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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06634420




Registration number
NCT06634420
Ethics application status
Date submitted
7/10/2024
Date registered
9/10/2024

Titles & IDs
Public title
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Scientific title
HAELO: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Secondary ID [1] 0 0
ITL-2002-CL-301
Universal Trial Number (UTN)
Trial acronym
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 - NTLA-2002
Treatment: Other - Normal Saline IV Administration

Active comparator: Arm A: NTLA-2002 - Arm A: NTLA-2002 (50 mg; single IV infusion)

Placebo comparator: Arm B: Placebo - Arm B: Placebo (saline; single IV infusion)


Treatment: Other: NTLA-2002
CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration

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

Outcomes
Primary outcome [1] 0 0
Time-normalized number of Investigator-confirmed HAE attacks
Timepoint [1] 0 0
From Week 5 through Week 28
Secondary outcome [1] 0 0
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment
Timepoint [1] 0 0
From Week 5 through Week 28
Secondary outcome [2] 0 0
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks
Timepoint [2] 0 0
From Week 5 through Week 28
Secondary outcome [3] 0 0
Investigator-confirmed HAE attack-free status
Timepoint [3] 0 0
From Week 5 through Week 28
Secondary outcome [4] 0 0
Change from baseline to Week 28 in AE-QoL Questionnaire total score
Timepoint [4] 0 0
From baseline to Week 28
Secondary outcome [5] 0 0
Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks
Timepoint [5] 0 0
From Week 5 through Week 28
Secondary outcome [6] 0 0
Time-normalized number of Investigator-confirmed HAE attacks
Timepoint [6] 0 0
From Week 1 through Week 28
Secondary outcome [7] 0 0
Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment
Timepoint [7] 0 0
from Week 1 through Week 28
Secondary outcome [8] 0 0
Time-normalized number of moderate or severe Investigator-confirmed HAE attacks
Timepoint [8] 0 0
From Week 1 through Week 28
Secondary outcome [9] 0 0
Investigator-confirmed HAE attack-free status
Timepoint [9] 0 0
From Week 1 through Week 28
Secondary outcome [10] 0 0
Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks
Timepoint [10] 0 0
From Week 1 through Week 28
Secondary outcome [11] 0 0
Long-term prophylaxis-free status
Timepoint [11] 0 0
From Week 5 through Week 104
Secondary outcome [12] 0 0
HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication
Timepoint [12] 0 0
From Week 5 through Week 104
Secondary outcome [13] 0 0
Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis
Timepoint [13] 0 0
From Week 5 through Week 104
Secondary outcome [14] 0 0
Reduction in the time-normalized number of Investigator-confirmed HAE attacks after crossover treatment (Week 33 through Week 104) compared to before crossover treatment (Screening through Week 28) in participants randomized to placebo
Timepoint [14] 0 0
From Week 33 through Week 104 and from Screening through Week 28

Eligibility
Key inclusion criteria
1. Age =16 years
2. Clinical history consistent with HAE-C1INH-Type 1 or -Type 2
3. Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement
4. Must agree to refrain from the use of long-term prophylactic therapies from the start of the screening period through the end of the Primary Observation Period. PI must be in agreement that it is medically acceptable for the participant to do so.
5. Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks
6. Adequate chemistry and hematology measures at screening
7. Must agree not to participate in another interventional study for the duration of this trial.
8. Must be capable of providing signed informed consent. Participants 16 to < 18 years of age, whose legal guardian provides informed consent, must provide assent.
9. Must agree to follow contraception requirements
Minimum age
16 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 or HAE with normal C1-INH
2. 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 administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
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
Actual
15/01/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2027
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Campbelltown Hospital - Campbelltown
Recruitment hospital [2] 0 0
The Alfred Hospital - Melbourne, VIC
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment postcode(s) [2] 0 0
3004 - Melbourne, VIC
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Michigan
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
France
State/province [13] 0 0
Grenoble
Country [14] 0 0
France
State/province [14] 0 0
Marseille
Country [15] 0 0
Germany
State/province [15] 0 0
Berlin
Country [16] 0 0
Germany
State/province [16] 0 0
Hannover
Country [17] 0 0
Netherlands
State/province [17] 0 0
Amsterdam
Country [18] 0 0
New Zealand
State/province [18] 0 0
Auckland
Country [19] 0 0
South Africa
State/province [19] 0 0
Cape Town
Country [20] 0 0
United Kingdom
State/province [20] 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
Trial Manager at Intellia Therapeutics
Address 0 0
Country 0 0
Phone 0 0
1-857-285-6200
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


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/NCT06634420