NCT06634420进行中（未招募）3 期
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)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 56
- 主要终点
- Time-normalized number of Investigator-confirmed HAE attacks
研究概览
简要总结
This Phase 3 study aims to evaluate the efficacy and safety of NTLA-2002 compared to placebo in participants with HAE.
详细描述
This is a multinational, multicenter, double-blind, placebo-controlled study in which approximately 60 participants will be randomized in a 2:1 ratio to receive a single IV infusion of NTLA-2002 or placebo. After the Primary Observation Period (Week 1 through Week 28), participants will have the option to receive a blinded, single IV infusion of the opposite treatment. Following the Primary Observation Period, participants will enter the Long-Term Observation Period (76 weeks), for a total of 104 weeks. Including the Screening and Run-In Period, prior to the first blinded dosing, the total study duration is approximately 28 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥16 years
- •Clinical history consistent with HAE-C1INH-Type 1 or -Type 2
- •Ability to provide evidence of HAE attacks (confirmed by the Investigator) to meet the screening requirement
- •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.
- •Must have access to, and the ability to use, on-demand medication(s) to treat potential angioedema attacks
- •Adequate chemistry and hematology measures at screening
- •Must agree not to participate in another interventional study for the duration of this trial.
- •Must be capable of providing signed informed consent. Participants 16 to < 18 years of age, whose legal guardian provides informed consent, must provide assent.
- •Must agree to follow contraception requirements
排除标准
- •Concurrent diagnosis of any other type of recurrent angioedema or HAE with normal C1-INH
- •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 subject.
- •Unwilling to comply with study procedures.
研究组 & 干预措施
Arm B: Placebo
Placebo Comparator
Arm B: Placebo (saline; single IV infusion)
干预措施: Normal Saline IV Administration (Biological)
Arm A: NTLA-2002
Active Comparator
Arm A: NTLA-2002 (50 mg; single IV infusion)
干预措施: NTLA-2002 (Biological)
结局指标
主要结局
Time-normalized number of Investigator-confirmed HAE attacks
时间窗: From Week 5 through Week 28
次要结局
- Time-normalized number of Investigator-confirmed HAE attacks requiring on-demand treatment(from Week 1 through Week 28)
- Time-normalized number of moderate or severe Investigator-confirmed HAE attacks(From Week 1 through Week 28)
- Investigator-confirmed HAE attack-free status(From Week 1 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)
- Time-normalized number of Investigator-confirmed HAE attacks(From Week 1 through Week 28)
- Reduction from baseline of at least 50%, 70%, and 90% in time-normalized number of Investigator-confirmed HAE attacks(From Week 1 through Week 28)
- Long-term prophylaxis-free status(From Week 5 through Week 104)
- HAE treatment-free status, defined as no usage of long-term prophylaxis or on-demand medication(From Week 5 through Week 104)
- Complete responder status, defined as zero Investigator-confirmed attacks with no long-term prophylaxis(From Week 5 through Week 104)
- 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(From Week 33 through Week 104 and from Screening through Week 28)
- 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)
研究者
研究点 (56)
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相关资讯
NTLA-2002 Demonstrates Significant Reduction in Angioedema Attacks via CRISPR-Cas9 Gene Editing- A single dose of NTLA-2002, an investigational CRISPR-Cas9 gene-editing therapy, significantly reduced angioedema attacks in patients with hereditary angioedema (HAE).
- The Phase 2 trial data showed a dose-related reduction in plasma kallikrein levels, with the 50-mg dose reducing kallikrein by 86% from baseline.
- A substantial portion of participants, particularly in the 50-mg group (73%), remained attack-free throughout the 16-week observation period without needing additional intervention.
- NTLA-2002 was generally well-tolerated, with mostly mild-to-moderate adverse events, supporting its further investigation in a Phase 3 trial.last yearPhase 3 Trial of NTLA-2002 for Hereditary Angioedema Now Recruiting- A Phase 3 clinical trial (NCT06634420) is actively recruiting adult participants with Hereditary Angioedema (HAE) to evaluate the efficacy and safety of NTLA-2002.
- The study is a multinational, multicenter, double-blind, placebo-controlled trial randomizing approximately 60 participants in a 2:1 ratio to receive NTLA-2002 or placebo.
- Participants will receive a single IV infusion of NTLA-2002 or placebo, followed by a Primary Observation Period (Week 1-28) and a Long-Term Observation Period (76 weeks), totaling 104 weeks.
- The trial aims to assess the impact of NTLA-2002 on HAE, with a total study duration of approximately 28 months, including screening and run-in periods.last yearNTLA-2002 Shows Promise as Functional Cure for Hereditary Angioedema in Phase 2 Trial- Phase 2 trial results show NTLA-2002 significantly reduces swelling attacks in hereditary angioedema (HAE) patients, with up to 80% attack reduction at the 50 mg dose.
- A substantial portion of patients receiving NTLA-2002 experienced complete attack-free periods, with some remaining attack-free through the latest assessments.
- NTLA-2002 was generally well-tolerated, with no serious side effects reported, suggesting a favorable safety profile for this gene-editing therapy.
- Intellia Therapeutics has initiated a Phase 3 trial based on these positive results, potentially redefining the treatment paradigm for HAE.last yearIntellia Therapeutics Initiates Phase 3 Trial of NTLA-2002 for Hereditary Angioedema- Intellia Therapeutics has dosed the first patient in its Phase 3 HAELO trial evaluating NTLA-2002 for hereditary angioedema (HAE).
- The HAELO trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE.
- NTLA-2002, a single-dose CRISPR-based therapy, targets the _KLKB1_ gene to reduce plasma kallikrein activity and prevent HAE attacks.
- Intellia anticipates completing enrollment in the second half of 2025 and plans for a potential U.S. launch in 2027, pending regulatory approval.last year