EUCTR2020-004556-15-DK进行中（未招募）1 期
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington’s Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 54
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. Documented ability to understand the written study ICF(s) and consent, and has provided signed written informed consent prior to any study procedures.
- •2. Ambulatory male or female
- •3. Age =25 to =60 years old
- •4. Body mass index (BMI) =32 kg/m2
- •5. Documented CAG triplet repeats =36 in the HTT gene
- •6. Documented heterozygosity at SNP3
- •7. Documented presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
- •8. Clinical diagnostic motor features of HD, defined as UHDRS Diagnostic Confidence Score = 4
- •9. UHDRS Total Functional Capacity (TFC) scores =9 and =13
- •10. In the opinion of the Investigator, the patient is able to tolerate all study procedures, and is willing to comply with all other protocol requirements.
- •11. Willingness to practice highly effective contraception for the duration of the study, and for 5 months (i.e., 5 elimination half-lives) 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
- •forego sperm or ova (egg) donation for the duration of the study and 5 months after completion of the study.
- •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 54
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
- •2. Positive for Hepatitis B virus (HBV) or Hepatitis C virus (HCV).
- •3. Known to be positive for human immunodeficiency virus (HIV).
- •4. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
- •5. Previously received tominersen.
- •6. Received prior treatment with viral or cellular-based gene therapy.
- •7. Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:
- •whichever is longer, with the exception of the following:
- •a. Received WVE-120101 within the last 3 months (i.e., 5 half-lives); or
- •b. Received WVE-120102 within the last 3 months (i.e., 5 half-lives)
- •8. Implantable central nervous system device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan.
- •9. History of substance abuse disorder (except nicotine) within 6 months prior to the Screening Visit.
- •10. Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit.
- •11. Started or changed dose for concomitant medication for the treatment of HD symptoms or psychiatric disorders within 30 days prior to the Screening Visit (concomitant medications that have been administered on a stable regimen for =30 days are permitted).
- •12. Pregnant (as determined by a serum pregnancy test) or breast feeding at the Screening Visit, or plans to become pregnant during the course of the study.
- •13. Clinically significant laboratory abnormality at Screening.
- •14. Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) =450 msec for males or =470 msec for females.
- •15. Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the patient's safe participation in the study or would interfere with the study assessments. Mental status, psychiatric medical history, and eligibility for the study must be documented in the screening questionnaire.
- •16. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture.
- •17. Inability to undergo brain MRI (with or without sedation).
- •18. Deemed to be at significant risk for suicidal behavior based on any of the following criteria:
- •a. The opinion of the Investigator; or
- •b. Answers yes to Actual Suicide Attempts or Suicidal Behaviors in the Suicidal Behaviors section of the Columbia-Suicide Severity Rating Scale (C-SSRS) with reference to a 2 year period prior to the Screening Visit; or
- •c. Answers yes on any items in the Suicidal Ideation section of the C-SSRS with reference to a 6-month period prior to the Screening Visit; or
- •d. Answers yes on any items in the Suicidal Ideation section of the C-SSRS at the Baseline Visit since the last visit (Screening Visit).
- •19. Involved directly or indirectly in the conduct and administration of this study as an Investigator, sub-investigator, study coo
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