EUCTR2021-003052-16-FR进行中（未招募）1 期
An Open-Label Phase I/II Dose Finding Study to evaluate the Safety, Tolerability, and Preliminary Efficacy of Striatal Administration of BV-101 in Adult Subjects with Early Manifest Huntington's Disease (HD)
Brainvectis0 个研究点目标入组 18 人开始时间: 2021年12月8日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 18
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. Male or Female subjects between ages 18 and 65 years (both inclusive) at time of consenting, able to provide Informed Consent and able to understand and comply with all study procedures.
- •2. Documented genetic confirmation of pathological CAG expansion in the huntingtin gene =40.
- •3. Early manifest HD as defined by a UHDRS total functional capacity (TFC) score of 9 to 13 and a diagnostic classification level (DCL) of 4, or a DCL of 3 if present with cognitive impairment and clear evidence of disease progression.
- •4. Striatal MRI volumes per hemisphere compatible with the local delivery of IMP: Putamen = 2.3 cm3 (per side); Caudate = 1.7 cm3 on Screening MRI.
- •5. All HD concomitant medications stable for at least 30 days prior to screening at the investigator’s discretion.
- •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 17
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •1. Prior or ongoing medical condition, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, would impact subject’s safety and compliance with the study procedures.
- •2. Metastatic neoplasms within the five years prior to screening.
- •3. Presence of clinically relevant immunologic, hematologic, hepatic, cardiac, or renal disease at the time of screening as per investigator’s clinical judgment.
- •4. History of or current active psychosis, confusional state, or violent behavior per the investigator’s judgement.
- •5. Cognitive impairment preventing the ability to understand and sign the informed consent as per the investigator’s judgement.
- •6. Current untreated and unstable depressive disorder or a serious mood disorder requiring hospitalization.
- •7. History of prior suicide attempt or imminent risk of self-harm based on investigator’s judgment or with a yes” answer on item 4 or 5 on the Columbia Suicide Severity Rating Scale (CSSRS).
- •8. Suspected or known history of alcohol and/or substance disorder within 12 months of screening as per the judgement of the investigator.
- •9. History of non-compliance with prescribed medications for HD management.
- •10. Patients with history of confirmed stroke, known intracranial neoplasms, vascular malformations, or intracranial hemorrhage.
- •11. Subjects not deemed suitable for the surgical procedure as per the Neurosurgeon’s judgment.
- •12. Positive for hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) at screening
- •13. Coagulopathy, use of antiplatelet or anticoagulant therapy or inability to temporarily stop any antithrombotic medication
- •14. Any infection or vaccination within 14 days prior to screening and within 14 days prior to the scheduled surgical IP dosing day.
- •15. Any history of gene therapy, cell transplantation or any other experimental brain surgery.
- •16.Any RNA or DNA targeted HD specific investigational agents such as antisense oligonucleotides within 6 months prior to screening.
- •17. Use of statins within 5 half-lives of baseline visit. If the subject is on a statin, he/she may transition to an alternative cholesterol lowering treatment, and baseline visit can occur after a duration of at least 5 half-lives after discontinuation of statin.
- •18. Contraindication to MRI such as claustrophobia, severe movement disorders or inability to lay flat that, in the investigator's judgment, precludes the subject's safe participation in and completion of the study. Subjects with a history of working with metal (using grinders, etc.) should have a safety check for residual metal fragments in their eyes to avoid damage from the magnetic fields.
- •19. Implanted devices such as stimulators, spinal rods, cardiac pacemaker that would interfere with MRI interpretation.
- •20. Subjects unable to tolerate or unwilling to undergo multiple lumbar punctures.
- •21. Participation in any clinical trial of an approved or non-approved investigational drug or intervention within 12 weeks or 5 half-lives whichever is longer prior to treatment.
- •22. Female subjects who are pregnant or breastfeeding or who may plan to become pregnant within 12 months of the expected/scheduled day of dosing.
- •23. Female subjects of childbearing potential unwilling to agree to contraception guidance detailed in section 9.4.2 for at least 12 months after the study intervention
- •24. Male subjects unwilling to agree to contraception guidance detailed in Section 9.4.2 for at least 6 months after the study interventi
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