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A Fabry Disease Gene Therapy Study (MARVEL1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04040049
Recruitment Status : Recruiting
First Posted : July 31, 2019
Last Update Posted : February 6, 2020
Sponsor:
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 2021
Estimated Study Completion Date : December 2021


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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

Inclusion Criteria:

  1. Adult males, ≥ 18 years of age with classic Fabry disease.
  2. Confirmed diagnosis of classic Fabry Disease
  3. Decreased plasma and/or leucocyte 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. Able to give full informed consent and able to comply with all requirements of the trial including long term follow-up.
  8. Lack of AAV neutralising antibodies.

Exclusion Criteria:

  1. Non-classical Fabry disease.
  2. Presence of antibodies to αGLA, Replagal, or Fabrazyme.
  3. Patients with chronic kidney disease.
  4. Patients 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. 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).
  9. Uncontrolled glaucoma, diabetes mellitus, or hypertension.
  10. Malignancy requiring treatment.
  11. Patients with uncontrolled cardiac failure, unstable angina, or myocardial infarction in the past 6 months.
  12. Prior treatment with any gene transfer medicinal product.
  13. Patients who have had a renal transplant.

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


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

Locations
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Italy
Universita Federico II di Napoli Recruiting
Napoli, Italy
Norway
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 6, 2020
Last Verified: February 2020

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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
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors