EUCTR2018-003898-94-NL进行中（未招募）1 期
AN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY AND TOLERABILITY OF INTRATHECALLY ADMINISTERED RO7234292 (RG6042) IN PATIENTS WITH HUNTINGTON’S DISEASE
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 1,050
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
- •- Prior enrollment in a Roche sponsored or Genentech-sponsored study in HD for the RO7234292 development program that made provision for entry into an OLE study
- •- Ability and willingness to comply with the study protocol including the visit schedule and all assessments, in the investigator's judgment (apart from patients joining from Study BN40697 who are unable to undergo MRI)
- •- For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures, during the treatment period and for 5 months after the final dose of study drug
- •- For men: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom, and agreement to refrain from donating sperm during the treatment period and for 5 months after the final dose of study drug to avoid exposing the embryo
- •The following eligibility criteria only apply to patients who were eligible for Study BN40423 (GENERATION HD1) but were not randomized due to logistical challenges resulting from the COVID-19 pandemic
- •- Age 25-65 years
- •- Manifest HD diagnosis, defined as a diagnostic confidence level (DCL) score of 4
- •- Independence Scale (IS) score >= 70
- •- Genetically confirmed disease by direct DNA testing with a CAP score > 400
- •- Ability to read the words red, blue, and green in native language
- •- Ability to walk unassisted without a cane or walker and move about without a wheelchair on a daily basis as determined at screening and baseline visit
- •- Body mass index 16-32 kg/m2; total body weight > 40 kg
- •- Ability to undergo and tolerate MRI scans
- •- Ability to tolerate blood draws and lumbar punctures
- •- Creatinine clearance (CrCl) >= 60 mL/min (Cockcroft-Gault formula)
- •- Ability and willingness, in the investigator's judgment, to comply with all aspects of the protocol including completion of interviews and questionnaires for the duration of the study
- •- Stable medical, psychiatric, and neurological status for at least 12 weeks prior to screening and at the time of enrollment
- •- For women of childbearing potential: Agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive measures during the treatment period and for 5 months after the final dose of study drug
- •- For men: Agreement to remain abstinent or use a condom, and agreement to refrain from donating sperm during the treatment period and for 5 months after the final dose of study drug to avoid exposing the embryo. Men must refrain from donating sperm during this same period
- •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 1045
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 55
排除标准
- •- Withdrawal of consent from the preceding study
- •- Permanent discontinuation of RO7234292 for any drug-related safety concern during the preceding study or meeting of any study treatment discontinuation criteria specified in the preceding study at the time of enrollment into this study
- •- An ongoing, unresolved, clinically significant medical problem that in the judgment of the investigator would make it unsafe for the patient to participate in this study
- •- Antiplatelet or anticoagulant therapy within 14 days prior to inclusion or anticipated use during the study, including, but not limited to, aspirin (unless <=81 mg/day), clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban
- •- History of bleeding diathesis or coagulopathy
- •- Platelet count less than the lower limit of normal
- •- Concurrent participation in any therapeutic clinical trial (other than the preceding study)
- •- Study treatment (RO7234292) is commercially marketed in the patient’s country for the patient-specific disease and is accessible to the patient
- •- Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
- •The following eligibility criteria only apply to patients who were eligible for Study BN40423 (GENERATION HD1) but were not randomized due to logistical challenges resulting from the COVID-19 pandemic
- •- History of attempted suicide or suicidal ideation with plan that required hospital visit and/or change in level of care within 12 months prior to screening
- •- Current active psychosis, confusional state, or violent behavior
- •- Any serious medical condition or clinically significant laboratory, or vital sign abnormality or claustrophobia at screening that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study - History known to the investigator or presence of an abnormal ECG that is clinically significant in the investigator's opinion, including complete left bundle branch block, second- or third degree atrioventricular heart block, or evidence of prior myocardial infarction - Lifetime clinical diagnosis of chronic migraines
- •- Pregnant or breastfeeding, or intending to become pregnant during the study or within 5 months after the final dose of study drug
- •- Presence of an implanted shunt for the drainage of CSF or an implanted CNS catheter
- •- Positive for hepatitis C virus (HCV) or hepatitis B surface antigen (HBsAg) at screening
- •- Known HIV infection
- •- Current or previous use of an antisense oligonucleotide (including small interfering RNA)
- •- Current or previous use of anti-psychotics prescribed for a primary independent psychotic disorder, cholinesterase inhibitors, memantine, amantadine, or riluzole within 12 weeks from initiation of study treatment
- •- Treatment with investigational therapy within 4 weeks or 5 drug elimination half lives prior to screening, whichever is longer
- •- Antiplatelet or anticoagulant therapy within 14 days prior to screening or anticipated use during the study
- •- History of bleeding diathesis or coagulopathy - Platelet count less than the lower limit of normal
- •- History of gene therapy, cell transplantation, or any experimental brain surgery
- •- Concurrent participation in any therapeutic clinical trial.
- •- Drug and/or alcohol abuse or psychological or physiological dependency within 12 months prior to screening, as per the investigator's judgment
- •- Poor peripheral venous
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