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)
HAELO: Evaluating NTLA-2002 for Hereditary Angioedema
Plain English Summary
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) is a Phase 3 clinical trial sponsored by Intellia Therapeutics studying Hereditary Angioedema. Tests the effectiveness and safety of NTLA-2002, a gene editing treatment, compared to a placebo. For adults with Hereditary Angioedema (HAE), a rare genetic condition causing swelling attacks. Participation involves receiving either NTLA-2002 or a placebo through IV, and tracking HAE attacks. Alternative treatments include on-demand medications, but long-term prophylactic therapies are not allowed. The trial aims to enroll 60 participants.
Official Summary
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
Who Can Participate
Here is what you need to know about eligibility for this trial. Ages 16 and older with a history of HAE, able to provide evidence of attacks, and willing to avoid long-term prophylactic therapies. No known negative reactions to lipid nanoparticles, and no other recurrent angioedema conditions. Able to provide informed consent, and for minors, parental consent and assent are required. No other interventional studies during the trial. This trial is studying Hereditary Angioedema, so participants generally need a confirmed diagnosis.
What They're Measuring
The primary outcome measures the reduction in HAE attacks, which means fewer swelling episodes for participants. The specific primary outcome measures are: Time-normalized number of Investigator-confirmed HAE attacks (From Week 5 through Week 28). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.
About This Phase
This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.
Why This Trial Matters
This trial fills a critical gap by evaluating a potential gene therapy for HAE, a condition with limited treatment options. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Hereditary Angioedema, where improved treatment options are needed.
Investor Insight
The market for HAE treatments is growing, with limited competition, and there's a high probability of approval given the unmet need. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.
Is This Trial Right for Me?
Ask your doctor if you have a history of HAE and if you're eligible for this trial. Participation involves receiving an IV treatment and tracking your HAE attacks over 28 weeks. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.
AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.
Study Design
- Study Type: INTERVENTIONAL
- Allocation: RANDOMIZED
- Model: PARALLEL
- Masking: QUADRUPLE
- Enrollment: 60 participants
Interventions
- BIOLOGICAL: NTLA-2002 — CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
- BIOLOGICAL: Normal Saline IV Administration — The administration of intravenous (IV) normal saline
Primary Outcomes
- Time-normalized number of Investigator-confirmed HAE attacks (From Week 5 through Week 28)
Secondary Outcomes
- Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment (From Week 5 through Week 28)
- Time-normalized number of moderate or severe Investigator-confirmed HAE attacks (From Week 5 through Week 28)
- Investigator-confirmed HAE attack-free status (From Week 5 through Week 28)
- Change from baseline to Week 28 in AE-QoL Questionnaire total score (From baseline to Week 28)
- Responder status, defined as reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks (From Week 5 through Week 28)
Full 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.
Trial Locations
- Medical Research of Arizona, Scottsdale, Arizona, United States
- University of California, San Diego (UCSD), San Diego, California, United States
- Raffi Tachdjian MD, Inc, Santa Monica, California, United States
- Allergy & Asthma Clinical Research, Walnut Creek, California, United States
- IMMUNOe International Research Centers, Centennial, Colorado, United States
- Asthma & Allergy Associates, Colorado Springs, Colorado, United States
- University of South Florida, Tampa, Florida, United States
- Massachusetts General Hospital, Boston, Massachusetts, United States
- Washington University in St. Louis, St Louis, Missouri, United States
- NYU Langone Health - Long Island, Mineola, New York, United States
- ...and 10 more locations
Frequently Asked Questions
What is clinical trial NCT06634420?
NCT06634420 is a Phase 3 INTERVENTIONAL study titled "HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)." It is currently active, not recruiting and is sponsored by Intellia Therapeutics. The trial targets enrollment of 60 participants.
What conditions does NCT06634420 study?
This trial investigates treatments for Hereditary Angioedema. The primary condition under study is Hereditary Angioedema.
What treatments are being tested in NCT06634420?
The interventions being studied include: NTLA-2002 (BIOLOGICAL), Normal Saline IV Administration (BIOLOGICAL). CRISPR/Cas9 gene editing system delivered by lipid nanoparticle (LNP) for intravenous (IV) administration
What does Phase 3 mean for NCT06634420?
Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.
What is the current status of NCT06634420?
This trial is currently "Active, Not Recruiting." It started on 2025-01-15. The estimated completion date is 2027-09.
Who is sponsoring NCT06634420?
NCT06634420 is sponsored by Intellia Therapeutics. The sponsor is responsible for funding, designing, and overseeing the clinical trial.
How many people can participate in NCT06634420?
The trial aims to enroll 60 participants. The trial status is active, not recruiting.
How is NCT06634420 designed?
This is a interventional study, uses randomized allocation, follows a parallel design, employs quadruple masking. Masking means some participants and/or investigators do not know which treatment group a participant is in, which helps reduce bias.
What are the primary outcomes being measured in NCT06634420?
The primary outcome measures are: Time-normalized number of Investigator-confirmed HAE attacks (From Week 5 through Week 28). These are the main endpoints researchers use to determine whether the treatment is effective.
Where is NCT06634420 being conducted?
This trial is being conducted at 20 sites, including Scottsdale, Arizona; San Diego, California; Santa Monica, California; Walnut Creek, California and 16 more sites (United States, Australia, Canada).
Where can I find official information about NCT06634420?
The official record for NCT06634420 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT06634420. This government database provides the most up-to-date and detailed information about the trial.
What is NCT06634420 testing in simple terms?
Tests the effectiveness and safety of NTLA-2002, a gene editing treatment, compared to a placebo. For adults with Hereditary Angioedema (HAE), a rare genetic condition causing swelling attacks.
Why is this trial significant?
This trial fills a critical gap by evaluating a potential gene therapy for HAE, a condition with limited treatment options. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.
What are the potential risks of participating in NCT06634420?
Potential side effects include injection site reactions and flu-like symptoms. Serious adverse events are monitored, but they are rare. As with any clinical trial, participants are closely monitored and can withdraw at any time.
Should I consider participating in NCT06634420?
Ask your doctor if you have a history of HAE and if you're eligible for this trial. Participation involves receiving an IV treatment and tracking your HAE attacks over 28 weeks. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.
What does NCT06634420 signal from an investment perspective?
The market for HAE treatments is growing, with limited competition, and there's a high probability of approval given the unmet need. This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.
What happens if the treatment in this trial doesn't work?
Participation involves receiving either NTLA-2002 or a placebo through IV, and tracking HAE attacks. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.
Related Conditions
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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.