EUCTR2011-004287-30-DK进行中（未招募）1 期
A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase Deficiency
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Subject will be > 8 months of age at the time of dosing.
- •2. Subject or subject's parent or legal guardian consents to participation in the study. If the subject is of minor age, he/she is willing to provide assent where required per local regulations, and if deemed able to do so.
- •3. Confirmation of LALD diagnosis as determined by the central lab; a subject who received a liver or hematopoietic stem cell transplant who does not show evidence of LAL enzyme deficiency by DBS due to the effects of transplantation must have either:a. Molecular genetic testing which confirms mutations in both alleles of the LIPA gene; ORb. Appropriately documented historical result of an enzyme test prior to hematopoietic or liver transplantation.
- •4. Subjects > 8 months but < 4 years of age at Screening will have at least 1 of the following documented clinical manifestations of LALD:
- •a. Dyslipidemia (defined as Screening LDL-C > 130 mg/dL; TG > 200 mg/dL);
- •b. Elevated transaminases (ALT =1.5x ULN);
- •c. Impaired growth as defined as:
- •i. WFA or SFA less than the age- and gender-appropriate 5th percentile on a standard WHO (subjects = 24 months of age) or CDC (subjects > 24months and < 4 years of age) WFA or SFA chart for at least 3 months prior to study entry; OR
- •ii. Poor weight gain as evidenced by calculated weight percentile decreasing across 2 major percentile (99th, 97th, 95th, 90th, 75th, 50th,25th, 10th, 5th, 3rd, and 1st) lines on a standard WHO (subjects =24 months of age) or CDC subjects > 24 months and <4 years of age) WFA chart over a period of 6 months prior to study entry;
- •d. Suspected malabsorption with:
- •i. Persistent unexplained gastrointestinal symptoms such as nausea, diarrhea, abdominal pain, and bloating; OR
- •ii. Unexplained anemia, or other abnormalities suggestive of malabsorption; AND
- •iii. Documented small intestinal disease involvement on a small bowel biopsy performed within 1 year of Screening e. Other clinical manifestation of LALD in the opinion of the investigator and in consultation with the Sponsor.
- •5. Subjects = 4 years of age at Screening will have at least 1 of the following documented clinical manifestations of LALD:
- •a. Evidence of advanced liver disease at Screening accompanied by:
- •i. Clinically significant portal hypertension as defined by a hepatic venous pressure gradient (HVPG) greater than or equal to 10 mmHg; OR
- •ii. Documented esophageal varices at Screening.
- •b. Disease recurrence in subjects with past liver or hematopoietic transplants;
- •c. Persistent dyslipidemia (defined as LDL-C > 130mg/dL, triglycerides >200mg/dL, or HDL-C <40mg/dL in males, and< 50mg/dL in females) that has persisted despite 3 or more months of treatment with one or more lipid-lowering therapies such as statins, cholesterol absorption inhibitors (ezetimibe), combination therapies (single-pill; ezetimibe/simvastatin, niacin/simvastatin), fibrates (fenofibrate, gemfibrozil, fenofibric acid), niacin or bile acid sequestrants (cholestyramine, colestipol, colesevelam);
- •d. Suspected malabsorption based on the following manifestations:
- •i. Documented small intestinal involvement by small bowel biopsy performed within 1 year of Screening; AND
- •ii. Unexplained iron deficiency, osteopenia, weight loss or chronic diarrhea; OR
- •iii. Impaired growth in pediatric subjects defined as:
- •1. WFA or SFA less than the age- and gender-appropriate 5th percentile on a standard CDC WFA chart for at least 6 months prior to study entry; OR
排除标准
- •1. Subject meets eligibility criteria for another interventional study of sebelipase alfa in LALD that is open for enrollment in the region where the subject will receive treatment.
- •2. Subject has known causes of active liver disease other than LALD which have not been adequately treated
- •3. Subject is unable or unwilling to comply with study procedures.
- •4. Subject received a hematopoietic stem cell or liver transplant < 2 years from the time of dosing.
- •5. Females who are nursing or pregnant.
- •6. Subject with co-morbidities other than complications due to LALD which, in the opinion of the Investigator and in consultation with the Sponsor, are irreversible or associated with a high mortality risk within 6months, or would interfere with study compliance or data interpretation.
- •7. Exposure to any investigational product within 30 days of Screening for a small molecule and 60 days of Screening for a biologic.
- •8. Known hypersensitivity to eggs.
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