EUCTR2018-002987-14-PL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
- 801
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 at the time of first dose administration
- •- 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
- •- 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 magnetic resonance imaging (MRI)
- •- 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 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 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
- •o Able and willing to attend in person all clinical visits for completion of
- •companion assessments and for input into the clinician-rated TFC scale
- •o Companions must have no cognitive, behavioral or motor change, in
- •the opinion of the investigator, that would question the validity of the
- •acquired observer-reported data
- •- 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 753
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 47
排除标准
- •- History of attempted suicide or suicidal 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 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 central nervous system catheter
- •- Positive for hepatitis C virus or hepatitis B surface antigen at
- •- 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
- •- 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
- •- 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 drugelimination 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 participation in any interventional clinical
- •study, including explicit pharmacological and non-pharmacological
- •interventions
- •- 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 access
- •- Scoliosis or spinal deformity or surgery making IT injection not
- •feasible in an outpatient setting and potentially interfering with
- •distribution of RO7234292 up the neuraxis
- •- An infection requiring oral or IV antibiotics within 14 days prior to
- •screening and prior to randomization
- •- Antiretroviral medications, including antiretroviral medication taken
- •as prophylaxis within 12 months of study enrollment
- •- Malignancy within 5 years prior to screening, except basal or
- •squamous cell carcinoma of the skin or carcinoma in situ of the cervix
研究者
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