NCT05683860终止1 期
A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 5
- 主要终点
- Safety: Number of patients with serious AEs (SAEs)
研究概览
简要总结
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, participants must have successfully completed Phase 1b/2a WVE-004-001 study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants successfully completed the Phase 1b/2a study with WVE-004, WVE-004-
- •Participant has the ability and is willing to provide informed consent prior to any study procedures. In instances where signed written informed consent is unable to be obtained it is acceptable for the participant to provide consent with legally authorized representative signing on the participant's behalf.
- •In the opinion of the Investigator, the participant is able to tolerate all study procedures, is willing to comply with all other protocol requirements, and tolerated study drug in the parent study.
- •Participant is willing to practice highly effective contraception for the duration of the study and for 5 months after the last dose of study drug if the participant or their partner are of childbearing potential. In addition, willingness to forego sperm or ova (egg) donation for the duration of the study and 5 months after completion of the study.
- •Participant has identified a study partner(s) for the duration of the study.
排除标准
- •Participant has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the participant unsuitable for participation in and/or completion of the trial procedures.
- •Participant received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Participant received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer.
- •Participant has a positive hepatitis B surface antigen or hepatitis C antibody test.
- •Participant who are pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening visit, or plans to become pregnant during the trial.
- •Participants deemed to be at significant risk for suicidal behavior, based on Investigator assessment and/or active suicidal ideation.
- •Participant has a bone, spine, bleeding (e.g., hemophilia, Von Willebrand disease, or liver disease), or other disorder that exposes the participant to a risk of injury or unsuccessful lumbar puncture (LP).
- •Participant received prior treatment with viral or cellular-based gene therapy.
- •Participant anticipates using antiplatelet or anticoagulant therapy during the course of the study. Participants who received antiplatelet or anticoagulant therapy must complete a washout period before the Screening visit.
- •Participant was noncompliant, in the opinion of the Investigator or Sponsor, when participating in study WVE-004-
- •Participant is directly or indirectly involved in the conduct and administration of this trial as an Investigator, sub-investigator, trial coordinator, or other trial staff member, or the participant is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the trial.
研究组 & 干预措施
WVE-004 (Dose A)
Experimental
干预措施: WVE-004 10 mg Q12W (Drug)
结局指标
主要结局
Safety: Number of patients with serious AEs (SAEs)
时间窗: Day 1 to Week 120 (end of study)
Safety: Number of patients who withdraw due to AEs
时间窗: Day 1 to Week 120 (end of study)
Safety: Number of patients with adverse events (AEs)
时间窗: Day 1 to Week 120 (end of study)
Safety: Number of patients with a severe AE
时间窗: Day 1 to Week 120 (end of study)
Number of Occurrences of Participants With Adverse Events (AEs) Severe AEs, Serious Adverse Events (SAEs), and Withdrawals Due to AEs
时间窗: Day 1 to Week 24 (early termination cutoff)
A treatment-emergent adverse event (TEAE) is defined as any event not present before exposure to study treatment or any event already present that worsens in either intensity or frequency after exposure to study treatment.
次要结局
- Change From Baseline in Clinical Dementia Rating Plus National Alzheimer's Coordinating Center Frontotemporal Lobar Degeneration (CDR Plus NACC FTLD)(Day 1 to Week 120 (end of study))
- Change From Baseline in ALS Functional Rating Scale-Revised (ALSFRS-R)(Day 1 to Week 120 (end of study))
- Change From Baseline in Handheld Dynamometry (HHD)(Day 1 to Week 120 (end of study))
- Change From Baseline in Pulmonary Function Testing (Forced Vital Capacity (FVC))(Day 1 to Week 120 (end of study))
- Change From Baseline in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ)-5(Day 1 to Week 120 (end of study))
- Change From Baseline in the Concentration of Poly-glycine-proline (Poly-GP) Levels in the Cerebrospinal Fluid (CSF)(From Baseline to Week 12)
研究者
研究点 (5)
Loading locations...
相似试验
撤回
2 期
Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency AnemiaIron Deficiency, AnaemiaNCT04968379American Regent, Inc.
已完成
2 期
A Study to Extend Efficacy and Safety of SHR-1819 Injection in Adult Patients With Moderate to Severe Atopic DermatitisModerate to Severe Atopic DermatitisNCT06012812Shanghai Hengrui Pharmaceutical Co., Ltd.79
已完成
1 期
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)Acute Intermittent PorphyriaNCT02949830Alnylam Pharmaceuticals16
招募中
1 期
OriCAR-017 Chimeric Antigen Receptor (CAR) Modified T Cells for the Treatment of R/RMMRelapsed and/or Refractory Multiple MyelomaNCT06182696OriCell Therapeutics Co., Ltd.83
终止
2 期
Open Label Extension of Efgartigimod in Adults With Post-COVID-19 POTSPost-COVID Postural Orthostatic Tachycardia Syndrome Postural Orthostatic Tachycardia SyndromeNCT05918978argenx33