A phase 2a, randomized, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of ptc518 in subjects with Huntington's disease - PIVOT HD

PTC Therapeutics, Inc.0 个研究点目标入组 24 人开始时间: 待定最近更新: 2024年4月15日

试验速览

阶段: 2 期
状态: 招募中
发起方: PTC Therapeutics, Inc.
入组人数: 24

研究概览入排标准研究者记录与标识符相似试验

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

年龄范围: 18 至 99（—）

入选标准

•1. Ambulatory male or female patient aged 25 years and older, inclusive
•2. Subject is willing and able to provide informed consent and comply with all
•protocol requirements
•3. Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG)
•repeat length from 42 to 50, inclusive. CAG repeat length may be determined
•analytically through amplification.
•Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C)
•4. A UHDRS IS score of 100
•5. A UHDRS Total Functional Capacity (TFC) score of 13
•6. 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)
•9. A UHDRS TFC score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS
•score of <100
•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. Women of childbearing potential are defined as women who are
•fertile, following menarche and until becoming postmenopausal unless
•permanently sterile. Permanent sterilization methods include hysterectomy,
•bilateral salpingectomy, and bilateral oophorectomy. A postmenopausal state is
•defined as no menses for 12 months without an alternative medical cause. A high
•follicle-stimulating hormone (FSH) level in the postmenopausal range may be
•used to confirm a postmenopausal state in women not using hormonal
•contraception or hormonal replacement therapy. However, in the absence of 12
•months of amenorrhea, a single FSH measurement is insufficient.
•Female partners of enrolled males who are of childbearing potential should
•consider use of highly effective methods of contraception while the enrolled
•male is taking study drug and for 6 months after stopping study drug.
•Highly effective contraception methods are defined as those that can achieve a
•failure rate of less than 1% per year when used consistently and correctly and
•include the following:
•Combined (estrogen and progestogen containing) hormonal contraception
•associated with inhibition of ovulation:
•* Intravaginal
•* Transdermal
•Progestogen-only hormonal contraception associated with inhibition of
•* Injectable
•* Implantable
•Intrauterine device
•Intrauterine hormone-releasing system
•Bilateral tubal occlusion
•Vasectomized partner
•Sexual abstinence
•In case of use of oral contraception, WOCBP should have been stable on the same
•pill for a minimum of 3 months prior to Screening.
•Abstinence is acceptable only as true abstinence when this is in line with the
•preferred and usual lifestyle of the subject. Periodic abstinence (eg,
•calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are
•not acceptable methods of contraception.
•8. Sexually active and fertile males must use a condom during intercourse while
另有 4 项未显示

排除标准

•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, including 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. Observational
•studies (such as ENROLL-HD) are not exclusionary.
•6. Family history of early onset cataracts or presence of significant cataracts
•at Baseline. Visually significant cataract is defined as any Lens Opacity
•Classification System II grading >=2, Best Corrected Visual Acuity <20/40,
•cataract as the primary cause of vision impairment, and self-reported vision of
•fair or worse.
•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 Baseline version
•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 process or 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 magnetic resonance imaging scans)
•14. Antidepressant, antipsychotic, 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. Benzodiazepine use for
•sedation for study-related procedures during the course of the study is
•15. History of illicit/illegal drug use, or alcohol use in the high-risk
•category of risk drinking levels according to the World Health Organization for
•a duration of 1 month or longer that in the opinion of the investigator could
•compromise the interpretability of study results
•16. Clinically significant medical condition, which in the opinion of the
•investigator could adversely affect the safety of the subject or impair the
•assessment of study results (eg, inability to fast or any known
•hypersensitivity to PTC518 or its excipients)
•17. Current significant renal impairment defined as estimated glomerular
•filtration rate <60 mL/min/1.73 m2 at Screening
•18. Current hepatic impairm

研究者

发起方

PTC Therapeutics, Inc.

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