A Fabry Disease Gene Therapy Study (MARVEL1)

  • End date
    Dec 15, 2022
  • participants needed
  • sponsor
    Freeline Therapeutics
Updated on 14 February 2022
gene therapy


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.


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).

Condition Fabry Disease, Lysosomal Storage Diseases
Treatment FLT190
Clinical Study IdentifierNCT04040049
SponsorFreeline Therapeutics
Last Modified on14 February 2022


Yes No Not Sure

Inclusion Criteria

Adult males, 18 years of age with classic Fabry disease
Confirmed diagnosis of classic Fabry Disease
Decreased plasma alpha galactosidase (GLA) activity at screening
One or more of the characteristic features of classic Fabry disease
Elevated plasma LysoGb3 levels at screening (Part 2 only)
Estimated glomerular filtration rate (eGFR) 60mL/min/1.73m2 at screening
Able to give full informed consent and able to comply with all requirements of the trial including long term follow-up
<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
Lack of AAV neutralising antibodies
Willingness to practice barrier contraception whilst vector shedding via semen is present
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

Non-classical Fabry disease
Presence of antibodies to GLA, Replagal, or Fabrazyme
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
Subjects with chronic kidney disease
Evidence of liver dysfunction
Platelet count < 100 xE9L
Subjects with severe myocardial fibrosis
Subjects receiving warfarin or other anticoagulants or subjects with a clinically significant bleeding disorder
- 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
\. Subjects with a history of or a positive screening test for tuberculosis
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
\. 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
\. Acute cardiac failure, unstable angina or myocardial infarction in the
past 6 months
\. Prior treatment with any gene transfer medicinal product. 20. Known or
suspected intolerance to Replagal, Fabrazyme or any NIMPs used in the study
\. 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
-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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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