2025-523828-51-00招募中3 期
IB1001-304: Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, randomized, placebo-controlled, double-blind, crossover study
Intrabio Limited, IntraBio Inc11 个研究点 分布在 4 个国家目标入组 25 人开始时间: 2026年8月1日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 25
- 试验地点
- 11
- 主要终点
- The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage.
研究概览
简要总结
To evaluate the efficacy of N-Acetyl-L-Leucine based on the Scale for the Assessment and Rating of Ataxia (SARA) for the chronic treatment of CACNA1A disorders.
研究设计
- 分配方式
- Na
- 主要目的
- Open Lable Extension Phase
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 0-17 Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness
- •An understanding of the implications of study participation, provided in the written patient information and informed consent by patients or their legal representative/parent, and demonstrates a willingness to comply with instructions and attend required study visits (for children this criterion will also be assessed in parents or appointed guardians).
- •Male or female aged ≥4 years with a genetically confirmed diagnosis of a CACNA1A disorder (including patients with loss-of-function and fain-of-function mutations, e.g. Episodic Ataxia Type 2 [EA2], Familial Hemiplegic Migraine Type 1 [FHM1], Spinocerebellar Ataxia type C (SCA6), Developmental and Epileptic encephalopathy 42 (DEE42), Congenital ataxia or cerebellar hypoplasia due to a CACNA1A mutation ) at the time of signing informed consent
- •Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose and confirm to continue through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in <1% failure rate when used consistently and correctly) 14 days prior to the first dose continuing through 28 days after the last dose: a) intrauterine device (IUD); b) surgical sterilization of the partner (vasectomy for 6 months minimum); c) combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal); d) progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable); e) intrauterine hormone releasing system (IUS); f) bilateral tubal occlusion.
- •Females of non-childbearing potential who have undergone one of the following sterilization procedures at least 6 months prior to the first dose: a) hysteroscopic sterilization; b) bilateral salpingectomy; c) hysterectomy; d) bilateral oophorectomy; OR be postmenopausal with amenorrhea for at least 1 year prior to the first dose and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal status. FSH analysis for postmenopausal women will be done at screening. FSH levels should be in the postmenopausal range as determined by the central laboratory.
- •Non-vasectomized male patient agrees to use a condom with spermicide until 90 days beyond the last dose of study medication and the female partner agrees to comply with inclusion criteria 3 or
- •For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non-vasectomized male
- •If male, patient agrees not to donate sperm from the first dose until 90 days after their last dose.
- •Patients who have ataxia symptoms which (outside of episodes, is applicable) fall within: a) A SARA score of 7 ≤ X ≤ 34 points (out of 40) AND b) Either: i. Within the 2-7 range (0-8 range) of the Gait subtest of the SARA scale OR ii. Be able to perform the 9-Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 ≤ X ≤150 seconds.
- •Weight ≥15 kg at screening
- •Patients are willing to disclose their existing medications/therapies for (the symptoms of) CACNA1A disorder including those on the prohibited medication list. Non-prohibited medications/therapies (e.g. speech therapy, physiotherapy) are permitted provided: a) The Investigator does not believe the medication/therapy will interfere with the study protocol/results b) Patients have been on a stable dose/duration and type of therapy for at least 42 days before Visit 1 (Baseline 1) c) Patients are willing to maintain a stable dose/do not change their therapy throughout the duration of the study.
排除标准
- •Patients who have any known hypersensitivity or history of hypersensitivity to: a. Acetyl-Leucine (DL-, L-, D-) or derivatives. b. Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry flavor). c. Excipients the placebo sachet (namely isomalt, hypromellose, strawberry flavor, citric acid, microcrystalline cellulose, lactose, denatonium benzoate).
- •Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product (IMP; ‘study drug’) for at least 42 days prior to Visit
- •At the discretion of the Investigator, Medical Monitor, and Sponsor, the washout period for specific IMPs may be longer based on the pharmacological activity and pharmacokinetics of the drug.
- •Patients with a physical, cognitive, or psychiatric condition which, at the Investigator’s discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient’s participation in the clinical study, i.e. reliably perform study assessments.
- •Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.
- •Current or planned pregnancy or women who are breastfeeding.
- •Patients with severe vision or hearing impairment (that is not corrected by glasses or hearing aids) that, at the Investigator’s discretion, interferes with their ability to perform study assessments
- •Patients who have been diagnosed with arthritis or other musculoskeletal disorders affecting joints, muscles, ligaments, and/or nerves that by themselves affect patient’s mobility and, at the Investigator’s discretion, interferes with their ability to perform study assessments.
- •Patients at non-EU trial sites unwilling and/or not able to undergo a 42-day washout period from any of the following prohibited medication prior to Visit 1 (Baseline 1) and remain without prohibited medication through Visit
- •a. N-Acetyl-DL-Leucine (e.g. Tanganil®); b. N-Acetyl-L-Leucine (prohibited if not provided as IMP in the IB1001-304 trial);
- •Patients at EU trial sites who have had any of the following prohibited medication 42-days prior to Visit 1 (Baseline 1) and unwilling and/or not able to remain without prohibited medication through Visit
- •a. N-Acetyl-DL-Leucine (e.g. Tanganil®); b. N-Acetyl-L-Leucine (prohibited if not provided as IMP in the IB1001-304 trial).
研究组 & 干预措施
N-Acetyl-L-Leucine
Test
干预措施: N-Acetyl-L-Leucine (Drug)
granules for suspension
Placebo
干预措施: granules for suspension (Drug)
结局指标
主要结局
The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage.
The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage.
次要结局
- • Spinocerebellar Ataxia Functional Index (SCAFI) • International Cooperative Ataxia Rating Scale (ICARS) • Quality of Life EQ-5D-5L for patients ≥18; EQ-5D-Y for children <18 years • Neuro Quality of Life – Upper Extremity Function (NeuroQOL-UEF) assessed by the Patient or the Caregiver • Investigator, Caregiver (if applicable), and Patient (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of Treatment Period I (Visit 4) to baseline (Visit 2).
- • Spinocerebellar Ataxia Functional Index (SCAFI) • Functional Scale for the Assessment and Rating of Ataxia (f-SARA) [US only - key secondary endpoint in the US] • Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for children aged <18 years • Neuro Quality of Life – Upper Extremity Function (NeuroQOL-UEF) assessed by the Patient or the Caregiver (if patient unable to complete)
研究者
Taylor Fileds
Scientific
Intrabio Limited
研究点 (11)
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