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A Gene Therapy Study in Patients With Gaucher Disease Type 1 (GALILEO-1)

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: NCT05324943
Recruitment Status : Recruiting
First Posted : April 13, 2022
Last Update Posted : December 15, 2023
Sponsor:
Information provided by (Responsible Party):
Freeline Therapeutics

Study Description
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Brief Summary:
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability and efficacy of FLT201, and to investigate the relationship of FLT201 dose to augmentation of residual glucocerebrosidase (GCase) expression (activity and concentration), and its potential to improve the clinical phenotype by reduction and prevention of cellular accumulation of GCase substrate.

Condition or disease Intervention/treatment Phase
Gaucher Disease, Type 1 Genetic: FLT201 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)
Actual Study Start Date : April 15, 2022
Estimated Primary Completion Date : November 30, 2024
Estimated Study Completion Date : January 31, 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Arms and Interventions
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Arm Intervention/treatment
Experimental: FLT201
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion.
 Genetic: FLT201
FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events [ Time Frame: Day 1 (dosing) through the final follow-up visit at Week 38 ]
    Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.


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

Inclusion Criteria:

  1. Adult ≥ 18 years of age.
  2. Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis.
  3. All female patients of childbearing potential must not be lactating and must have a negative serum pregnancy test at screening and confirmed negative by urine testing prior to dosing on Day 1. Female patients of childbearing potential and male patients must be willing to follow protocol guidelines for barrier protection/contraception.
  4. Able to give full informed consent for the trial.
  5. Treatment status at screening (screening period is 16 weeks):

Treated with either enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) and started this treatment at least 2 years prior to dosing with no change in regimen for the prior 3 months. ERT dose ≥15 U/kg and ≤60 U/kg every other week.

Exclusion Criteria:

  1. Diagnosed or suspected Type 2 or Type 3 Gaucher disease (including any patient with eye movement abnormality on clinical examination).
  2. Positive for neutralising antibodies to AAVS3 at screening.
  3. Evidence of significant and persistent liver dysfunction at Screening defined as >1.5 x upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.

  4. Evidence of any of the following at screening:

    1. Hb <8 g/dL.
    2. Platelets <45,000/mm3.
    3. Pulmonary hypertension.
    4. New osteonecrosis within 12 months of screening.
    5. Fragility fracture or bone crisis within 12 months of screening.
  5. Hepatitis B surface antigen (HBsAg) positive at screening.
  6. Hepatitis C antibody (Hep C Ab) positive and hepatitis C RNA polymerase chain reaction (PCR) (as follow-up test if Hep C Ab-positive)-positive at screening.
  7. Cytomegalovirus (CMV) immunoglobulin G (IgG) and CMV DNA PCR-positive at screening.
  8. Human immunodeficiency virus (HIV)-1 or -2 antibody positive at screening.
  9. Patient has received live attenuated vaccination within 12 weeks prior to screening or intends to receive such vaccination during the study.
  10. History of clinically-advanced liver disease e.g. cirrhosis, portal hypertension.
  11. History of bone marrow transplant.
  12. History of splenectomy (partial or total).
  13. History of splenic infarct within 12 months of screening.
  14. History of receiving any gene transfer medicinal product.
  15. History of receiving any investigational therapy for Gaucher disease within 60 days of screening.
  16. Participation in any other clinical study of an investigational medicinal product (IMP), and/or receiving any other IMP during the study.
  17. History of idiopathic thrombocytopaenic purpura, thrombotic thrombocytopaenic purpura, thrombocytopaenia, anaemia, hepatomegaly, splenomegaly, and/or osteoporosis, unrelated to Gaucher disease.
  18. History of, or active neoplastic disease within 5 years of screening (except for basal or squamous cell carcinoma of the skin or carcinoma in situ which has been definitively treated).
  19. Subjects with uncontrolled cardiac failure, unstable angina, myocardial infarction, pulmonary hypertension or cardiac presentations including cardiac instability deemed significant by the investigator in the past 6 months
  20. History of acute myocarditis or presence of acute myocarditis during screening.
  21. History of substance abuse, including alcohol abuse or alcohol dependence.
  22. Known or suspected intolerance, hypersensitivity or contraindication to the investigational medicinal product (IMP) and non-investigational medicinal products (NIMPs) or their excipients.
  23. History of anaphylaxis or infusion related reactions to ERT.
  24. Contraindication(s) to MRI. (e.g. ferromagnetic metallic implants, some types of pacing and defibrillator devices, nerve stimulators).
  25. Any clinical condition (medical or psychiatric) that, in the opinion of the investigator, could jeopardise safety or compromise ability of the patient to participate in this study.
Contacts and Locations
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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): NCT05324943


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

Locations
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United States, California
Kaiser Permanente Recruiting
Los Angeles, California, United States, 90027
Contact: Divya Vats    323-361-5704    DIVYA.VATS@kp.org   
Principal Investigator: Divya Vats         
United States, New York
New York Presbyterian/Columbia University Irving Medical Center (NYPH/CUMC) Not yet recruiting
New York, New York, United States, 10032
Contact: Gustavo Maegawa    212-305-6731    lsdprogram@cumc.columbia.edu   
Principal Investigator: Gustavo Maegawa         
United States, Virginia
Lysosomal Rare Disorders Research and Treatment Center Recruiting
Fairfax, Virginia, United States, 22030-6066
Contact: Lauren Noll    703-261-6220    lnoll@ldrtc.org   
Contact: Ozlem Goker-Alpan    (703) 2616220    ogoker-alpan@ldrtc.org   
Principal Investigator: Ozlem Goker-Alpan         
Brazil
Hospital de Clinicas de Porto Alegre (HCPA) Recruiting
Porto Alegre, Brazil
Contact: Ida Schwartz    +55 51 999017418    idadschwartz@gmail.com   
Principal Investigator: Ida Vanessa Schwartz         
Germany
Universitätsklinikum Hamburg Eppendorf Recruiting
Hamburg, Germany
Principal Investigator: Nicole Muschol         
SphinCS Recruiting
Höchheim, Germany
Contact: Eugen Mengel    0049-6146-904820    info@sphincs.de   
Principal Investigator: Eugen Mengel         
Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel
Contact: Ari Zimran    +972-2-6555143    azimran@gmail.com   
Principal Investigator: Shoshana Revel-Vilk         
Rabin Medical Center - PPDS Recruiting
Petah Tikva, Israel
Contact: Noa Lev-El Halabi    +972-3-9377522    noale1@clalit.org.il   
Principal Investigator: Noa Ruhrman Shahar         
Tel Aviv Sourasky Medical Center Recruiting
Tel Aviv, Israel
Contact: Dorin Trigubov    +972-3-6974905    dorint@tlvmc.gov.il   
Principal Investigator: Hagit Baris-Feldman         
Paraguay
Instituto Privado de Hematologia e Investigaciones Clinicas Completed
Asunción, Paraguay
Spain
Hospital Universitario de Bellvitge Recruiting
Barcelona, Spain
Contact: Xavier Solanich Moreno    932 60 75 00 ext 2324    xsolanich@bellvitgehospital.cat   
Principal Investigator: Xavier Solanich Moreno         
Hospital Universitario Vall d'Hebrón Recruiting
Barcelona, Spain
Contact: Maria Camprodon    (0034)677072506    maria.camprodon@vallhebron.cat   
Principal Investigator: Maria Camprodon         
Hospital Universitario Ramon y Cajal Recruiting
Madrid, Spain
Contact: Esther Agea    91 3368967    ec.hemato.esther.agea@gmail.com   
Principal Investigator: Jesus Villarrubia         
Hospital Quironsalud Zaragoza Recruiting
Zaragoza, Spain
Contact: Pilar Giraldo    +34670285339    giraldocastellano@gmail.com   
Principal Investigator: Pilar Giraldo         
United Kingdom
Royal Free Hospital Recruiting
London, United Kingdom
Contact: Derralynn Hughes    02077940500    derralynnhughes@nhs.net   
Principal Investigator: Derralynn Hughes         
Salford Royal Hospital Recruiting
Salford, United Kingdom
Contact: Marie Meehan    07799797743    Marie.meehan@nca.nhs.uk   
Principal Investigator: Reena Sharma         
Sponsors and Collaborators
Freeline Therapeutics
More Information
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Responsible Party: Freeline Therapeutics
ClinicalTrials.gov Identifier: NCT05324943    
Other Study ID Numbers: FLT201-01
First Posted: April 13, 2022    Key Record Dates
Last Update Posted: December 15, 2023
Last Verified: December 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders