EUCTR2022-001991-32-AT进行中（未招募）1 期
A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, DOSE-FINDING STUDY TO EVALUATE THE SAFETY, BIOMARKERS, AND EFFICACY OF TOMINERSEN IN INDIVIDUALS WITH PRODROMAL AND EARLY 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
- 300
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
- 性别
- All
入选标准
- •- Age 25-50 years
- •- Huntington’s disease (HD) gene expansion mutation carrier status
- •- Eighter:
- •-Prodromal HD or Early manifest HD
- •- Estimated glomerular filtration rate >= 60 mL/min/1.73 m2 in at least one out of two maximum screening samples
- •- Total body weight > 40 kg and a body mass index within the range of 18-32 kg/m2
- •- Ability to read the words red, blue, and greenand be fluent in the language of the Informed Consent and the tests used at the study site
- •- 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 (Day 1)
- •- Ability to undergo and tolerate MRI scans
- •- Ability to tolerate blood draws and lumbar punctures (LPs)
- •- Stable medical, psychiatric, and neurologic status as assessed by physician for at least 12 weeks prior to screening and at the time of enrollment
- •- Negative hepatitis B surface antigen test at screening
- •- Negative hepatitis C virus (HCV) antibody test at screening, or a positive HCV antibody test followed by a negative HCV ribonucleic acid (RNA) test at screening
- •- For female participants of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception and agree to refrain from donating eggs during the treatment period and for 5 months after the final dose of the study drug. Female participants must refrain from donating eggs during this same period
- •- For male participants: agreement to remain abstinent (refrain from heterosexual intercourse) or use a condom and agree to refrain from donating sperm during the treatment period and for 5 months after the final dose of the study drug to avoid exposing the embryo. Male participants 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 300
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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 screening
- •- Active psychosis, confusional state, or violent behavior, including aggression that could cause harm to self or others, over the 12 weeks prior to screening
- •- Clinical diagnosis of chronic migraines as per the International Headache Society Classification of Headache Disorders
- •- Pregnancy or breastfeeding, or intention of becoming 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 (CNS) catheter
- •- Hydrocephalus
- •- History of deep brain stimulation
- •- Known human immunodeficiency viruses (HIV) infection
- •- An infection requiring oral or intravenous antibiotics within 14 days prior to screening, during screening and prior to randomization
- •- Anti-retroviral medications, including anti-retroviral medication taken as prophylaxis within 12 months of study enrollment
- •- Current or previous use of an antisense oligonucleotide (ASO) or any huntingtin protein (HTT) lowering therapy
- •- Treatment with investigational therapy within 4 weeks or 5 drug-elimination half-lives, whichever is longer, prior to screening
- •- Current or previous history of a primary independent psychotic disorder
- •- Current use of anti-psychotics 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 during the study, including at screening
- •- Cholinesterase inhibitors, memantine, or riluzole use within 12 weeks
- •from initiation of study treatment
- •- Current use of amantadine, anti-depressant or benzodiazepine at a
- •dose that has not been stable for at least 12 weeks prior to screening or
- •is anticipated to change during the study, including at screening
- •- Current use of supplements used to treat HD symptoms, including in an
- •experimental setting, at a dose that has not been stable for at least 6
- •weeks prior to screening or is anticipated to change during the study,
- •including at screening
- •- Anti-platelet or anticoagulant therapy within 14 days prior to screening
- •- History of bleeding diathesis or coagulopathy
- •- Platelet count less than the lower limit of normal
- •- History of gene therapy, cell transplantation, or brain surgery
- •- Planned brain surgery during the study
- •- Concurrent or planned participation in any interventional clinical study,
- •including explicit pharmacological and non-pharmacological
- •interventions. Observational studies are acceptable; however, data
- •collection is not permitted between screening and baseline visits and
- •throughout the duration of the study
- •- Poor peripheral venous access
- •- Scoliosis or spinal deformity or surgery making intrathecal (IT)
- •injection not feasible in an outpatient setting and potentially interfering
- •with distribution of tominersen up the neuraxis
- •- 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 intra-axial or extra-axial lesions as assessed by a centrally read MRI scan during the screening period
研究者
相似试验
进行中（未招募）
1 期
A Study of Lebrikizumab in Patients With Idiopathic Pulmonary Fibrosis.IDIOPATHIC PULMONARY FIBROSISEUCTR2013-001163-24-PLF. Hoffmann-La Roche Ltd480
进行中（未招募）
不适用
A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ONCE-DAILY ORALLY ADMINISTERED PH-797804 (0.5, 3, 6 AND 10 MG) IN ADULTS WITH MODERATE TO SEVERE CHRONIC OBSTRUCTIVE PULMONARY DISEASE (COPD) - N/AMedDRA version: 9.1Level: LLTClassification code 10010952Term: COPDChronic Obstructive Pulmonary DiseaseEUCTR2007-004466-41-HUPfizer Ltd, Ramsgate Road, Sandwich, Kent640
招募中
2 期
A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND PHARMACOKINETICS OF OBINUTUZUMAB IN ADOLESCENT PATIENTS WITH ACTIVE CLASS III OR IV LUPUS NEPHRITIS, INCLUDING AN EVALUATION OF OPEN LABEL SAFETY AND PK IN A COHORT OF PEDIATRIC PATIENTS (AGED 5 to < 12).11 NULLN118 Other chronic tubulo-interstitial nephritisOther chronic tubulo-interstitial nephritisN118PER-051-21F. HOFFMANN-LA ROCHE LTD.2
进行中（未招募）
1 期
A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals with Prodromal and Early Manifest Huntington’s DiseaseHuntington's disease (HD)MedDRA version: 20.0Level: PTClassification code 10070668Term: Huntington's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2022-001991-32-PTF.Hoffmann-La Roche Ltd300
进行中（未招募）
不适用
A PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY AND EFFICACY OF LEBRIKIZUMAB (MILR1444A)IN ADULT PATIENTS WITH ASTHMA WHO ARE INADEQUATELY CONTROLLED ON INHALED CORTICOSTEROIDS. - MILLYAsthma in patients who are inadequately controlled by inhaled corticosteroidsMedDRA version: 9.1Level: PTClassification code 10003553Term: AsthmaEUCTR2009-010359-28-CZGenentech, Inc210