NL-OMON55873已完成3 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 - BN40423 - GENERATION HD1
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 20
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
- •* Signed Informed Consent Form
- •* Age 25 to 65 years, inclusive
- •* Manifest HD diagnosis, defined as a diagnostic confidence level (DCL) score
- •* Independence Scale (IS) score * 70
- •* Genetically confirmed disease by direct DNA testing with a CAP score >400,
- •calculated as follows: CAP <= 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
- •o Long-distance (e.g., > 50 meters) use of wheelchairs for convenience or
- •transfer is permitted.
- •* Body mass index 16-32 kg/m2; total body weight > 40 kg
- •* Ability to undergo and tolerate MRI scans, to tolerate blood draws and lumbar
- •* 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
- •* Ability and willingness, in the investigator's judgment, to carry a
- •smartphone, wear a digital monitoring device, and complete smartphone-based
- •* 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
- •criteria as specified in protocol section 4.1.1.
- •* For women of childbearing potential: agreement to remain abstinent (refrain
- •from heterosexual intercourse) or use contraceptive measures, as defined in
- •protocol section 4.1.1.
- •* For men: agreement to remain abstinent (refrain from heterosexual
- •intercourse) or use a condom, and agreement to refrain from donating sperm, as
- •defined in protocol section 4.1.1.
排除标准
- •* 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
- •* Current suicidal ideation is demonstrated by the C-SSRS per judgment of the
- •investigator. If suicidal ideation is present, a risk assessment should be done
- •by an appropriately qualified mental health professional to assess whether it
- •is safe for the patient to participate in the study.
- •* 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
- •* 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
- •* Women of childbearing potential must have a negative serum pregnancy test
- •result at baseline and a confirmatory urine pregnancy test prior to initiation
- •of study drug.
- •* Presence of an implanted shunt for the drainage of CSF or an implanted CNS
- •* Positive for hepatitis C virus (HCV) antibody 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 (i.e., schizophrenia, schizoaffective disorder,
- •bipolar disorder type I, severe with psychotic features), cholinesterase
- •inhibitors, memantine, amantadine, or riluzole within 12 weeks from initiation
- •of study treatment
- •* Current use of antipsychotics for motor symptoms or mood stabilization (i.e.,
- •irritability or aggressive behavior) 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
- •* Current use of supplements (e.g., coenzyme Q10, vitamins, creatine) 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
- •(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
- •* Platelet counts between 125,000 and 150,000 mm3 are permissible as long as
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