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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05120830




Registration number
NCT05120830
Ethics application status
Date submitted
3/11/2021
Date registered
15/11/2021

Titles & IDs
Public title
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Scientific title
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Secondary ID [1] 0 0
ITL-2002-CL-001
Universal Trial Number (UTN)
Trial acronym
NTLA-2002
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Biological NTLA-2002
Other interventions - Normal Saline IV Administration

Experimental: Phase 1 Study Arm - Participants assigned to 1 of 3 dose-escalation cohorts will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.

Experimental: Phase 2 Experimental Study Arm - Participants randomized to NTLA-2002 (2 dose levels), will receive a single dose of NTLA-2002 on Day 1 and will then be followed for 104 weeks. Primary observation period is 16 weeks.

Placebo comparator: Phase 2 Placebo Comparator Study Arm - Participants randomized to placebo will receive IV normal saline on Day 1 and will then be followed for up to 104 weeks. Primary observation period is 16 weeks.

Experimental: Placebo Crossover and Follow-On Dosing Substudy Arm - Participants assigned to this Substudy Arm (participants who previously received either 25mg or placebo only) will have the opportunity to receive a single dose of NTLA-2002 (50mg) and will then be followed for 52 weeks.


Treatment: Other: Biological NTLA-2002
CRISPR/Cas9 gene editing system delivered by LNP for IV administration

Other interventions: Normal Saline IV Administration
The administration of IV normal saline
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of NTLA-2002 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)
Timepoint [1] 0 0
From NTLA-2002 infusion up to week 104 post-infusion
Primary outcome [2] 0 0
Number of HAE attacks per month (Weeks 1-16)
Timepoint [2] 0 0
From study drug infusion up to week 16 post-infusion
Secondary outcome [1] 0 0
Change from baseline in total plasma kallikrein protein level
Timepoint [1] 0 0
From NTLA-2002 infusion up to week 104 post-infusion
Secondary outcome [2] 0 0
Plasma and urine concentrations for DMG-PEG2k, LP000001, Cas9 mRNA, and sgRNA
Timepoint [2] 0 0
From NTLA-2002 infusion up to week 104 post-infusion
Secondary outcome [3] 0 0
Safety and tolerability of NTLA-2002 as determined by AEs
Timepoint [3] 0 0
From study drug infusion up to week 104 post-infusion
Secondary outcome [4] 0 0
Number of HAE attacks per month (Weeks 5-16)
Timepoint [4] 0 0
From week 6 post-infusion up to week 16 post-infusion
Secondary outcome [5] 0 0
Number of HAE attacks per month requiring acute therapy (Weeks 1-16, Weeks 5-16)
Timepoint [5] 0 0
From study drug infusion up to week 16 post-infusion

Eligibility
Key inclusion criteria
1. Age >18 years
2. Diagnosis of HAE Types I or II
3. Ability to provide evidence of HAE attacks to meet the screening requirement
4. Subjects must have access to, and the ability to use, = 1 acute medication(s) to treat angioedema attacks.
5. Adequate chemistry and hematology measures at screening
6. Subjects must agree not to participate in another interventional study for the duration of this trial.
7. Subjects must be capable of providing signed informed consent
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Concurrent diagnosis of any other type of recurrent angioedema
2. Subjects who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
3. Any condition that, in the Investigator's opinion, could adversely affect the safety of the subject.
4. Unwilling to comply with study procedures.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
10/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/03/2026
Actual
Sample size
Target
Accrual to date
Final
37
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Clinical Trial Site - Campbelltown
Recruitment postcode(s) [1] 0 0
- Campbelltown
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Grenoble
Country [2] 0 0
France
State/province [2] 0 0
Lille
Country [3] 0 0
France
State/province [3] 0 0
Paris
Country [4] 0 0
Germany
State/province [4] 0 0
Berlin
Country [5] 0 0
Germany
State/province [5] 0 0
Frankfurt
Country [6] 0 0
Netherlands
State/province [6] 0 0
Amsterdam
Country [7] 0 0
New Zealand
State/province [7] 0 0
Auckland
Country [8] 0 0
United Kingdom
State/province [8] 0 0
Cambridge

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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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/NCT05120830