An Open-Label Study to Evaluate Safety of long-term AL001 dosing in FTD
- Conditions
- Frontotemporal DementiaMedDRA version: 21.1Level: PTClassification code 10068968Term: Frontotemporal dementiaSystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-000138-20-GB
- Lead Sponsor
- Alector Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 32
Participants must meet all of the criteria specific to their applicable participant 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 knows 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 ( 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 participant (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 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 with the local regulations, guidelines, and institutional review board (IRB) or independent ethics committee (IEC)
•Participant has availability of a person (study partner”) who has frequent and sufficient contact with the participant (at least 5 hours per week of in person contac
• 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 currently has or has had an acute illness or infection that requires oral or IV antibiotics within 30 days prior to study drug administration that may affect safety assessments.
•Participant has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise their ability to comply with the protocol required testing or procedures or compromise the participant's well-being, safety, or clinical interpretability.
•Participant has had any surgery or hospitalization during the 30 days prior to study drug administration
• Participant has history of cancer
• Participant has history or presence of intracranial tumor that is clinically relevant (e.g. glioma, cerebral metastasis).
• 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.
•Participant has had unstable or clinically significant cardiovascular disease (e.g., myocardial infarction, angina pectoris, New York Heart Association Class III or more cardiac failure) within the last 2 years.
• 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method