NL-OMON52346撤回2 期
A Phase 2a Study of TPN-101 in Patients with C9ORF72 ALS/FTD (Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia) - TPN-101-C9-201
适应症
试验速览
- 阶段
- 2 期
- 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
- •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
- •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
- •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
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
- •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/*L; infection at
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