EUCTR2019-004066-18-NL进行中（未招募）1 期
A Phase 3, Multicenter, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk For or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Alector Inc.
- 入组人数
- 180
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. Is a known carrier of a heterozygous loss-of-function GRN mutation causative of FTD with a global CDR® plus NACC FTLD score of 0 to 2, and:
- •A CDR® plus NACC FTLD-SB score =0.5 with an elevated level of serum NfL, or
- •A CDR® plus NACC FTLD-SB score of >0.5 with 1 or more of the 6 behavioral/cognitive symptoms required for a diagnosis of possible bvFTD
- •(Rascovsky 2011), or a diagnosis of PPA
- •2. Age 25 to 85 years, inclusive, at Screening
- •3. At Screening, women must be nonpregnant and nonlactating, and one of the following conditions must apply:
- •a. Not a woman of childbearing potential (WOCBP) (either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1-year postmenopausal [amenorrhea duration of 12 consecutive months with no identified cause other than menopause]).
- •b. Is a WOCBP and agrees to use an acceptable contraceptive method from Screening until 10 weeks after the last dose of study treatment. Acceptable contraception is defined as using hormonal contraceptives (e.g. combined oral contraceptive pill) or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom. In addition, total abstinence, if in accordance with the lifestyle of the participant, is acceptable.
- •c. A WOCBP must have a serum pregnancy test conducted at screening. Additional requirements for pregnancy testing during and after study intervention are described in the Schedules of Assessments.
- •4. Men must agree to use acceptable contraception and not donate sperm from Day 0 until 10 weeks after the last dose of study treatment. Acceptable contraception for the male participant when having sexual intercourse with a WOCBP who is not currently pregnant is defined as using a condom. In addition, WOCBP partners must use hormonal contraceptives (e.g. combined oral contraceptive pill) or an intrauterine device.
- •5. Agrees not to donate blood or blood products for transfusion for the duration of the study and for 1 year after the final dose of study treatment.
- •6. Willing to and can comply with the study protocol requirements, in the opinion of the investigator.
- •7. Willing and able to give informed consent. If the patient is not competent, a legally authorized representative must provide informed consent on their behalf, and the patient must provide assent, in accordance with local regulations, guidelines, and IRB or IEC.
- •8. Patient has the availability of a person (study partner”) who has frequent and sufficient contact with the patient (at least 5 hours per week of in-person contact) and who can provide accurate information to the study site regarding the patient's behavior, cognitive, and functional abilities, as well as their health, throughout the study. Requirements for the study partner include:
- •a. Willing and able to provide informed consent to participate in the study as a study partner.
- •b. The study partner must have sufficient cognitive capacity to accurately report upon the participant’s behavior, cognitive, and functional abilities, in the opinion of the investigator.
- •c. The study partner should be in sufficiently good general health to have a high likelihood of maintaining the same level of interaction with the participant and participation in study procedures throughout the study duration.
- •d. The same study partner should participate throughout the duration of Part 1 of the study. If a change in study partner is necessary, the medical monitor must
排除标准
- •1. Dementia due to a condition other than FTD including, but not limited to, Alzheimer’s disease, Parkinson’s disease, Parkinsonism, REM behavior disorder, dementia with Lewy bodies, Huntington disease, or vascular dementia2.Known mutation causative of neurodegenerative disorder(s) other than heterozygous loss-of-function GRN mutations causative of FTD.3.Known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.4.Signs or symptoms of progressive supranuclear palsy or bulbar dysfunction, such as postural instability, eye problems, and swallowing difficulties5. History of moderate or severe substance use disorder within the past 2 years, with the exception of nicotine, as defined by the Diagnostic and Statistical Manual of Mental Disorders, fifth edition criteria6.Clinically significant vitamin B12 or folate deficiency (if treated, must be on a stable regimen for at least 3 months prior to first study treatment administration)7.Untreated hypothyroidism8.Insufficiently controlled diabetes mellitus9.Any surgery (major or emergent) or hospitalization within 30 days prior to first study treatment administration10.History of cancer except if: a.Is considered likely to be cured or in remission for at least 12M,b. Is not being actively treated and, in the opinion of the PI, is not likely to require treatment witin 3 Y, c. Is considered to have low probability of recurrence, d. For prostate cancer, has not had significant progression within the pastY, and is stable and adequately controlled,e. For localized skin basal or squamous cell carcinoma, participants should continue with screening and seek treatment for the skin carcinoma11. Positive for hepatitis B surface antigen, human immunodeficiency virus-1 or -2 antibodies or antigen, or history of spirochetal infection of the CNS. Participants with a positive hepatitis C virus antibody will be allowed if hepatitis C RNA is negative. 12. Significant kidney disease as indicated by either of the following: g. Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m2, according to the re-expressed abbreviated (four-variable) MDRD Study equationb.Creatinine =2 mg/dL.13. Impaired hepatic function as indicated by screening aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =2.5 the upper limit of normal (ULN), or total bilirubin =2.0 × ULN. Note: Participants with Gilbert's syndrome are eligible to participate if approved by the Medical Monitor. 14. Hematologic abnormalities as indicated by hemoglobin =10 g/dL; white blood cells (WBC) =3 000/mm3; absolute neutrophil count = 1,000/mm3; or platelet count =150,000/mm3.15. Participants with hypertension who are not adequately and stably controlled as per the ACC/AHA 16. History or presence of an abnormal ECG that is clinically significant, or evidence of acute or subacute myocardial infarction or ischemia17. History of or concurrent clinically significant cardiovascular disease such as but not limited to myocardial infarction, angina pectoris, New York Heart Associating Class III or IV cardiac failure ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease If the condition is stable per cardiologist, then the patient can enrol at PI discretion18.Clinically significant electrolyte abnormalities 19. For participants who consent to lumbar puncture, participant has contraindication to lumbar dura
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