EUCTR2018-002097-51-IT进行中（未招募）1 期
A Phase 1/2, Baseline-controlled, Non-randomised, Open-label, Singleascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease - MARVEL1
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
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
- Male
入选标准
- •1. Adult males, = 18 years of age with classic Fabry disease.
- •2. Confirmed diagnosis of classic Fabry disease (including historical documentation of a classic pathological GLA mutation).
- •3. Plasma and/or leucocyte alpha galactosidase activity at screening (measured by central laboratory activity assay at trough) less than 5% of normal according to the central laboratory reference ranges.
- •4. One or more of the characteristic features of classic Fabry disease: neuropathic pain, corneal verticillata, clustered skin angiokeratoma.
- •5. Plasma LysoGb3 levels > 83ng/ml at screening (Part 2 only)
- •6. Estimated glomerular filtration rate (eGFR) =60mL/min/1.73m2 at screening, per serum creatinine Epidemiology Collaboration (CKD-EPI).
- •7. <500mg/g UPCR in a spot urine sample OR <1g/24 hours of urinary protein (24 hour urine analysis), at screening.
- •8. Provision of full informed consent and able to comply with all requirements of the study including 5-year long term follow-up.
- •9. Willing to practice barrier contraception until at least three consecutive semen samples taken at separate visits at least 1 week apart (as specified in the study schedule of assessments) after vector
- •administration are negative for vector sequences.
- •10. Lack of neutralising anti-AAVS3 antibodies using an in vitro transduction inhibition assay within 6 weeks prior to vector administration.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Presence of either R118C, A143T, or other GLA mutations leading to non-classical Fabry disease manifestation and any mutations that have not yet been classified. NOTE: Refer to Fabry Disease mutation database to determine classic and non-classical mutations. E.g., International Fabry Disease Genotype-Phenotype Database
- •(https://lih16.u.hpc.mssm.edu/pipeline/js/dbFabry/Mutation.html).
- •2. Presence of antibodies to aGLA, Replagal, or Fabrazyme as defined by a positive result in the screening assay.
- •3. Subjects with a history of chronic kidney disease, stages 3-5 Kidney Disease: Improving Global Outcomes (KDIGO 2012 classification), documented in medical records for a minimum of 3 months.
- •4. Subjects with severe myocardial fibrosis (=3 segments) identified by MRI at screening.
- •5. Use of investigational therapy for Fabry disease within 60 days before enrolment. In addition, participation in any other clinical study of an investigational medicinal product (IMP), and/or receiving any other IMP during the course of the study
- •6. Elevated ALT, aspartate aminotransferase (AST) and bilirubin >1.5 x upper limit of normal, during screening).
- •7. Platelet count < 100 x 109/L.
- •8. Subjects receiving warfarin or other anticoagulants interfering with the ability to perform renal or skin biopsies, or subjects with a clinically significant bleeding disorder.
- •9. Either history of, or a positive serology test at screening for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCAb), or human immunodeficiency virus (HIV).
- •10. Uncontrolled glaucoma, diabetes mellitus, or hypertension.
- •11. History of malignancy requiring treatment.
- •12. Subjects with uncontrolled cardiac failure, unstable angina, or myocardial infarction in the past 6 months.
- •13. History of acute myocarditis or presence of acute myocarditis during screening.
- •14. Prior treatment with any gene transfer medicinal product.
- •15. Known or suspected intolerance, hypersensitivity or contraindication to replagal, fabrazyme, the IMP and non-investigational medicinal products (NIMPs) or their excipients.
- •16. Subjects who are assessed as having any contraindications to MRI. Including subjects with ferromagnetic metallic implants, including pacing and defibrillator devices, nerve stimulators and cochlear implants.
- •17. Subjects who have had a renal transplant.
- •18. Cytomegalovirus (CMV) Immunoglobulin (Ig)G postive subjects who are CMV polymerase chain reaction (PCR) positive at screening.
研究者
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