EUCTR2020-005193-94-SE进行中（未招募）1 期
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients with C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 42
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
- •ALS-Specific Inclusion Criteria:
- •1. Diagnosis of ALS based on clinical manifestations.
- •2. ALS: Clinically diagnosed possible, laboratory supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology revised El Escorial criteria.
- •3. Patients receiving riluzole have been on a stable dose for a minimum of 30 days.
- •4. Patients on edaravone have received a minimum of 1 cycle (28 days).
- •5. Patients discontinuing riluzole or edaravone had the last dose administered =1 month prior to Screening.
- •FTD-Specific Inclusion Criteria:
- •6. FTD: Must have Global Clinical Dementia Rating – Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) score of 0.5 or 1.
- •7. FTD: Able to undergo periodic magnetic resonance imaging (MRI) of the brain. Patients with mixed phenotype (ALS and FTD) need not undergo MRI if their ALS symptoms prevent it.
- •Mixed Phenotype (ALS and FTD) Inclusion Criteria:
- •8. Patients who are mixed phenotype (ALS and FTD) must meet both the ALS-specific and FTD-specific criteria.
- •Inclusion Criteria Common to Both Diseases:
- •9. Patient must have the ability and be willing to provide written informed consent prior to any trial-related procedures.
- •10. Documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the C9orf72 gene.
- •11. Body mass index (BMI) =32 kg/m2.
- •12. Forced vital capacity (FVC) of >50% predicted.
- •13. Age of =18 and =80 years at Screening visit.
- •14. Willing and able to comply with scheduled visits, drug administration plan, laboratory tests, trial restrictions, and all trial procedures.
- •15. Willingness to practice highly effective contraception for the duration of the trial and for 5 months after the last dose of study drug if patients or their partners are of childbearing potential. Non-childbearing potential and highly effective methods of contraception are defined in the protocol. In addition, willingness to forego sperm or ova (egg) donation for the duration of the study and 5 months after last dose.
- •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 31
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 11
排除标准
- •Exclusion Criteria:
- •1. Clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the trial procedures, including, but not limited to:
- •a. Prior or ongoing medical conditions, including acute illness, within 28 days of Screening visit;
- •b. Clinically significant abnormality on laboratory testing at Screening, including, but not limited to:
- •- Renal insufficiency, which is defined as creatinine clearance <40 mL/min.
- •2. Positive hepatitis B surface antigen or hepatitis C antibody test.
- •3. Known to be positive for human immunodeficiency virus (HIV).
- •4. History of substance use disorder (except nicotine) within 6 months prior to the Screening Visit.
- •5. Significant cognitive impairment; or unstable psychiatric illness, including active psychosis, active suicidal ideation, recent suicide attempt, or untreated major depression, within the last 90 days, as determined by the Investigator. Mental status, psychiatric medical history, and eligibility for the study must be documented in the screening questionnaire.
- •6. Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the trial.
- •7. Clinically significant abnormality on Screening electrocardiogram (ECG), including, but not limited to, a confirmed QT interval using Fridericia’s correction method (QTcF) of =450 msec for males or =470 msec for females.
- •8. Bone, spine, bleeding (e.g. hemophilia, Von Willenbrand disease, liver disease), or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture.
- •9. Dementia due to a condition other than C9orf72-associated ALS or FTD, including, but not limited to, Alzheimer disease, Parkinson disease, dementia with Lewy bodies, Huntington's disease, or vascular dementia.
- •Prior or Concomitant Medications
- •10. Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, and phencyclidine at the Screening Visit.
- •11. Changes in nutritional or herbal supplements or concomitant medications within 1 month prior to Screening visit or plans to modify dose or regimen during the trial.
- •12. Received prior treatment with viral or cellular-based gene therapy.
- •13. Received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Received an investigational oligonucleotide, within the past 6 months or 5 half-lives of the drug, whichever is longer. Received prior treatment
- •with investigational product BIIB078.
- •14. Anticipates using antiplatelet or anticoagulant therapy during the course of the study. Patients who received antiplatelet or anticoagulant therapy must complete one of the
- •following washout periods before the Screening Visit:
- •a. A 7-day washout period for antiplatelet therapy,
- •b. A 1-day washout period for anticoagulants (except warfarin), or
- •c. A 5-day washout period for warfarin.
- •Trial Compliance
- •15. Implantable central nervous system (CNS) device that may interfere with ability to administer trial drug via lumbar puncture or undergo MRI scan.
- •16. Deemed to be at significant risk for suicidal behavior based on Investigator assessment and/or active suicidal ideation.
- •17. Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction, such as changes in pulse, b
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