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

Phase 1RecruitingNCT07401121

Celosia Therapeutics Pty Ltd15 enrolled

Overview

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

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Interventions

AAV9 Gene therapyDRUG

Single dose gene therapy

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (1)

Macquarie University Hospital

Sydney, New South Wales, Australia

RECRUITING

Outcomes

Primary Outcomes

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)

Time frame: 52 weeks

Secondary Outcomes

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)

Time frame: 3 years

To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants

Changes in pre-dose biomarkers (Neurofilament light) in cerebrospinal fluid and anti-drug antibody (ADA)

Time frame: 3 years

To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants

Changes (reduction) from pre-dose Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)

Time frame: 3 years

Data from ClinicalTrials.gov

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