NL-OMON53802撤回不适用
A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) - AVB-PGRN-001
适应症
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •Subjects will be eligible to be included in the study if all of the following
- •criteria apply:
- •1. Are male or female, 30 to 75 years of age, inclusive, at Screening;
- •2. Are carriers of a pathogenic GRN mutation as confirmed by a Sponsor approved
- •genetic test;
- •3. Have EITHER symptomatic FTD OR pre-symptomatic carrier of a pathogenic GRN
- •mutation at risk of conversion based on plasma NfL >20 pg/mL;
- •4. If symptomatic, presence of 1 or more of the criteria for diagnosis of
- •possible behavioral variant FTD or PPA;
- •5. Have an identified, informed study partner able to support the subject for
- •the duration of the study;
- •6. For women of childbearing potential, must have a negative serum pregnancy
- •test at Screening, a negative urine dipstick, and not be breastfeeding within 2
- •weeks prior to treatment;
- •7. For sexually active subjects, must agree to use a highly effective barrier
- •method of contraception until at least 3 consecutive negative blood (or semen
- •for male subjects, where possible) vector shedding samples are collected at
- •least 1 week apart;
- •8. Able and willing to comply with all procedures and the study visit schedule
- •as outlined in the protocol; and
- •9. The subject and/or legally authorized representative is able and willing to
- •give written informed consent prior to study participation. If a subject lacks
- •the capacity to consent in the Investigator*s opinion, the subject*s assent
- •should be obtained, if required in accordance with local laws, regulations, and
- •customs, plus the written informed consent of a legal representative should be
- •obtained. In countries where local laws, regulations, and customs do not permit
- •subjects who lack capacity to consent to participate in this study, they will
- •not be enrolled.
排除标准
- •Subjects will be excluded from the study if any of the following criteria apply:
- •1. Have a classification of the mutation in the GRN gene as *not pathogenic,*
- •*likely benign variant,* or *benign variant* in the Alzheimer*s Disease and
- •Frontotemporal Dementia Mutation Database;
- •2. Have severe dementia, defined as Clinical Dementia Rating (CDR)+National
- •Alzheimer*s Coordinating Center (NACC) frontotemporal lobar degeneration (FTLD)
- •global score of 3.0, that precludes the ability to comply with study procedures
- •and/or poses unacceptable safety
- •risk to the subject;
- •3. Have any concurrent disease that may cause cognitive impairment unrelated to
- •mutations in the GRN gene, such as other causes of dementia, neurosyphilis,
- •hydrocephalus, stroke, small vessel ischemic disease, uncontrolled
- •hypothyroidism, or vitamin B12 deficiency;
- •4. Have a clinically significant abnormality on MRI at Screening considered to
- •be a contraindication to intrathalamic infusion;
- •5. Have a surgically significant pattern of brain atrophy on MRI at Screening
- •that interferes with planned neurosurgical trajectory;
- •6. Have had previous treatment with any gene or cell therapy;
- •7. Have had previous treatment with any investigational medicinal product
- •within 60 days or 5 half-lives (whichever is longer) prior to study drug
- •8. Have had a concomitant disease, any clinically significant laboratory
- •abnormality, or treatment which, in the opinion of the Investigator, may pose
- •an unacceptable safety risk to the subject or interfere with study conduct or
- •the subject's ability to comply with study procedures;
- •9. Have a malignancy within 5 years of Screening, except for basal or squamous
- •cell carcinoma of the skin or carcinoma in situ of the cervix that has been
- •successfully treated;
- •10. Have any contraindications to MRI as per local guidelines;
- •11. Have any contraindications to gadolinium-based contrast agents per local
- •guidelines;
- •12. Have any contraindications to general anesthesia for a period of up to 10
- •hours and/or cardiopulmonary disorders that would result in higher American
- •Society of Anesthesiology risk classification;
- •13. Have any contraindications to lumbar puncture as per local guidelines;
- •14. Have been hospitalized for any major medical or surgical procedure
- •involving general anesthesia within 12 weeks of Screening or planned procedure
- •during the study;
- •15. Are using anticoagulants at Screening, or will have an anticipated need
- •during the period of treatment. Antiplatelet therapies are acceptable
- •concomitant medication if they can be stopped at least 48 hours prior to
- •16. Have a history of previous serious or recent Coronavirus Disease 2019
- •(COVID-19) as defined by (1) any history of hospitalization for severe illness
- •at any time, (2) any history of significant respiratory symptoms at any time,
- •or (3) any pre-symptomatic or mildly symptomatic
- •COVID-19 positivity within 12 weeks prior to planned treatment;
- •17. Have a positive drug screen for drugs of abuse;
- •18. Have a history of substance abuse disorder;
- •19. Have the presence of an implanted deep brain stimulation device,
- •ventriculoperitoneal or other cerebrospinal fluid shunt, or other implanted
- •20. Have evidence of suicide risk, as assessed by the Columbia-Suicide Severity
- 另有 1 项未显示
研究者
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