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FLT190-01



Short title: FLT190-01

Investigated disease: Morbus Fabry

Principal Investigator: Dr. Sima Canaan-kühl


Clinic, Institute: Medizinische Klinik m.S. Nephrologie und Internistische Intensivmedizin CCM

Patient (diseased)
Age: 18 - 99
Gender: All

Status: Participants wanted
Last change: 25.05.2022

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Study Description

Title

FLT190-01

Short title

FLT190-01

General Short Description

A Phase 1/2, Baseline-controlled, Non-randomised,
Open-label, Single-ascending Dose Study of a Novel
Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease

Scientific Short Description

A Phase 1/2, Baseline-controlled, Non-randomised,
Open-label, Single-ascending Dose Study of a Novel
Adeno-associated Viral Vector (FLT190) in Patients With Fabry disease

EudraCT-No.

2018-002097-51

Investigated Disease, Health Issue

ICD-Code
  • N08.4 - Glomeruläre Krankheiten bei sonstigen endokrinen, Ernährungs- und Stoffwechselkrankheiten

Inclusion Criteria

Age

18 - 99

Gender

All

Additional Inclusion Criteria

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 CKD Epidemiology Collaboration.
7.<500 mg/g UPCR in a spot urine sample OR < 1g/24 hours of urinary protein (24hour 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.

Exclusion Criteria

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 classifieda.
2. Presence of antibodies to αGLA, 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 magnetic resonance imaging (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 alanine aminotransaminase (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 noninvestigational medicinal products (NIMPs) or their excipients.
. 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 positive patients who are CMV polymerase chain reaction (PCR) positive at screening.

Characteristics

Studytype

Interventional

Studyphase

1-2

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