Lung DiseasePhase 1/2AATDNCT06622668
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
A Phase 1/2 study testing Biological NTLA-3001 in people with Lung Disease and Pulmonary Disease, run by Intellia Therapeutics at 1 site in New Zealand.
Trial summary
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Lung Disease, Pulmonary Disease, AATD
- What is being tested?
- Biological NTLA-3001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteNew Zealand
- Study dates
- 20 Nov 2024to 9 Jan 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18 years to 75 years
- Diagnosis of AATD ZZ/ZNull genotypes
- FEV1 ≥35% and ≤65%
- No evidence of liver cirrhosis
- Adequate chemistry and hematology measures at screening
- Participants must agree not to participate in another interventional study for the duration of this trial.
- Participants must be capable of providing signed informed consent
Who cannot join
- AATD genotypes outside of ZZ or ZNull
- Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
- Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
- Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
- Unwilling to comply with study procedures.
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
Read the detailed description
This study consists of 2 parts: Phase 1 is an open-label, single-arm ascending dose study to characterize the safety and activity of NTLA3001 and identify the dose for evaluation in Phase 2. Phase 2 will follow as an open-label, dose expansion study to further characterize the safety and clinical activity of NTLA-3001 and provide an initial assessment of the effect of NTLA-3001 on clinical measures of pulmonary function.
- Conditions
- Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease
- Being tested
- Biological NTLA-3001
- Keywords
- AATD
- Medical subject headings
- Lung Diseases, alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biological NTLA-3001Biological
IV administration of AAV and CRISPR/Cas9 gene editing system
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Not described Receives: Biological: Biological NTLA-3001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
Measured: From NTLA-3001 infusion up to week156 post infusion
Secondary outcomes (3)
Pharmacodynamics
To evaluate the PD effect of NTLA-3001
Measured: From NTLA-3001 infusion up to week 156 post infusion
Immune Response
To evaluate the immune response to NTLA-3001
Measured: From NTLA-3001 infusion up to week 156 post infusion
Vector Shedding
To evaluate vector shedding following administration of NTLA-3001
Measured: From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2024
- Study start20 Nov 2024
- Primary completion9 Jan 2025 (estimated)
- Study completion9 Jan 2025
Why it stopped: Clinical trial ITL-3001-CL-101 for the investigational product NTLA-3001 was withdrawn due to prioritization of other Sponsor programs.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lung DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Intellia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06622668
- Sponsor's protocol ID
- ITL-3001-CL-101
- Acronym
- AATD
- Status last verified
- Jan 2025
- First submitted
- 30 Sep 2024
- Last updated
- 17 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06622668
- Acronym / sponsor ID
- AATD / ITL-3001-CL-101
- Conditions
- Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease
- Keywords
- AATD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Clinical trial ITL-3001-CL-101 for the investigational product NTLA-3001 was withdrawn due to prioritization of other Sponsor programs.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Intellia TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 20 Nov 2024 (actual)
- Primary completion
- 9 Jan 2025 (estimated)
- Study completion
- 9 Jan 2025 (actual)
- First posted
- 2 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
Detailed description
This study consists of 2 parts: Phase 1 is an open-label, single-arm ascending dose study to characterize the safety and activity of NTLA3001 and identify the dose for evaluation in Phase 2. Phase 2 will follow as an open-label, dose expansion study to further characterize the safety and clinical activity of NTLA-3001 and provide an initial assessment of the effect of NTLA-3001 on clinical measures of pulmonary function.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Biological: Biological NTLA-3001 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age 18 years to 75 years
- Diagnosis of AATD ZZ/ZNull genotypes
- FEV1 ≥35% and ≤65%
- No evidence of liver cirrhosis
- Adequate chemistry and hematology measures at screening
Key exclusion criteria (5 listed)
- AATD genotypes outside of ZZ or ZNull
- Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
- Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
- Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
- Unwilling to comply with study procedures.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biological NTLA-3001IV administration of AAV and CRISPR/Cas9 gene editing system | Biological | Arm 1 |
Primary outcome
Safety and Tolerability
Time frame: From NTLA-3001 infusion up to week156 post infusion
To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
3 secondary outcomes
Pharmacodynamics
Time frame: From NTLA-3001 infusion up to week 156 post infusion
Immune Response
Time frame: From NTLA-3001 infusion up to week 156 post infusion
Vector Shedding
Time frame: From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Biological NTLA-3001 in people with Lung Disease and Pulmonary Disease, run by Intellia Therapeutics at 1 site in New Zealand.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 17 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability (time frame: From NTLA-3001 infusion up to week156 post infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jan 2025.