NL-OMON46978已完成3 期
Phase III, multi-center, randomized, 48 weeks, double-blind, parallel-group, placebo-controlled study to evaluate efficacy and safety of CER-001 on vessel wall area in patients with genetically definded familial primary hypoalphalipoproteinemia and receiving background optimized lipid therapy, with optional open-label safety extension - TANGO
Cerenis Therapeutics SA0 个研究点目标入组 10 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
研究概览
简要总结
Trial ended prematurely
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •Patients with apoA-I <= 110 mg/dL and HDL <= 35 mg/dL, with suspected homozygous or heterozygous mutation in the ABCA1, and/or ApoA-I genes confirmed by genetic testing, and background symptomatic or asymptomatic cardiovascular disease will be eligible for this study.
- •1. Male and female patients, aged 18 and above.
- •2. Female patients who are not either surgically sterile (e.g., tubal ligation or removal of ovaries or uterus) or post-menopausal (no spontaneous menstrual periods for at least one year) must agree to use one of the following forms of contraception from screening until 90 days after the completion of the study medication:(1) systemic hormonal treatment (2) an IUD which was implanted at least 2 months prior to screening or (3) double-barrier contraception (condom, diaphragm and spermicide are each considered a barrier), or (4) agree to remain sexually abstinent during the entire study period (when contraception is not acceptable for cultural or religious beliefs).
- •3. Sign written informed consent after the scope and nature of the investigation have been explained to them before screening evaluations and willing to comply with the study restrictions.
- •4. Are fluent in the language of the investigator, study staff (including raters), and the informed consent.
- •5. Diagnosis of genetically confirmed HDL-c deficiency due to defects in genes coding for e.g. ABCA1 and/or ApoA -I.
- •6. IF the subject is on lipid lowering therapy or NEEDS to be treated with lipid lowering therapy then the subject must be on a stable dose at least 6 weeks prior to baseline procedures.
- •7. Background symptomatic or a-symptomatic cardiovascular disease should been present as such:
- •- for symptomatic cardiovascular disease: i) history of cardio or cerebrovascular events, ii) diagnosed coronary artery disease (CAD), iii) diagnosed carotid or peripheral stenosism iv) previous myocardial revascularisation- percutaneous coronary intervention (PCI) , or coronary artery bypass graft (CABG).
- •- for asyptomatic cardiovascular disease: patients with subclinical atherosclerosis diagnosed using imaging method such as i) vDoppler ultrasound, ii) vB-mode ultrasonography- measurement of carotid intima media thickness, iii) v intravascular ultrasonography, iv) Computed Tomography, v) Magnetic Resonance Imaging.
- •8. ApoA-1 <= 110 mg/dL.
- •9. HDL cholesterol <= 35 mg/dL or 0.09 mmol/L
排除标准
- •Patients meeting any of the following criteria are not eligible for the study:
- •1. Patient with LCAT mutation will be excluded;
- •2. Patient who experienced a cardiovascular event within 6 months prior to the onset of screening;
- •3. Patient who experienced stroke or other cerebrovascular event within 1 year prior to the onset of screening;
- •4. Patient with triglycerides level above 500 mg/dL;
- •5. The patient has evidence of clinically significant, uncontrolled or unstable cardiovascular, renal, hepatic (incl. AST or ALT at or above 3x ULN, or bilirubin at or above 2x ULN), gastrointestinal, hematologic, immunological, neurological, endocrine, metabolic or pulmonary disease (as determined by medical history, clinical laboratory or ECG results, or physical examination) or any other medical disorder that would increase the risk associated with taking study medication or would confound the interpretation of study results;
- •6. Patients with a body mass index (BMI) < 17 kg/m2 or > 40 kg/m2;
- •7. Patients with severe anemia defined as hemoglobin level below or equal to 10 g/dL;
- •8. Any clinically significant abnormal laboratory data, vital signs, physical examination at screening or baseline, which in the opinion of the investigator, would interfere with safety assessments;
- •9. Clinically significant electrocardiogram (ECG) abnormality at screening, including sinus bradycardia (resting heart rate < 50 beats per minute), 2nd or 3rd degree atrioventricular block, prolonged QTc (QTcF >= 450 ms in males and >= 470 ms in females) history of congenital long QT syndromes, or risk of Torsades de Pointes because of family history of sudden death, etc.;
- •10. Positive result on the serum pregnancy test or are breast feeding at screening, or intend to become pregnant during the course of the trial;
- •11. Male intending to father a child during the study;
- •12. Likely to be unreliable as a study participant based on the Investigator's (or designee*s) knowledge of the patient (e.g., alcohol or other drug abuse, inability or unwillingness to adhere to the protocol, or psychosis);
- •13. Symptomatic (NYHA Class II or greater) congestive heart failure requiring and persisting despite appropriate medical treatment;
- •14. Uncontrolled blood pressure: systolic blood pressure >= 160 mmHg and/or diastolic blood pressure >=100 mmHg at screening or any other pre-randomization visit;
- •15. Uncontrolled diabetes mellitus defined as HbA1c >10%;
- •16. Unexplained creatine phosphokinase level > 3 times the ULN;
- •17. History of malignancy during the 3 years prior to screening, with the exception of basal cell carcinoma of the skin;
- •18. Current alcohol or drug abuse or history thereof within 5 years prior to screening
- •19. Contraindication to MRI scanning such as imbedded metal (e.g., schrapnel), implanted metal objects (e.g., pacemaker), claustrophobia;
- •20. Participated in any investigational study or taken an investigational drug within 30 days (or 5 times the half-life of the investigational drug, whatever is longer);
- •21. Ever received CER-001within 6 month from the onset of screening;
- •22. Medically non-compliant in the management of their disease in the investigator*s opinion.
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