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Gene Therapy (FLT201) for Gaucher Disease (GALILEO-1 Trial)
GALILEO-1 Trial Summary
This trial will evaluate the safety and effectiveness of a new treatment for Gaucher disease Type 1 in adults.
GALILEO-1 Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowGALILEO-1 Trial Timeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.GALILEO-1 Trial Design
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Who is running the clinical trial?
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- 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.
- Group 1: FLT201
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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
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."
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."
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."
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."
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