

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Browse Branches

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