NL-OMON50474已完成3 期
A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. - MODIFY
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 4
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
- •Screening visit criteria
- •1. Signed and dated ICF prior to any study-mandated procedure;
- •2. Male or female subjects; 18-years old and above;
- •3. FD diagnosis confirmed with local genetic test results (i.e., resence of at
- •least 1 mutation in GLA, the gene coding for a-galactosidase A *a-GalA*);
- •4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3
- •months prior to screening;
- •5. ERT treatment status:
- •a) Subject never treated with ERT; or
- •b) Subject has not received ERT for at least 6 months prior to screening; or
- •c) Subject treated with ERT at the time of the screening visit and meeting all
- •of the following criteria at the time of screening:
- •i) ERT administration for the last 12 months;
- •ii) Stable ERT dose regimen during the last 3 months;
- •iii) Subject agrees to stop ERT administration at the screening visit for
- •approximately 8 months (6-7 weeks screening + 6 months of double-blind
- •treatment).
- •6. A woman of childbearing potential [see definition in Section 4.5.1 of the
- •core protocol] is eligible only if the following applies:
- •- Negative serum pregnancy test at screening and a negative urine pregnancy
- •test at randomization;
- •- Agreement to undertake monthly urine pregnancy tests during the study and up
- •to at least 30 days after study treatment discontinuation;
- •- Agreement to follow a highly effective contraception scheme as described in
- •Section 4.5.2 of the core protocol from screening up to at least 30 days after
- •study treatment discontinuation.
- •- Agreement not to donate ova from screening visit and up to 30 days after
- •study treatment discontinuation.
- •7. A fertile male (physiologically capable of conceiving a child according to
- •investigator judgment) who is sexually active with a woman of childbearing
- •potential is eligible only if the following applies:
- •- Agreement to use a condom during the treatment period (starting at
- •randomization) and for up to 3 months after study treatment discontinuation; and
- •- Agreement not to father a child during this period.
- •In addition, male subjects must agree not to donate sperm (except for study
- •semen sampling) during the treatment period (starting at randomization) and for
- •up to 3 months after study treatment discontinuation.
- •Randomization visit criteria
- •8. Adequate subject compliance with completion of an electronic diary (eDiary)
- •during the screening period;
- •9. Subjects with moderate or severe neuropathic pain as determined from daily
- •entries of the modified Brief Pain Inventory-Short Form item 3 (BPI-SF3) score
- •of *neuropathic pain at its worst in the last 24 hours* in the eDiary during
- •the screening period.
排除标准
- •Screening visit criteria:
- •Disease/condition:
- •1. Pregnant / planning to become pregnant up to 30 days after study treatment
- •discontinuation or lactating subject;
- •2. Severe renal insufficiency defined as an estimated glomerular filtration
- •rate (eGFR) per the Chronic Kidney Disease Epidemiology Collaboration
- •creatinine equation < 30 mL/min/1.73 m2 at screening (as reported by the
- •central laboratory);
- •3. Subject on regular dialysis for the treatment of chronic kidney disease;
- •4. Subject has undergone, or is on a waiting list for, or is scheduled to
- •undergo kidney or other organ transplantation.
- •5. Known and documented transient ischemic attack, stroke, unstable angina or
- •myocardial infarction within 6 months prior to screening;
- •6. Clinically significant unstable cardiac disease in the opinion of the
- •investigator (e.g., uncontrolled symptomatic arrhythmia, New York Heart
- •Association class III or IV congestive heart failure);
- •7. Any other subject at high risk of developing clinical signs of organ
- •involvement within the time period of the study, as per investigator judgment.
- •8. Any known factor or disease that might interfere with treatment compliance,
- •study conduct or interpretation of the results such as:
- •a) Other disease or condition associated with a pain component that could
- •confound assessment of neuropathic pain (e.g., diabetic neuropathy,
- •chemotherapy- or radiation-induced peripheral neuropathy, chronic inflammatory
- •demyelinating polyneuropathy);
- •b) Other disease of the GI tract that could interfere with the assessment of GI
- •symptoms in FD (e.g., inflammatory bowel disease);
- •c) Documented poorly controlled diabetes mellitus (i.e., HbA1c > 8.0% at
- •screening as reported by the central laboratory);
- •d) Significant neurological disorder;
- •e) Significant psychiatric disease; suicidal ideation at screening or history
- •of suicide attempt or behavior within 6 months prior to screening as per
- •investigator judgment;
- •f) History of drug dependence (including opioids) or alcohol dependence;
- •g) Inability to complete an eDiary on a daily basis.
- •9. Known concomitant life-threatening disease with a life expectancy < 18
- •Treatments:
- •10. Subject planned for imminent initiation of treatment with ERT;
- •11. Known hypersensitivity to lucerastat or drug of the same chemical class of
- •iminosugars (e.g., miglitol, miglustat, migalastat), or any of their excipients;
- •12. Initiation or treatment at an unstable dose within 4 weeks prior to
- •screening with any of the following medications:
- •a) Angiotensin-converting enzyme (ACE) inhibitor and/or angiotensin receptor
- •blocker (ARB);
- •b) Anti-epileptic;
- •c) Tricyclic antidepressant (TCA) and/or other antidepressants belonging to the
- •serotonin-norepinephrine re-uptake inhibitor (SNRI) and selective serotonin
- •re-uptake inhibitor (SSRI) classes.
- •13. Planned or current treatment with another investigational treatment within
- •3 months prior to screening;
- •14. Treatment with any inhibitor of the glucosylceramide synthase (GCS) (e.g.,
- 另有 4 项未显示
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