NL-OMON49037已完成不适用
An open-label, adaptive multiple-dose study to investigate the pharmacokinetics and pharmacodynamics of RO7234292 in csf and plasma, and safety and tolerability following intrathecal administration in patients with Huntington's disease. - BP40410
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4
研究概览
简要总结
Trial is onging in other countries
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. Signed Informed Consent Form.
- •2. Age 25 to 65 years, inclusive, at the time of signing Informed Consent Form.
- •3. Manifest HD diagnosis, defined as a Diagnostic Confidence Level (DCL) score
- •4. Independence Scale score of >= 70.
- •5. Genetically confirmed disease by direct DNA testing with a CAP score > 400
- •(Zhang et al. 2011), calculated as follows:
- •CAP = Age x (CAG repeat length - 33.66).
- •6. Ability to read the words red, blue and green in the patient's native
- •7. Ability to walk unassisted without a cane or walker and move about without a
- •wheelchair on a daily basis as reviewed at screening and baseline visit. Long
- •distance use of wheelchairs for convenience (e.g., greater than 50 meters) for
- •transfer is permitted.
- •8. Body mass index >= 16 and <= 32 kg/m2; total body weight > 40 kg.
- •9. Ability to tolerate blood draws and lumbar punctures.
- •10. Estimated glomerular filtration rate >= 60 mL/min/1.73 m2 (Cockcroft-Gault
- •11. Ability and willingness, in the Investigator's judgment, to comply with all
- •aspects of the protocol including completion of interviews and assessments for
- •the duration of the study.
- •12. Stable medical, psychiatric, and neurological status for at least 12 weeks
- •prior to screening and at the time of enrollment.
- •13. Signed study companion consent for participation if a study companion is
- •available and fulfills the following criteria:
- •- Age >= 18 years.
- •- Reliable and competent, in the Investigator*s judgement.
- •- 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 judgement.
- •- Able to comment on the study participant*s symptoms and functioning
- •experience, as required per Appendix 1.
- •Note: Companions with genetic confirmation of the mutant gene can only
- •participate if they do not have confirmation of motor symptoms onset and, in
- •the opinion of the Investigator, do not display any disease symptoms (i.e., the
- •companion must have a DCL of < 4, as well as no cognitive or behavioral change
- •that would question the validity of the acquired observer-reported data).
- •All effort should be made to retain the study companion; however, should this
- •not be possible, a study companion can be replaced and new consent obtained.
- •14a. For women of childbearing potential: agreement to remain abstinent
- •(refrain from heterosexual intercourse) or use acceptable contraceptive
- •methods, and agreement to refrain from donating eggs.
- •Acceptable contraceptive methods have been listed in paragraph 4.1.1 of
- •protocol v2.0.
- •14b. For men: agreement to remain abstinent (refrain from heterosexual
- •intercourse) or use a condom, and agreement to refrain from donating sperm.
- •15. Ability to undergo and tolerate MRI scans (e.g., no claustrophobia; no
- •severe chorea or other condition that precludes MRI scans or renders scanning
- •intolerable for the patient; no MRI incompatible intrauterine devices, metallic
- •dental braces, or other metal implants).
排除标准
- •1. History of attempted suicide or suicidal ideation with plan (i.e., active
- •suicidal ideation) that required hospital visit and/or change in level of care
- •within 12 months prior to screening.
- •Current suicidal ideation is demonstrated by the C-SSRS per judgement 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. Mild passive suicidal
- •ideation (i.e., occasional thoughts that life is not worth living or is hard)
- •without history of attempts or hospitalization over the past 12 months is
- •generally acceptable for study participation, but final decision on
- •participation should be made carefully and in consultation with appropriately
- •qualified mental health professional.
- •2. Current active psychosis, confusional state, or violent behavior.
- •3. Any serious medical condition or clinically significant laboratory, vital
- •signs, or ECG abnormalities at screening that, in the Investigator's judgment,
- •precludes the patient's safe participation in and completion of the study.
- •4. Increased QTc interval (QT interval corrected through use of Fridericia's
- •formula [QTcF] > 470 ms), baseline resting bradycardia < 45 bpm, or baseline
- •resting tachycardia > 100 bpm.
- •5. Family history of long QT syndrome or other risk factors for torsades de
- •6. 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.
- •7. Clinical diagnosis of chronic migraines or history of low pressure headache
- •after lumbar puncture requiring hospitalization or blood patch.
- •8. Pregnant or breastfeeding, or intending to become pregnant during the study
- •or until the follow-up visit (6 months ± 2 weeks after the last dose of study
- •Women of childbearing potential must have a negative serum pregnancy test
- •result within 14 days prior to initiation of study drug.
- •9. Presence of implanted shunt for the drainage of CSF or an implanted CNS
- •10. Positive for hepatitis C virus antibody or hepatitis B surface antigen at
- •11. Positive for human immunodeficiency virus (HIV)-1 or HIV-2 at screening.
- •12. Current or previous use of an ASO (including small interfering ribonucleic
- •acid [RNA]).
- •13. Current or previous use of antipsychotics 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 of enrollment.
- •14. Current use of antipsychotics for motor symptoms or mood stabilization
- •(i.e., irritability or aggressive behavior) 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.
- •15. Current use of tetrabenazine, valbenazine, or deutetrabenazine within 2
- •weeks prior to screening or within 6 x the elimination half-life of the
- •medication prior to screening (whichever is longer) or anticipated use during
- •16. Current use of supplements
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