EUCTR2021-002251-11-DE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)
适应症
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 hexanucleotide repeat expansion (HRE) in the C9orf72 gene from a laboratory using an assay that has the relevant mark of conformity (United Kingdom Conformity Assessed [UKCA], Conformitè Europëenne [CE], or CE United Kingdom Northern Ireland [UKNI]) or Clinical Laboratory Improvement Amendments (CLIA) certification in the United States (Note: The HRE in the C9orf72 gene is present in about 10-15% of patients with ALS or FTD. Therefore, confirmation of this mutation should be part of the pre-screening for inclusion in the study. Patients who are not already known to have this mutation should not be screened for inclusion, i.e., genetic testing for this mutation is not a part of the study procedures.)
- •3. 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, e.g., combined (estrogen and progestogen containing) or progestogen-only hormonal
- •contraception associated with inhibition of ovulation; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomized partner (provided that the partner is the sole sexual partner of the women of child-bearing potential [WOCBP] trial participant and that the vasectomized partner has received medical assessment of the surgical success); or sexual abstinence (defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments), from Screening through 3 months after the last dose of the study medication
- •4. 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, e.g., combined (estrogen and progestogen containing) or progestogen-only hormonal contraception associated with inhibition of ovulation; IUD; IUS; bilateral tubal occlusion; vasectomy (provided that he has received medical assessment of the surgical success); or sexual abstinence (defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments), from Screening through 3 months after the last dose of the study
- •5. 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
- •6. Have a reliable caregiver/informant to accompany the patient to all study visits. Caregiver/informant 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/informant 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
- •7. Able to understand and provide written informed consent at Screening, or has a legally authorized representation (LAR) who can provide such consent
- •8. Patient or LAR agree to allow data sharing across observational longitudinal and interventional studies using an encrypted global unique iden
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; progressive supranuclear
- •palsy; Parkinson's disease; multiple sclerosis; other causes of
- •neuromuscular weakness; a
- •primary or severe psychotic disorder; severe bipolar or unipolar
- •2. Prior history of suicidal thoughts or behavior that are believed to
- •represent a current safety risk, or a Yes response to Questions 4 or 5
- •on the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening
- •3. History of clinically significant brain abnormality, including, but not
- •limited to, stroke due to hemorrhage or infarction; cerebral contusion;
- •encephalomalacia; aneurysm; vascular malformation (angiomas or other
- •benign vascular malformations that are judged by the investigator to be
- •stable and not clinically significant are not exclusionary); hydrocephalus;
- •epilepsy; space-occupying lesion (e.g., abscess or brain tumor); severe
- •head injury within the past 20 years; 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
- •4. Active alcohol, drug, or substance abuse/dependence, or any other
- •reason that makes it unlikely that the patient will comply with study
- •procedures in the opinion of the investigator
- •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. Tracheostomy or diaphragmatic pacing
- •7. Autoimmune disease requiring treatment or management (quiescent
- •rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are
- •acceptable)
- •8. History of human immunodeficiency virus (HIV), hepatitis B, or any
- •active infection during Screening, unless the patient will have been
- •symptom-free for at least 30 days prior to randomization. Treated
- •hepatitis C with no laboratory evidence of active disease and liver
- •enzymes <2 × upper limit of normal (ULN) is allowed
- •9. History of cancer within 5 years of Screening, with the exception of
- •fully treated non-melanoma skin cancers
- •10. Receipt of an investigational agent within 30 days or 5 half-lives
- •prior to Screening, whichever is longer
- •11. Treatment with an immunomodulator within 6 months of Screening
- •12. Receipt of systemic corticosteroids within 30 days prior to Screening
- •13. Any vaccination within 30 days prior to randomization
- •14. Hypertension, defined as confirmed systolic blood pressure (SBP) >
- •170 mmHg and/or diastolic blood pressure (DBP) > 100 mmHg at Screening
- •15. Hypotension, defined as confirmed SBP < 90 mmHg and/or DBP < 60
- •mmHg at Screening
- •16. Any major surgery within 4 weeks of Screening or a planned major
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