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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

Tracking Information
First Submitted Date  ICMJE July 26, 2019
First Posted Date  ICMJE July 31, 2019
Last Update Posted Date February 6, 2020
Actual Study Start Date  ICMJE July 8, 2019
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 30, 2019)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Fabry Disease Gene Therapy Study
Official Title  ICMJE 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
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.
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).

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Fabry Disease
  • Lysosomal Storage Diseases
Intervention  ICMJE Genetic: FLT190
Gene Therapy product.
Study Arms  ICMJE Experimental: FLT190
FLT190 is a replication-incompetent adeno- associated viral (AAV) vector. Administered by a single intravenous infusion.
Intervention: Genetic: FLT190
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 4, 2020)
15
Original Estimated Enrollment  ICMJE
 (submitted: July 30, 2019)
12
Estimated Study Completion Date  ICMJE December 2021
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Operations +44 1438 906870 contact@freeline.life
Listed Location Countries  ICMJE Italy,   Norway,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04040049
Other Study ID Numbers  ICMJE FLT190-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Freeline Therapeutics
Study Sponsor  ICMJE Freeline Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Freeline Therapeutics
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP