A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis

Orphazyme A/S0 个研究点目标入组 231 人开始时间: 2018年10月9日最近更新: 2019年1月7日

试验速览

阶段: 1 期
状态: 进行中（未招募）
发起方: Orphazyme A/S
入组人数: 231

研究概览入排标准研究者记录与标识符相似试验

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. Capable of- and willing to- provide written informed consent and comply with trial procedures.
•2. Subject is male or female =18 years of age.
•3. Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS.
•4. 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath).
•5. ALSFRS-R =35 and erect SVC % predicted = 70% at Screening.
•6. Able and willing to travel to the site, and in the investigator’s opinion is likely to attend visits for at least 24 weeks.
•7. All sexually active female subjects of child-bearing potential (postmenarchal)* must agree not to intend to become pregnant and use a highly effective method of contraception** during the trial through 1 month after the last dose of trial medication. If the subject is a sexually active male with female partners of child-bearing potential (postmenarchal) he must use a condom with or without spermicide in addition to the birth control used by their partners during the trial until 3 months after the last dose of trial medication.
•8. Stable dose of riluzole (50 mg twice daily) for a minimum of 14 days prior to Day 1 (Baseline), or has not taken it for 14 days prior to Day 1.
•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 160
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 71

排除标准

•1. Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of Screening and Baseline visits.
•2. Pregnant or breast-feeding.
•3. Current or anticipated use of diaphragmatic pacing during the trial.
•4. Exposure to any investigational treatment within 4 weeks or <5 half-lives of the Screening visit, whichever is longest and/or participated in any prior ALS clinical trial receiving active drug treatment (with the exception described in exclusion criterion 5).
•5. Previously or currently treated with edaravone. However, up to 18 subjects on stable (i.e. minimum 6 months’) treatment with edaravone and who otherwise fulfil the eligibility criteria are planned for enrolment (limited to countries where edaravone has a marketing authorisation for treatment of ALS).
•6. Any of the following medically significant conditions:
•a) Neurological impairment/dysfunction or unstable psychiatric illness that in the investigator’s opinion is likely to interfere with assessment of ALS disease progression.
•b) Clinically significant unstable medical condition other than ALS, which would present a risk to a subject to participate in the trial
•c) Presence of dementia that impairs the ability of the subject to provide informed consent according to the PI decision.
•d) Known or suspected allergy or intolerance to the IMP (arimoclomol or constituents);
•e) Chronic infection particularly HIV or Hepatitis B or C.
•f) Clinically significant renal or hepatic disease
•g) Aspartate aminotransferase, alanine aminotransferase, and lactate dehydrogenase =3 times the upper limit of normal [ULN], bilirubin=2 times the ULN, or creatinine =1.5 times the ULN). Laboratory tests may be repeated once at Screening. Reasons to repeat laboratory tests may include that the medication causing laboratory abnormality was suspended, any other suspected cause may no longer exist, or to rule out laboratory error.
•h) Cancer that is currently under active treatment or is likely to require treatment during the trial that may alter the subject´s function and thereby interfere with assessment of ALS disease progression.
•i) Any other condition that in the investigator’s opinion would present a risk to a subject to participate in the trial, interfere with the assessment of safety or has an increased risk of causing death during the trial.
•* Non child-bearing potential is defined as post-menopausal (minimum of 12 months with no menses and follicle-stimulating hormone in the post-menopausal range) or sterilisation (hysterectomy, oophorectomy, or bilateral tubal ligation).
•** Highly effective methods of contraception include combined (oestrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; and vasectomised partner.
•According to the recommendations from the Clinical Trial Facilitation Group (CTFG, 2014), sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the trial until 1 week after the last dose of trial medication (for female subjects of child-bearing potential) and for 3 months after the last dose of trial medication (for male subjects with female partners of child-bearing potential)

研究者

发起方

Orphazyme A/S

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