EUCTR2021-002251-11-ES进行中（未招募）1 期
A Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
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. Males or females = 18 years of age at the time of informed consent
- •2. Have documentation of a clinical genetic test demonstrating the presence of a confirmed repeat expansion in the C9orf72 gene from a CLIA certified laboratory
- •3. Body weight range of = 41 kg (90 lbs) to = 118 kg (260 lbs)
- •4. Score = 18 on the Mini-Mental State Exam (MMSE) at Screening
- •5. If female, must be postmenopausal (for at least 2 years), surgically sterilized (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), or agree to use highly effective methods of contraception from Screening through Week 52
- •6. If male, with a partner who is not postmenopausal (for at least 2 years) or surgically sterilized (bilateral tubal ligation, bilateral oophorectomy, or hysterectomy), the patient must agree that he and his partner will use highly effective methods of contraception from Screening through Week 52
- •7. Able to perform all protocol-specified assessments, including neuropsychological tests; and comply with taking study medication and the study visit schedule, as judged by the investigator
- •8. Have a reliable caregiver to accompany the patient to all study visits. Caregiver must be able to read, understand, and speak local language fluently to ensure comprehension of informed consent and informant-based assessments of the patient. Caregiver must also have frequent contact with patient (at least 3 hours per week at one time or at different times) and be willing to monitor the patient's health and concomitant medications throughout the study
- •9. Able to understand and provide written informed consent at Screening
- •10. Agree to allow data sharing across observational longitudinal and interventional studies using an encrypted global unique identifier (GUID) so that potential prior or future data on biomarkers and disease progression can be made accessible to the sponsor
- •11. Stable doses of all concomitant medications for 1 month prior to Screening (e.g., edaravone, riluzole, dextromethorphan/quinidine, psychotropic medications, cognitive enhancers, etc.)
- •For patients with ALS (with or without FTD):
- •12. Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria
- •13. Onset of weakness within 3 years prior to Screening
- •14. SVC = 60% of predicted normal adjusted for sex, age, and height (from the sitting position)
- •15. Able to perform reproducible pulmonary function tests
- •16. ALSFRS-R = 30 at Screening
- •For patients with FTD:
- •17. A gradual, progressive decline in behavior, language, or motor function consistent with C9orf72 hexanucleotide expansion-related syndrome such as behavioral variant FTD, primary progressive aphasia, or amnestic syndrome
- •18. CDR plus NACC FTLD global score of 0.5-2.0 at Screening
- •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 10
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 10
排除标准
- •1. Presence of other significant neurological or psychiatric disorders including (but not limited to) biomarker confirmed Alzheimer's disease; dementia with Lewy bodies; prion disease; Parkinson's disease; multiple sclerosis; a primary or severe psychotic disorder; severe bipolar or unipolar depression; prior history of suicidal thoughts or behavior that are believed to represent a current safety risk; seizure; brain tumor or other space-occupying lesion; history of stroke; or history of severe head injury within the past 20 years
- •2. History of significant brain abnormality, including, but not limited to, prior hemorrhage or infarct, cerebral contusion, encephalomalacia, aneurysm, vascular malformation, subdural hematoma, hydrocephalus, space-occupying lesion (e.g., abscess or brain tumor such as meningioma); symptoms or signs of elevated intracranial pressure, e.g., symptoms or history of head injury or abnormal funduscopic exam. If there is history or evidence on neurologic exam suggesting possible subdural hematoma (SDH), patients should be fully evaluated, including magnetic resonance imaging (MRI) if indicated, to exclude significant, new SDH
- •3. Active alcohol, drug abuse or substance abuse, or any other reason that makes it unlikely that the patient will comply with study procedures in the opinion of the investigator
- •4. Clinically significant findings on Screening laboratory testing, physical examination or vital signs that are not specific to ALS/FTD that could interfere with the conduct of the study, the interpretation of the data, or increase patient risk
- •5. Clinically significant intercurrent illness or medical condition (e.g., hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease) that would jeopardize the safety of the patient, limit participation, or compromise the interpretation of the data derived from the patient
- •6. History of HIV infection, hepatitis B or hepatitis C, or any active infection
- •7. History of cancer within 5 years of Screening, with the exception of fully excised non-melanoma skin cancers
- •8. Receipt of an investigational agent within 30 days or 5 half-lives prior to Screening, whichever is longer
- •9. Prior treatment with any monoclonal antibody within 6 months of Screening
- •10. Receipt of systemic corticosteroids within 30 days prior to Screening
- •11. Any vaccination within 30 days prior to study drug administration
- •12. Has smoked or used tobacco products within 6 months prior to study drug administration
- •13. Hypertension, defined as confirmed systolic blood pressure (SBP) > 170 mmHg and/or diastolic blood pressure (DBP) > 100 mmHg at Screening
- •14. Hypotension, defined as confirmed SBP < 90 mmHg and/or DBP < 60 mmHg at Screening
- •15. Any major surgery within 4 weeks of Screening
- •16. Females who are pregnant (positive pregnancy test at Screening or prior to administration of study drug), breastfeeding, or unable or unwilling to use highly effective methods of contraception throughout the study
- •17. Contraindication to undergoing a lumbar puncture (LP) including, but not limited to: inability to tolerate an appropriately flexed position for the time necessary to perform an LP; international normalized ratio (INR) > 1.4 or other coagulopathy; platelet count of < 120,000/µL; infection at the desired LP site; taking anti-platelet or anti-coagulant medication within 30 days of Screening (Note: aspirin is permitted); severe degenerative arthritis of the lumbar spine; suspected non-commu
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