EUCTR2021-001693-33-NL进行中（未招募）1 期
Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 55
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Subjects > 18 years of age at the time of signing the informed consent.
- •2. Documented diagnosis of HAE (Type I or II).
- •3. Investigator-confirmed attacks:
- •a. Subjects in Phase 1 must have an Investigator-confirmed and documented
- •historical HAE attack number of at least 3 during the previous 3 months
- •(90 days) from the start of screening. Phase 1 subjects may not have
- •received HAE prophylaxis during the 3 months (90 days) historical attack
- •b. Subjects in Phase 2 must have an Investigator-confirmed and documented
- •historical HAE attack number of at least 3 during the previous 3 months
- •(90 days) to enter the run-in period, and must have an
- •Investigator-confirmed and documented historical HAE attack number of
- •at least 2 during the 8-week (56-day) run-in-period to be eligible for
- •enrollment and randomization.
- •4. Subjects must have access to, and the ability to use, = 1 acute medication(s) to
- •treat angioedema attacks.
- •5. Subjects must meet the following laboratory criteria during Screening:
- •a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), total
- •bilirubin (see exception for Gilbert’s Syndrome below), and international
- •normalized ratio (INR) = upper limit of normal (ULN) range at
- •b. For subjects with a history of Gilbert’s Syndrome, total bilirubin
- •= 2 × ULN on screening evaluation.
- •c. Serum creatinine is < ULN, or, for subjects in whom serum creatinine is
- •above the ULN, they can be included if the estimated glomerular
- •filtration rate (eGFR) is > 45 mL/min/1.73 m2 as measured by the
- •Modification of Diet in Renal Disease equation at Screening.
- •d. Platelet count = 100,000 cells/mm3 at Screening.
- •e. Within reference range or Principal Investigator (PI)-determined
- •clinically non-significant partial thromboplastin time (aPTT),
- •prothrombin time (PT), fibrinogen and d-dimer levels at Screening.
- •6. Male subjects with partners of child-bearing potential must agree to using a
- •condom prior to Screening and for 90 days after study drug administration.
- •7. Male subjects must agree not to donate sperm for 90 days after study drug
- •administration. The time frame may be extended beyond the 90 days, if sperm
- •donation is contraindicated based on country-specific guidelines.
- •8. A female subject must be:
- •? Postmenopausal (defined as no menses for 12 months without an
- •alternative medical cause) prior to Screening. In addition, at least 2 high
- •follicle stimulating hormone (FSH) measurements in the postmenopausal
- •range may be used to confirm a postmenopausal state in women with less
- •than 12 months of amenorrhea and not using hormonal contraception or
- •hormonal replacement therapy; OR
- •? Surgically sterile (i.e., hysterectomy, bilateral salpingectomy, and
- •bilateral oophorectomy) at least 1 month prior to Screening.
- •9. Subjects must agree not to participate in another interventional study for the
- •duration of this trial.
- •10. Subjects must be capable of providing signed informed consent.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 45
- 另有 2 项未显示
排除标准
- •1. Use of long-term prophylaxis for HAE within 5 half-lives prior to the start of
- •Screening or during the Phase 1 historic attack period; see Section 10.2 for list
- •of prophylaxis agents, half-lives, and recommended washout period.
- •2. Use of ecallantide, attenuated androgens, or anti-fibrinolytics for HAE within
- •2 days prior to entering the start of Screening visit, or during the Phase 1
- •historic attack period, or during the Phase 2 run-in period.
- •3. Use of C1 esterase inhibitor (C1-INH) for HAE within 5 half-lives of the
- •agent before initiation of the Phase 2 run-in period; i.e., 24-hour washout is
- •required before starting the run-in period after the use of rabbit purified
- •C1-INH (ruconest), and 4-day washout is required before starting the run-in
- •period after the use of human-plasma-purified C1-INH (berinert). Note:
- •during the run-in period, C1-INH may be used to treat an acute HAE attack.
- •4. Concurrent diagnosis of any other type of recurrent angioedema, including
- •acquired or idiopathic angioedema.
- •5. Subjects who have known hypersensitivity to any lipid nanoparticles (LNP)
- •component or who have previously received LNP and experienced any
- •treatment-related clinically significant laboratory abnormalities or adverse
- •event listed below:
- •a. ALT or AST > 3 × ULN if baseline was normal or > 3 × baseline if
- •baseline was above normal.
- •b. INR, aPTT or d-dimer > 1.5 × ULN if baseline was normal or
- •> 1.5 × baseline if baseline was above normal.
- •c. Any LNP treatment-related adverse event classified as CTCAE
- •Grade 3 or higher.
- •d. Infusion-related reaction (IRR) to an LNP containing product
- •requiring treatment or discontinuation of infusion; NOTE: slowing of
- •the infusion rate to mitigate an IRR is not considered exclusionary.
- •e. Any LNP treatment-related adverse event which in the opinion of the
- •Investigator should be exclusionary.
- •6. Exposure to angiotensin-converting enzyme (ACE) inhibitors or any
- •estrogen-containing medications with systemic absorption within 90 days
- •prior to study drug administration.
- •7. Unable or unwilling to take the required pre-treatment medication regimen.
- •8. Antithrombotic therapy other than aspirin (e.g., warfarin, dabigatran,
- •apixaban) within 14 days prior to study drug administration.
- •9. History of thrombophilia, or positive genetic test for Factor V Leiden and/or
- •prothrombin 20210.
- •10. History of cirrhosis.
- •11. Known or suspected systemic viral, parasitic, or fungal infection including
- •coronavirus disease (COVID)-19 or received antibiotics for bacterial infection
- •within 14 days prior to Screening.
- •12. History of Hepatitis B or C infection or positive Hepatitis B surface antigen
- •(HbsAg) or Hepatitis C virus antibody (HCVAb) test at Screening.
- •13. History of positive human immunodeficiency virus (HIV) status.
- •14. Prior liver, heart, or other solid organ transplant or bone marrow transplant or
- •anticipated transplant within 1 year of Screening. Note: prior history of or
- •planned corneal transplant is not exclusionary.
- •15. Subject has a history of alcohol or drug abuse within 3 years prior to
- •16. Any condition, laboratory abnormality, psycho-social stressor,
- •pattern-of-behavior, or other reason that, in the Investigator’s opinion, could
- 另有 4 项未显示
研究者
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