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Gene Therapy
Gene Therapy (FLT201) for Gaucher Disease (GALILEO-1 Trial)
Phase 1
Recruiting
Research Sponsored by Freeline Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1 (dosing) through the final follow-up visit at week 38
Awards & highlights
GALILEO-1 Trial Summary
This trial will evaluate the safety and effectiveness of a new treatment for Gaucher disease Type 1 in adults.
Who is the study for?
Adults over 18 with Gaucher Disease Type 1, on stable enzyme or substrate therapy for at least 2 years, can join this gene therapy study. They must have low GCase enzyme activity and agree to use contraception. Excluded are those with recent cancer (except certain skin cancers), past gene therapies, bone diseases not due to Gaucher's, severe liver dysfunction, blood disorders unrelated to Gaucher's, certain infections like HIV/Hepatitis C/CMV, allergies to the treatment components or a history of substance abuse.Check my eligibility
What is being tested?
FLT201 is being tested for safety and effectiveness in increasing GCase enzyme levels and reducing symptoms in Gaucher Disease Type 1 patients. This first-in-human trial will also explore how different doses affect these outcomes.See study design
What are the potential side effects?
As FLT201 is a new treatment under investigation, potential side effects include immune reactions such as inflammation or allergy-like responses due to the body recognizing it as foreign; however specific side effects will be monitored throughout the trial.
GALILEO-1 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have Gaucher disease Type 1 with low GCase enzyme activity.
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I am not pregnant, not breastfeeding, and willing to use birth control as per the study's requirements.
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I've been on the same enzyme or substrate therapy for over 2 years without changes in the last 3 months.
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I am 18 years old or older.
GALILEO-1 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day 1 (dosing) through the final follow-up visit at week 38
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1 (dosing) through the final follow-up visit at week 38
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of treatment-emergent adverse events
GALILEO-1 Trial Design
1Treatment groups
Experimental Treatment
Group I: FLT201Experimental Treatment1 Intervention
FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion.
Find a Location
Who is running the clinical trial?
Freeline TherapeuticsLead Sponsor
5 Previous Clinical Trials
59 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your hemoglobin level is less than 8 grams per deciliter.I have had a partial or complete spleen removal.I am not currently participating in another clinical trial or taking any investigational medicines.Your liver function tests show significant and ongoing liver problems.I have developed new bone tissue death within the last year.I have a history of blood disorders or liver, spleen, bone issues not related to Gaucher disease.I have had a spleen injury in the last year.I am allergic or react badly to the trial medication or its ingredients.I have been diagnosed with or suspected to have Type 2 or Type 3 Gaucher disease.I have had acute myocarditis or it was found during my screening.I have Gaucher disease Type 1 with low GCase enzyme activity.I have not had cancer, except for certain skin cancers or treated early-stage cancers, in the last 5 years.I am not pregnant, not breastfeeding, and willing to use birth control as per the study's requirements.I have tested positive for CMV both through antibody and DNA tests.You have tested positive for HIV antibodies during screening.I have had a fracture or severe bone issue in the last year.Your platelet count is less than 45,000 per cubic millimeter.I have a serious liver condition, like cirrhosis.You have had a severe allergic reaction or infusion-related reactions to enzyme replacement therapy (ERT) in the past.You cannot have an MRI scan because you have certain metal implants or medical devices.I've been on the same enzyme or substrate therapy for over 2 years without changes in the last 3 months.I have received gene therapy before.I am 18 years old or older.You have a history of using drugs or drinking alcohol excessively.I have high blood pressure in the lungs.You have developed antibodies that can block the effects of AAVS3.I have tested positive for Hepatitis C.I have not had a live vaccine in the last 12 weeks and do not plan to during the study.I have had a bone marrow transplant.I have not had serious heart problems in the last 6 months.You tested positive for hepatitis B surface antigen (HBsAg) during screening.
Research Study Groups:
This trial has the following groups:- Group 1: FLT201
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is FLT201 a risk-free approach for patients?
"FLT201's safety was judged to be a 1 on the scale due to its Phase 1 status, which signals there is minimal evidence for both efficacy and risk."
Answered by AI
What is the scope of participants for this trial?
"A total of 18 qualified patients must enroll in this clinical study. Those hoping to participate may do so from Ann and Robert H Lurie Childrens Hospital of Chicago located in the state of Illinois or Columbia University Irving Medical Center situated in New york City, NY."
Answered by AI
How many medical institutions are hosting this experimental investigation?
"This clinical trial is currently running in 24 medical facilities, located both locally and abroad. In order to reduce travel expenses, those enrolling should consider the location closest to them. Specifically, sites are available in Chicago, New york City and Pittsburgh as well other urban regions."
Answered by AI
Are there any vacancies available for prospective participants in this trial?
"Affirmative. Data from clinicaltrials.gov attests that the medical trial, which was originally posted on April 15th 2022 is presently accepting participants. 18 individuals must be recruited between 24 distinct locations."
Answered by AI
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