EUCTR2014-001489-85-NL进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 2a Safety, Tolerability, and Pharmacodynamic Study of Two Doses of an Histone Deacetylase Inhibitor (FRM-0334) in Subjects with Prodromal to Moderate Frontotemporal Dementia with Granulin Mutation
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
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
- •1.Informed consent form (ICF) signed by the subject or legally acceptable representative indicating that the subject or legally acceptable representative understands the purpose of and procedures required for the study before any study-specific procedures are performed
- •2.A support person may be required to participate in the study (in the judgment of the investigator at screening or as required by local regulations). The support person must sign an acknowledgement of responsibilities form at the study center before any study-specific activities required for the support person are performed. The first required activity will be performed at screening. The support person will accompany the subject to the study center at screening and on Days 1 and 28 (or be available by telephone on Days 1 and 28), and if not living in the same household, interacts with the subject approximately 4 times per week, and be able to complete the study
- •3.Male or female subjects aged =21 and =75 years
- •4.Genotyped positive for a FTD-GRN mutation
- •5.Prodromal to moderate FTD-GRN, and for subjects who require a support person (refer to inclusion criterion No. 2), a Clinical Dementia Rating Sum of the Boxes (CDR SB) score <16 at screening
- •6. Heterosexually active subjects (men and women) of childbearing potential and their partners must practice true abstinence (periodic abstinence [eg, calendar, ovulation, symptothermal, post-ovulation methods] and withdrawal are not acceptable) or use 2 highly-effective methods of contraception, including at least 1 barrier method, from informed consent until 1 week after last dose. Non-childbearing potential and highly-effective methods of contraception are defined in Section 4.4
- •7.Women of childbearing potential must have a negative pregnancy test at screening and Day 1
- •8.Resides in a stable living situation, living at home, senior residential setting, or an institutional setting without the need for continuous (ie, 24-hour) nursing care
- •9.Proficiency (oral and written) in the language in which study-related documents, including the ICF and standardized tests, will be administered
- •10.Able to swallow capsules
- •11.Be in good general health, willing and able to comply with the protocol requirements, and expected to complete the study as designed (in the judgment of the investigator)
- •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 20
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 10
排除标准
- •1.Employees of Inv.or study center or family members or employees of FORUM Pharma orWCT directly involved in conduct of study
- •2.Female subj.who are pregnant,breastfeeding or planning to become pregnant during study
- •3.Unstable medical condition that is C.S.(in Inv.judgment)within 30days before screen
- •4.Untreated vitamin B12or folate deficiency(must be stably treated for at least 6months before screen)
- •5.C.S.untreated hypothyroidism(if treated,thyroid-stimulating hormone lev. and thyroid supplementation dose must stable for at least 6months before screen)
- •6.C.S.abnormal serum electrolytes(sodium,potassium and magnesium)after repeat testing(in Inv.judgment)
- •7.ALTorAST>2.5times upper limit of normal
- •8.Renal insufficiency with serum creatinine>2.0mg/dL,unless receiving current treatment with an ACE inh.in which case M.M. should be contacted
- •9.Insufficiently controlled diabetes mellitus(in Inv.judgment)
- •10.C.S.hematologic abnormalities including thrombocytopenia and leukocytosis(in Inv.judgment)
- •11.Malign. tumor within 3years before screen.with exception of squamous and basal cell carcinoma or cervical carcinoma in situ or brachytherapy for localized prostate cancer
- •12.Systemic infection of any kind or any acute,subacute or chronic inflammatory process(eg, rheumatoid arthritis,chronic obstructive pulmonary dis.or gastrointestinal inflammatory dis.)
- •13.MRI/CT scan performed within 12mnths before screen.with findings with C.S.comorbid pathology other than FTD.If MRI/CT is unavailable or occurred>12mnts screen, an MRI scan must be be done & findings confirmed before the Day -7 procedures are performed, a copy of the DICOM standard image &report must be available
- •14.Diagnosis of motor neuron disease,including probable amyotrophic lateral sclerosis
- •15.History of brain tumor,subdural hematoma or other C.S.(in Inv.judgment)space-occupying lesion on CTorMRI
- •16.Stroke within 18months before screening or history of stroke concomitant with onset of dementia
- •17.Head trauma with C.S.(in Inv.judgment)loss of consciousness within 12months before screen.or concurrent with onset of dementia
- •18.Onset of dementia within 12months before screen.secondary(in Inv. judgment)to cardiac arrest,surgery with general anesthesia or resuscitation
- •19.Specific degenerative CNSdisease diagnosis other thanFTD(eg.Parkinson’s,Alzheimer’s,Huntington’s,Creutzfeldt Jakob,Down’s)
- •20.Wernicke’s encephalopathy
- •21.Epilepsy if present antiseizure therapy is required for seize control
- •22.Current diagnosis of severe major depressive disorder with psychotic features,if present condition or treatment interferes with subj’s ability to complete study(in Inv. judgment)
- •23.Significant suicide risk as defined by1)suicidal ideations as endorsed on items 4or5 on Columbia Suicide Severity Rating Scale within past year at screen.or baseline2)suicidal behaviors detected by C-SSRS within 2years before screen3)Inv.assessment
- •24.History or current diagnosis of psychosis
- •25.History within 2years before screen or current evidence of substance abuse as defined by Diagnostic and Statistical Manual of Mental Disorders 4th Edition Text Rev
- •26.C.S.urine drug screen or serum alcohol test result(in Inv. judgment)at screen(may be repeated once)
- •27.C.S.abnormality on screen.or baseline ECG,including but not necessarily limited to confirmed corrected QTc value=450msec for males or=470msec for fem.In subj.with QRS value>120msec those with QTc value>480msec are NOTeligible
- •28.History of my
研究者
相似试验
进行中（未招募）
1 期
This is a randomized, double-blinded, Placebo controlled trial with a 3-part treatment period that will evaluate the efficacy and safety of up to 70 weeks of treatment with tildacerfont in subjects with classic Congenital adrenal hyperplasia (CAH) who have elevated blood hormones at baselineClassic Congenital Adrenal HyperplasiaMedDRA version: 20.0Level: LLTClassification code 10010323Term: Congenital adrenal hyperplasiaSystem Organ Class: 100000004850EUCTR2019-004764-22-DESpruce Biosciences, Inc.72
进行中（未招募）
1 期
A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic FibrosisCystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10.000 children. In Cystic Fibrosis chloride transport accross the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli 1901 corrects the abnormal transport of chloride thereby reducing the formation of mucus plugs and improving clearance.EUCTR2006-006693-24-FRAOP Orphan Pharmaceuticals AG160
进行中（未招募）
不适用
A Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation Study to Investigate the Safety, Tolerability, Pharmacokinetics and Antiviral Activity of Multiple Doses of ACH-0137171 in Subjects with Chronic Hepatitis C InfectioEUCTR2006-002696-40-DEAchillion Pharmaceuticals, Inc.30
进行中（未招募）
不适用
A study to evaluate the efficacy of different doses of LY3009104 therapy compared to placebo in patients with Rheumatoid Arthritis who are on ongoing methotrexate background therapy.MedDRA version: 14.1Level: PTClassification code 10039073Term: Rheumatoid arthritisSystem Organ Class: 10028395 - Musculoskeletal and connective tissue disordersRheumatoid ArthritisEUCTR2010-022504-42-HUEli Lilly and Company270
进行中（未招募）
1 期
Study to test the efficacy and safety of Padsevonil as adjunctive treatment of focal-onset seizures in adults with drug-resistant epilepsyFocal-Onset SeizuresMedDRA version: 21.1Level: LLTClassification code 10065337Term: Focal epilepsySystem Organ Class: 100000004852EUCTR2017-003200-48-CZCB Biopharma SPR411