EUCTR2018-002987-14-NL进行中（未招募）1 期
A RANDOMIZED, MULTICENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED, PHASE III CLINICAL STUDY TO EVALUATE THE EFFICACY AND SAFETY OF INTRATHECALLY ADMINISTERED RO7234292 (RG6042) IN PATIENTS WITH MANIFEST 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
- 600
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
- •- Age 25 to 65 years
- •- Manifest HD diagnosis (defined as DCL score of 4)
- •- Independence Scale (IS) score >= 70
- •- Genetically confirmed disease by direct DNA testing with a CAP score >= 400 (Age x [CAG repeat length – 33.66])
- •- 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
- •- Estimated glomerular filtration rate >= 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 and to carry a smartphone, wear a digital monitoring device, and complete smartphone-based tasks
- •- Stable medical, psychiatric, and neurological status for at least 12 weeks prior to screening and at the time of enrollment
- •- Signed study companion consent for participation if a study companion is available who fulfills all of the following criteria:
- •o Age >= 18 years
- •o Reliable and competent, in the investigator's judgment
- •o Sufficiently knowledgeable of the patient's condition to complete study companion assessments of the patient, and likely to remain sufficiently knowledgeable throughout the study, in the investigator's judgment
- •o Able to comment on study participant's symptoms and functioning experience
- •- For women of childbearing potential: agreement to remain abstinent or use contraceptive measures, women must remain abstinent or use two methods of contraception, including at least one method with a failure rate of < 1% per year, 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, with a female partner of childbearing potential or pregnant female partner, men must remain abstinent or use a condom during the treatment period and for 5 months after the final dose of study drug to avoid exposing the Embryo
- •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 620
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 40
排除标准
- •- History of attempted suidide or suididal ideation with plan (i.e., active suididal ideation) 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 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
- •- 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 central nervous system catheter
- •- Positive for hepatitis C virus antibody or hepatitis B surface antigen at screening
- •- Known HIV infection
- •- Current or previous use of an antisense oligonucleotide
- •- 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
- •- Current use of antipsychotics for motor symptoms or mood stabilization and/or tetrabenazine, valbenazine, or deutetrabenazine at a dose that has not been stable for at least 12 weeks prior to screening or is anticipated to change between screening and treatment initiation
- •- Current use of supplements at a dose that has not been stable for at least 6 weeks prior to screening or is anticipated to change during the study
- •- Current use of an antidepressant or benzodiazepine at a dose that has not been stable for at least 12 weeks prior to screening or is anticipated to change between screening and treatment initiation
- •- 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, including, but not limited to, aspirin, clopidogrel, dipyridamole, warfarin, dabigatran, rivaroxaban, and apixaban
- •- 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 or planned concurrent participation in any interventional clinical study, including explicit pharmacological and non-pharmacological interventions
- •- Illicit drug or alcohol abuse within 12 months prior to screening, as per the investigator's judgment
- •- Poor peripheral venous access
- •- Scoliosis or spinal deformity or surgery making IT injection not feasible in an outpatient setting
- •- Serious infection requiring oral or IV antibiotics within 14 days prior to screening
- •- Antiretroviral medications
- •- Malignancy within 5 years prior to screening, except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
- •- Preexisting structural brain lesion as assessed by a centrally read MRI scan during the screening period
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