NL-OMON46863已完成3 期
International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months - CLN-PXP3003-02
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pharnext
- 入组人数
- 20
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. Male or female, aged from 16 to 65 years;
- •2. Patient with a proven genetic diagnosis of CMT1A;
- •3. Mild-to-moderate severity assessed by CMTNS-v2 with a score >2 and *18;
- •4. Muscle weakness in at least foot dorsiflexion (clinical assessment);
- •5. Motor nerve conduction of the ulnar nerve of at least 15m/sec;
- •6. Providing signed written informed consent to participate in the study and willing and
- •able to comply with all study procedures and scheduled visits.
排除标准
- •1. Any other associated cause of peripheral neuropathy such as diabetes;
- •2. Patients with another significant neurological disease or a concomitant major systemic
- •3. Clinically significant history of unstable medical illness since the last 30 days (unstable
- •angina, cancer*) that may jeopardize the participation in the study;
- •4. Significant hematologic disease, hepatitis or liver failure, renal failure;
- •5. Limb surgery within six months before randomization or planned before trial
- •completion;
- •6. Clinically significant abnormalities on the pre-study laboratory evaluation, physical
- •evaluation, electrocardiogram (ECG);
- •7. Elevated ASAT/ALAT (> 3 x ULN) and elevated serum creatinine levels (> 1.25 x ULN);
- •8. History of recent alcohol or drug abuse or non-adherence with treatment or other
- •experimental protocols;
- •9. Patients using unauthorized concomitant treatments including but not limited to baclofen, naltrexone, sorbitol (pharmaceutical form), opioids, levothyroxin and
- •potentially neurotoxic drugs such as amiodarone, chloroquine, cancer drugs susceptible
- •to induce a peripheral neuropathy; (list provided in appendix 1). Patients who
- •can/agree to stop these medications 4 weeks before randomization and during the
- •whole study duration can be included;
- •10. Female of childbearing potential (apart of patients using adequate contraceptive
- •measures), pregnant or breast feeding;
- •11. Known hypersensitivity to any of the individual components of PXT3003;
- •12. Porphyria as it is a contra indication to baclofen, and it may also induce neuropathy;
- •13. Suspected inability to complete the study follow-up (foreign workers, transient visitors,
- •tourists or any others for whom follow-up evaluation is not assured);
- •14. Limited mental capacity or psychiatric disease rendering the subject unable to provide
- •written informed consent or comply with evaluation procedures;
- •15. Patients who have participated in another trial of investigational drug(s) within the past
- •16. If a patient from the same family, living in the same household, has already been
- •included in this study, it will not be possible to include another patient from the same
- •family to avoid mixing of therapeutic units; therefore there would be a risk of inversion
- •of the blind treatments which could jeopardize the interpretation of study results.
研究者
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