ACTRN12622000927729已完成1 期
An open-label, phase 1b study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of two doses of PMX205 in patients with amyotrophic lateral sclerosis
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 8
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 18 Years 至 75 Years（—）
- 性别
- All
入选标准
- •1.Male or female volunteers 18-75 years of age.
- •2.Able and willing to provide written informed consent prior to the performance of any study-specific procedures.
- •3.Have possible, probable, probable laboratory-supported, definite, or definite familial laboratory-supported amyotrophic lateral sclerosis (ALS) in accordance with the El-Escorial criteria
- •4.Have familial or sporadic ALS.
- •5.Have slow vital capacity (SVC) of greater than or equal to 50% of predicted value for gender, height and age
- •6.If on riluzole, must be on a stable dose for at least 30 days
- •7.If on edaravone, must have completed 2 cycles and are expected to remain on the same dose throughout the study
- •8.Able to provide informed consent. If the patient is not able to provide written consent due to aggravation of disease condition, written informed consent may be provided by a legally authorized representative
- •9.Have venous access sufficient to allow for blood sampling
- •10.Have a coagulation profile within normal reference range for the population or study site, or results with acceptable deviations that are judged by the Principal Investigator (PI) to be not clinically significant.
- •11.Have adequate bone marrow reserve, renal function and liver function as defined below (and all other laboratory results within normal reference range or with acceptable deviations that are judged by the PI to be not clinically significant):
- •Haemoglobin greater than or equal to 100 g/L
- •Absolute neutrophil count greater than or equal to 1.5 × 109/L
- •Platelet count greater than or equal to 100 × 109/L
- •Estimated creatinine clearance greater than or equal to 60 mL/min (calculated using the Cockcroft-Gault formula)
- •ALT, AST, and/or alkaline phosphatase less than or equal to 2 × ULN
- •Total bilirubin less than or equal to 1.5 × ULN
- •Serum albumin greater than or equal to 2.8 g/dL
- •12.BMI between 18.0 and 30.0 kg/m2, inclusive.
- •13.Non-smokers or ex-smokers who have ceased smoking greater than 6 months prior to the screening visit and have no more than one pack-year history of smoking.
- •14.Women who are not of child-bearing potential may be enrolled and are defined as women who:
- •have been surgically sterilized; or
- •are post-menopausal, as defined by amenorrhea for at least 12 consecutive months (and not induced by medical conditions such as anorexia nervosa and not taking medications that induced the amenorrhea, e.g., oral contraceptives, hormones, gonadotropin releasing hormones, anti-estrogens, selective estrogen receptor modulators, or chemotherapy) and confirmed by a serum follicle-stimulating hormone (FSH) level in the post-menopausal range.
- •15.Women of childbearing potential may be enrolled if they are not pregnant or lactating and agree to use both a barrier method of contraception (i.e. a condom or diaphragm) and a highly-effective method of contraception, from screening until 60 days after the last dose of study treatment. Highly-effective methods of contraception are associated with a pregnancy rate of less than 1% when used correctly and consistently, and include hormonal contraception and intrauterine devices. Abstinence from heterosexual intercourse is an acceptable alternative if this is the usual and preferred lifestyle of the subject.
- •16.Male subjects, if not surgically sterilized, must agree to use adequate contraception and not donate sperm from admission to the clinical research unit until 120 days after the last dose of study treatmen
排除标准
- •Have a history or presence of medical illness including, but not limited to, any cognitive, cardiovascular, hepatic, haematological, renal, endocrine, or psychiatric, or any clinically significant laboratory abnormality that indicates a medical problem that would preclude study participation
- •2.Positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody or human immunodeficiency virus (HIV)-1 or HIV-2 antibody at screening.
- •3.Show evidence of hepatitis B virus (HBV) and/or positive hepatitis B surface antigen
- •4.Are women who are lactating.
- •5.Have undergone a tracheostomy unless it was removed at least 6 months prior
- •6.Are on feeding tube, unless the insertion of a feeding tube is considered prophylactic
- •7.Are on nasal intermittent positive pressure ventilation (NIPPV) > 4 hours per day or at the discretion of the medical monitor
- •8.Have undergone stem cell therapy
- •9.Have any other unbalanced progressive pathology
- •10.Have experienced weight loss > 10% of their weight before disease
- •11.Have been on drugs that may interfere with the process of neuroinflammation: drugs with anti-inflammatory properties within 30 days of screening, e.g., corticosteroids, azathioprine, anti-tumor necrosis factor (TNF), antibiotics. [NSAIDs are allowed, except from 48 hours before dosing until 24 hours following dosing.]
- •12.Previous treatment with PMX205 or other complement system inhibitor.
- •13.Presence of an active infection or body temperature > 37.5°C at screening or admission to the clinical trial unit.
- •14.Clinically significant history of chronic or recurrent infections, including opportunistic infections.
- •15.History of splenectomy.
- •16.Use of systemic immunosuppressive medications in the past 2 years.
- •17. Is taking anti-coagulant medication(s) and cannot stop the medication(s) for 2 weeks before lumbar puncture.
- •18.History of any medical or surgical condition where lumbar puncture is contraindicated.
- •19.Presence of a local skin infection or other dermatological condition in the lumbar region, which the PI considers would complicate a lumbar puncture.
- •20.Received an investigational therapy within 30 days (or 5 half-lives, whichever is longer) prior to PMX205 administration.
- •21.History of clinically-significant allergic reactions, including hypersensitivity to penicillins, cephalosporins and related antibiotics.
- •22.History of alcohol or drug abuse.
- •23.Positive screen for drugs of abuse (including opiates, cocaine, amphetamines, MDMA, cannabinoids, barbiturates and benzodiazepines) or alcohol at screening.
- •24.Average intake of more than 14 units of alcohol per week (1 unit of alcohol equals approximately 250 mL of beer, 100 mL of wine or 35 mL of spirits).
- •25.Donation or loss of more than 400 mL of blood within 60 days of trial.
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