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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06622668




Registration number
NCT06622668
Ethics application status
Date submitted
30/09/2024
Date registered
2/10/2024

Titles & IDs
Public title
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
Scientific title
Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
Secondary ID [1] 0 0
ITL-3001-CL-101
Universal Trial Number (UTN)
Trial acronym
AATD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lung Disease 0 0
Pulmonary Disease 0 0
AATD 0 0
Alpha-1 Antitrypsin Deficiency 0 0
Alpha-1 Antitrypsin Deficiency-associated Lung Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Biological NTLA-3001

Experimental: Arm 1 -


Treatment: Other: Biological NTLA-3001
IV administration of AAV and CRISPR/Cas9 gene editing system
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and Tolerability
Timepoint [1] 0 0
From NTLA-3001 infusion up to week156 post infusion
Secondary outcome [1] 0 0
Pharmacodynamics
Timepoint [1] 0 0
From NTLA-3001 infusion up to week 156 post infusion
Secondary outcome [2] 0 0
Immune Response
Timepoint [2] 0 0
From NTLA-3001 infusion up to week 156 post infusion
Secondary outcome [3] 0 0
Vector Shedding
Timepoint [3] 0 0
From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.

Eligibility
Key inclusion criteria
Inclusion:

1. Age 18 years to 75 years
2. Diagnosis of AATD ZZ/ZNull genotypes
3. FEV1 =35% and =65%
4. No evidence of liver cirrhosis
5. Adequate chemistry and hematology measures at screening
6. Participants must agree not to participate in another interventional study for the duration of this trial.
7. Participants must be capable of providing signed informed consent
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. AATD genotypes outside of ZZ or ZNull
2. Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
3. Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
4. Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
5. Unwilling to comply with study procedures.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/10/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/05/2030
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Aukland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Intellia Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Manager at Intellia
Address 0 0
Country 0 0
Phone 0 0
1-857-285-6200
Fax 0 0
Email 0 0
medicalinformation@intelliatx.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06622668