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Gene Therapy Trial Report

Summary

HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)


NCTID NCT06634420 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Hereditary Angioedema
Disease Ontology Term DOID:14735
Compound Name NTLA-2002
Compound Alias lonvoguran ziclumeran
Sponsor Intellia Therapeutics
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 60 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant KLKB1
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Gene inactivation
Route of Administration Intravenous
Drug Product Type MRNA, LNP
Target Tissue/Cell Hepatocyte
Delivery System Lipid encapsulation
Vector Type LDLR
Editor Type Cas9 mRNA
Dose 1 Phase 1/2: 25mg, 50mg, 75mg
Dose 2 Phase 3: 50 mg

Study Record Dates


Current Stage Phase3
Submit Date 2024-10-07
Completion Date 2027-09
Last Update 2025-12-18

Participation Criteria


Eligible Age >=16 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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 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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 29
Locations New Zealand,Canada,Netherlands,United States,South Africa,United Kingdom,Australia,France,Germany

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation, Regenerative Medicine Advanced Therapy
Recent Updates Completed enrollment in Phase 3 trial in September 2025, topline data expected by mid-2026, BLA submission planned H2 2026

Resources/Links