EUCTR2019-000138-20-IT进行中（未招募）1 期
A Phase 2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AL001 in Heterozygous Carriers of Granulin or C9ORF72 Mutations Causative of Frontotemporal Dementia -
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
- 32
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
- •Participants must meet all of the key inclusion criteria specific to their applicable participant category. These bullet points are some of the important criteria within each category:
- •1) Participant completed Study AL001-1 through the Day 57 visit and did not experience AEs that the investigator deems would prevent safe participation in Study AL001-2.
- •2) Participant meets 1 or more of the 6 behavioral/cognitive symptoms required for a diagnosis of possible behavioral variant frontotemporal dementia (bvFTD; Rascovsky 2011) or has diagnosis of primary progressive aphasia (PPA; Gorno Tempini 2011)
- •3) Participant completed Study AL001-1 through the Day 43 visit and did not experience AEs that the investigator deems would prevent safe participation in Study AL001-2.
- •4) Participant is a carrier of a loss of function GRN mutation causative of FTD and known their mutation status
- •5) Participant has a CDR® plus NACC global score of 0.5, 1, or 2; and 1 or more of the 6 behavioral/cognitive symptoms required for a diagnosis of possible bvFTD (Rascovsky 2011) or a diagnosis of PPA (Gorno Tempini 2011)
- •In addition, this is the list of the important inclusion criteria:
- •Participants are 18 to 85 years of age.
- •At screening, female participants must be nonpregnant and nonlactating, and at least one of the following conditions must apply:
- •- Participant is 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]).
- •- Participant is a WOCBP and using an acceptable contraceptive method from screening until 8 weeks after the last dose of study drug. Acceptable contraception is defined as using hormonal contraceptives 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, in accordance with the lifestyle of the participant, is acceptable.
- •- A WOCBP must have a serum pregnancy test conducted at screening. Additional requirements for pregnancy testing during and after study intervention are located in the Schedule of Assessments (Table 14-1 in the protocol).
- •Male participants, if not surgically sterilized, must agree to use acceptable contraception and not donate sperm from Day 1 until 8 weeks after the last dose of study drug.Acceptable contraception for the male patient (and his female partner) is defined as using hormonal contraceptives 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, in accordance with the lifestyle of the participant, is acceptable.
- •Participant is in good physical health on the basis of no clinically significant findings from medical history, PEs, laboratory tests, ECGs, and vital signs.
- •Participant agrees not to donate blood or blood products for transfusion for the duration of the study and for 1 year after final dose of study drug.
- •Participant is willing and has the ability to comply with the study protocol requirements, in the opinion of the investigator.
- •Participant is willing and able to give informed consent. If the study participant is not competent, a legally authorized representative must provide informed consent on their behalf, and the participant must provide assent, in accordance wit
排除标准
- •Participant has a known history of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric, human, or humanized antibodies or fusion proteins.
- •Participant has history of substance use disorder (drug or alcohol) within the past 2 years, with the exception of nicotine, as defined by the Diagnostic and Statistical Manual of Mental Disorders, fifth edition criteria (American Psychiatric Association 2013).
- •Participant has donated or lost more than 100 mL of blood within 30 days prior to Day 1.
- •Participant has had a blood transfusion within 30 days prior to screening.
- •Participant has had clinically significant and/or acute illness within 5 days prior to drug administration that may affect safety assessments.
- •Participant had surgery, hospitalization, or clinically significant infection requiring oral or IV antibiotics during the 30 days prior to screening.
- •Participant has planned procedure or surgery during the study that would interfere with the ability to perform study assessments.
- •Participant has past history of seizures, with the exception of childhood febrile seizures.
- •Participant has clinically, significant systemic immunocompromised condition because of continuing effects of immune suppressing medication.
- •Participant has major depressive disorder or history of schizophrenia, schizoaffective disorder, or bipolar disorder.
- •Participant has history of cancer
- •Participant has history or presence of intracranial tumor that is clinically relevant.
- •Participant has any clinically significant medical condition or laboratory abnormality that precludes the participant’s safe participation in and completion of the study.
- •Participant is positive for hepatitis B surface antigen, hepatitis C virus antibodies, or human immunodeficiency virus 1 and 2 antibodies or antigen, or history of spirochetal infection of the CNS.
- •Participant has significant kidney disease as indicated by a screening creatinine clearance <30 mL/min as calculated by the central laboratory using the Cockcroft Gault formula, which remains <30 mL/min if retested.
- •Participant has 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, which remains above either of these limits if retested or other abnormalities in synthetic function that are clinically significant.
- •The participant has, within the last 2 years, had unstable or clinically significant cardiovascular disease.
- •Participant has uncontrolled hypertension.
- •Participant has history or presence of an abnormal ECG that is clinically significant including complete left bundle branch block, second or third degree heart atrioventricular block, or evidence of prior acute or subacute myocardial infarction or ischemia.
- •Participant has QT interval corrected using Fridericia formula (QTcF) >450 ms for male participants and >470 ms for female participants.
- •Participant has history of ventricular dysrhythmias or risk factors for ventricular dysrhythmias such as structural heart disease, coronary heart disease, clinically significant electrolyte abnormalities, or family history of sudden unexplained death or long QT syndrome.
- •Participant has contraindication to lumbar dural puncture.
- •Participant has dementia or a milder, symptomatic syndrome (eg, mild cognitive impairment, mild behavioral impairment, or mild motor impair
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