ISRCTN97180769进行中（未招募）2 期
A Phase 2a, randomized, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of PTC518 in subjects with Huntington's disease
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 252
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •Current inclusion criteria as of 25/07/2023:
- •Key Inclusion Criteria:
- •1. Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 40 to 50, inclusive
- •Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C):
- •1. A Unified Huntington’s Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100
- •2. A UHDRS Total Functional Capacity (TFC) score of 13
- •3. A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)
- •Eligibility for HD-ISS Mild Stage 3 Group (Parts D, E, and F):
- •1. A UHDRS Total Functional Capacity (TFC) score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS score of <100
- •Note: Other inclusion criteria may apply.
- •Previous inclusion criteria:
- •1. Ambulatory male or female patients aged 25 years and older, inclusive
- •2. Subject (or legally authorized representative) is willing and able to provide informed consent and comply with all protocol requirements
- •3. Genetically confirmed HD diagnosis with a CAG repeat length from 42 to 50, inclusive
- •4. A UHDRS-IS score of 100
- •5. A UHDRS TFC score of 13
- •6. A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)
- •7. Women of childbearing potential (WOCBP) must agree to use highly effective methods of contraception during dosing and for 6 months after stopping the study medication.
- •8. Sexually active and fertile males must agree to use a condom during intercourse while taking the study drug and for 6 months after stopping the study drug and should neither father a child nor donate sperm in this period. A condom is required to be used also by vasectomized men in order to prevent the potential delivery of the drug via seminal fluid.
排除标准
- •Current exclusion criteria as of 25/07/2023:
- •Key Exclusion Criteria:
- •1. Receipt of an experimental agent within 90 days or 5 half-lives prior to Screening or anytime over the duration of this study, including ribonucleic acid (RNA)- or deoxyribonucleic acid (DNA)-targeted HD-specific investigational agents such as antisense oligonucleotides, cell transplantation, or any other experimental brain surgery
- •2. Any history of gene therapy exposure for the treatment of HD
- •3. Participation in an investigational study or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc) within 90 days prior to Screening or anytime over the duration of this study
- •4. Any medical history of brain or spinal disease that would interfere with the lumbar puncture process safety assessments
- •5. Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (for example, implanted shunt, conditions precluding magnetic resonance imaging [MRI] scans)
- •6. Pregnancy, planning on becoming pregnant during the course of the study or within 6 months of the end of treatment, or currently breastfeeding
- •Note: Other exclusion criteria may apply.
- •Previous exclusion criteria:
- •1. Inability or unwillingness to swallow oral tablets
- •2. Receipt of an experimental agent within 90 days or 5 half-lives prior to Screening or anytime over the duration of this study, RNA- or DNA-targeted HD-specific investigational agents such as antisense oligonucleotides, cell transplantation, or any other experimental brain surgery
- •3. Any history of gene therapy exposure for the treatment of HD
- •4. Participation in an investigational study or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc) within 90 days prior to Screening or anytime over the duration of this study
- •5. Presence of an implanted deep brain stimulation device
- •6. Family history of early onset cataracts or presence of cataracts at Baseline
- •7. Brain and spinal pathology that may interfere with CSF homeostasis and circulation, increased intracranial pressure (including presence of a shunt for the drainage of CSF or an implanted central nervous system catheter), malformations, and/or tumors
- •8. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks of Screening or planned during the study
- •9. At significant risk of suicide as measured by the C-SSRS with a moderate risk rating or higher score
- •10. Risk of a major depressive episode, psychosis, confusional state, or violent behavior as assessed by the investigator
- •11. Any medical history of brain or spinal disease that would interfere with the lumbar puncture processor safety assessments
- •12. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases
- •13. Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (eg, implanted shunt, conditions precluding MRI scans)
- •14. Antidepressant or benzodiazepine use, unless receiving a stable dose for at least 6 weeks prior to Screening and with a dose regimen that is not anticipated to change during the study
- •15. History of illicit/illegal drug use, or alcohol use in the high-risk category of risk drinking leve
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