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

December 14, 2023 updated by: Freeline Therapeutics

A Phase 1, Open-label, Safety, Tolerability, and Efficacy Study of FLT201 in Adult Patients With Gaucher Disease Type 1 (GALILEO-1)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Brazil
      • Porto Alegre, Brazil
        • Recruiting
        • Hospital de Clinicas de Porto Alegre (HCPA)
        • Principal Investigator:
          • Ida Vanessa Schwartz
        • Contact:
  • Germany
      • Hamburg, Germany
        • Recruiting
        • Universitätsklinikum Hamburg Eppendorf
        • Principal Investigator:
          • Nicole Muschol
      • Höchheim, Germany
        • Recruiting
        • SphinCS
        • Principal Investigator:
          • Eugen Mengel
        • Contact:
  • Israel
      • Jerusalem, Israel
        • Recruiting
        • Shaare Zedek Medical Center
        • Principal Investigator:
          • Shoshana Revel-Vilk
        • Contact:
      • Petah Tikva, Israel
        • Recruiting
        • Rabin Medical Center - PPDS
        • Principal Investigator:
          • Noa Ruhrman Shahar
        • Contact:
      • Tel Aviv, Israel
        • Recruiting
        • Tel Aviv Sourasky Medical Center
        • Principal Investigator:
          • Hagit Baris-Feldman
        • Contact:
  • Paraguay
      • Asunción, Paraguay
        • Completed
        • Instituto Privado de Hematologia e Investigaciones Clinicas
  • Spain
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitario Vall d'Hebron
        • Principal Investigator:
          • Maria Camprodon
        • Contact:
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitario de Bellvitge
        • Principal Investigator:
          • Xavier Solanich Moreno
        • Contact:
      • Madrid, Spain
        • Recruiting
        • Hospital Universitario Ramón y Cajal
        • Principal Investigator:
          • Jesus Villarrubia
        • Contact:
      • Zaragoza, Spain
        • Recruiting
        • Hospital Quironsalud Zaragoza
        • Principal Investigator:
          • Pilar Giraldo
        • Contact:
  • United Kingdom
      • London, United Kingdom
        • Recruiting
        • Royal Free Hospital
        • Principal Investigator:
          • Derralynn Hughes
        • Contact:
      • Salford, United Kingdom
        • Recruiting
        • Salford Royal Hospital
        • Principal Investigator:
          • Reena Sharma
        • Contact:
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Kaiser Permanente
        • Principal Investigator:
          • Divya Vats
        • Contact:
    • New York
      • New York, New York, United States, 10032
        • Not yet recruiting
        • New York Presbyterian/Columbia University Irving Medical Center (NYPH/CUMC)
        • Principal Investigator:
          • Gustavo Maegawa
        • Contact:
    • Virginia
      • Fairfax, Virginia, United States, 22030-6066
        • Recruiting
        • Lysosomal Rare Disorders Research and Treatment Center
        • Principal Investigator:
          • Ozlem Goker-Alpan
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Day 1 (dosing) through the final follow-up visit at Week 38

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Freeline Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 15, 2022

Primary Completion (Estimated)

November 30, 2024

Study Completion (Estimated)

January 31, 2025

Study Registration Dates

First Submitted

February 28, 2022

First Submitted That Met QC Criteria

April 8, 2022

First Posted (Actual)

April 13, 2022

Study Record Updates

Last Update Posted (Estimated)

December 15, 2023

Last Update Submitted That Met QC Criteria

December 14, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FLT201-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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