NL-OMON53858招募中3 期
A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants with Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX) - Phase IIIb study of AMX0035 for the treatment of ALS
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 25
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
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Previous participation in Study A35-004 (PHOENIX), including completion of
- •the randomized-controlled phase through Week 48 (this timepoint may be upcoming
- •at the time of screening). Participants who do not complete
- •randomized-controlled phase through Week 48 for medical reasons may be included
- •on a case-by-case basis, in consultation with the sponsor; 2. Capable of
- •providing informed consent; 3. Capable and willing to follow trial procedures
- •including visits to the trial clinic, remote visits, and survival status
- •reporting requirements; 4. Women of childbearing potential (WOCBP; e.g., not
- •post-menopausal for at least one year or surgically sterilea must agree to use
- •adequate birth controlb for the duration of the trial and 3 months after the
- •last dose of AMX0035; a. 12 months of spontaneous amenorrhea or 6 months of
- •spontaneous amenorrhea with serum FSH levels > 40 mIU/ml or 6 weeks
- •postsurgical bilateral oophorectomy with or without hysterectomy. b. Acceptable
- •contraception methods for use in this trial are: - Hormonal methods, such as
- •birth control pills, patches, injections, vaginal ring, or implants; Barier
- •methods (such as a condom or diaphragm) used with a spermicide (a foam, cream,
- •or gel that kills sperm); - Intrauterine device (IUD); - Abstinence (no
- •heterosexual sex); - Unique partner who is surgically sterile (men) or not of
- •childbearing potential (female). 5. Women must not be pregnant or planning to
- •become pregnant for the duration of the trial and 3 months after last dose of
- •AMX0035; 6. Men must agree to practice contraception for the duration of the
- •trial and for at least 3 months after last dose of AMX0035; 7. Men must not
- •plan to father a child or to provide sperm for donation for the duration of the
- •trial and 3 months after the last dose of AMX0035.
排除标准
- •1. History of known allergy to phenyl butyrate or bile salts; 2. Abnormal liver
- •function defined as bilirubin levels and/or aspartate aminotransferase (AST)
- •and/or alanine aminotransferase (ALT) > 5 times the upper limit of the normal
- •(obtained within 12 weeks from first dose); 3. Renal insufficiency as defined
- •by eGFR <60 mL/min/1.73m2 normal (obtained within 12 weeks from first dose); 4.
- •Pregnant women or women currently breastfeeding; 5. Current severe biliary
- •disease which may result in the Investigator medical judgement in biliary
- •obstruction including for example active cholecystitis, primary biliary
- •cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the
- •gallbladder, abscess of the gallbladder; 6. History of Class III/IV heart
- •failure (per New York Heart Association - NYHA); 7. Participant under severe
- •salt restriction where the added salt intake due to treatment would put the
- •participant at risk, in the Investigator clinical judgment; 8. Presence of
- •unstable psychiatric disease, cognitive impairment, dementia or substance abuse
- •that would impair ability of the participant to provide informed consent,
- •according to Investigator judgment; 9. Clinically significant unstable medical
- •condition (other than ALS) (e.g., cardiovascular instability, systemic
- •infection, untreated thyroid dysfunction, severe laboratory test anomaly or
- •clinically significant electrocardiogram [ECG] changes) that would pose a risk
- •to the participant if he/she were to participate in the trial, according to
- •Investigator judgment; 10. Currently enrolled in another trial (excluding Study
- •A35-004 (PHOENIX)) involving use of an investigational therapy (or within 5
- •plasma half-lives) prior to first dose at Baseline Visit; 11. Implantation of
- •Diaphragm Pacing System (DPS); 12. Currently or previously treated within the
- •last 30 days (or 5 half-lives, whichever is longer) from first dose at the
- •Baseline Visit or planned exposure during the treatment period to any
- •prohibited medications listed in Section 6.7 of the protocol.
研究者
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