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Trial record 1 of 2 for:    freeline | Fabry Disease
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A Fabry Disease Gene Therapy Study (MARVEL1)

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ClinicalTrials.gov Identifier: NCT04040049
Recruitment Status : Unknown
Verified February 2021 by Freeline Therapeutics.
Recruitment status was:  Recruiting
First Posted : July 31, 2019
Last Update Posted : February 3, 2021
Information provided by (Responsible Party):
Freeline Therapeutics

Brief Summary:
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.

Condition or disease Intervention/treatment Phase
Fabry Disease Lysosomal Storage Diseases Genetic: FLT190 Phase 1 Phase 2

Detailed Description:

Patients who provide consent to participate in this study will be screened for eligibility.

Eligible patients will attend the study site on the day prior to infusion (Day -1) for a baseline visit. On Day 0, FLT190 will be administered as a single dose, slow intravenous infusion. Following FLT190 treatment the patient will be discharged from the investigational site and will continue to be monitored at outpatient visits for a period of 9 months; following which, the patient will enter a period of long-term follow-up conducted under a separate protocol.

The study will be conducted in 2 parts;

Part 1: Enrolment of previously treated patients (Dose escalation)

Part 2: Enrolment of previously untreated patients (Dose expansion).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 8, 2019
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Arm Intervention/treatment
Experimental: FLT190
FLT190 is a replication-incompetent adeno- associated viral (AAV) vector. Administered by a single intravenous infusion.
Genetic: FLT190
Gene Therapy product.

Primary Outcome Measures :
  1. Frequency of treatment-emergent adverse events (AEs) [ Time Frame: From screening to 9 months post infusion ]
    To investigate the safety of systemic administration of FLT190.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Adult males, ≥ 18 years of age with classic Fabry disease.
  2. Confirmed diagnosis of classic Fabry Disease
  3. Decreased plasma alpha galactosidase (αGLA) activity at screening.
  4. One or more of the characteristic features of classic Fabry disease.
  5. Elevated plasma LysoGb3 levels at screening (Part 2 only)
  6. Estimated glomerular filtration rate (eGFR) ≥60mL/min/1.73m2 at screening.
  7. <500 mg/g Urine Protein to Creatinine Ratio (UPCR) in a spot urine sample OR < 1g/24 hours of urinary protein (24hour urine analysis), at
  8. Able to give full informed consent and able to comply with all requirements of the trial including long term follow-up.
  9. Willingness to practice barrier contraception whilst vector shedding via semen is present.
  10. Lack of AAV neutralising antibodies.
  11. For inclusion in Part 1, subjects must have received a licensed ERT or PCT for at least 12 months prior to dosing. For inclusion in Part 2, subjects must never have been previously dosed with ERT or PCT.

Exclusion Criteria:

  1. Non-classical Fabry disease.
  2. Presence of antibodies to αGLA, Replagal, or Fabrazyme.
  3. Subjects with chronic kidney disease.
  4. Subjects with severe myocardial fibrosis.
  5. Use of investigational therapy for Fabry disease within 60 days before enrolment. In addition, participation in any other clinical trial of an investigational medicinal product (IMP), and/or receiving any other IMP during the course of the study
  6. Evidence of liver dysfunction.
  7. Platelet count < 100 xE9L.
  8. Subjects receiving warfarin or other anticoagulants or subjects with a clinically significant bleeding disorder.

9 - 12. Either history of, or a positive serology test at screening for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCAb) and human immunodeficiency virus (HIV) or a negative test at screening for anti-varicella zoster virus (VZV) IgG.

13. Subjects with a history of or a positive screening test for tuberculosis. 14. Subjects who have received a live attenuated vaccination within 12 weeks prior to screening or intend to receive such a vaccine within the course of the study.

15. Uncontrolled glaucoma, diabetes mellitus, or hypertension. 16. Malignancy requiring treatment. 17. Subjects with uncontrolled cardiac failure, unstable angina, or myocardial infarction in the past 6 months.

18. Acute cardiac failure, unstable angina or myocardial infarction in the past 6 months.

19. Prior treatment with any gene transfer medicinal product. 20. Known or suspected intolerance to Replagal, Fabrazyme or any NIMPs used in the study.

21. Subjects with contraindications to MRI. 22. Subjects who have had a renal transplant. 23. Cytomegalovirus immunoglobulin positive subjects who are CMV PCR positive at screening.

24-25.History of physical or psychiatric illness that could affect the subject's ability to participate or a history of substance abuse including alcohol abuse.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04040049

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Contact: Clinical Operations +44 1438 906870 contact@freeline.life

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Charité - Universitätsmedizin Berlin Recruiting
Berlin, Germany
University of Würzburg Recruiting
Würzburg, Germany
Universita Federico II di Napoli Recruiting
Napoli, Italy
Haukeland University Hospital Recruiting
Bergen, Norway
United Kingdom
Royal Free Hospital Recruiting
London, United Kingdom
Sponsors and Collaborators
Freeline Therapeutics
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Responsible Party: Freeline Therapeutics
ClinicalTrials.gov Identifier: NCT04040049    
Other Study ID Numbers: FLT190-01
First Posted: July 31, 2019    Key Record Dates
Last Update Posted: February 3, 2021
Last Verified: February 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Freeline Therapeutics:
Gene Therapy
Additional relevant MeSH terms:
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Fabry Disease
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolic Diseases
Lipid Metabolism Disorders
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors