Gene Therapy (FLT201) for Gaucher Disease

(GALILEO-1 Trial)

No longer recruiting at 23 trial locations

Overseen By Clinical Operations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Freeline Therapeutics

Must be taking: Enzyme replacement, Substrate reduction

Disqualifiers: Type 2/3 Gaucher, Liver dysfunction, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called FLT201 for individuals with Gaucher disease Type 1. Researchers aim to determine the treatment's safety, effectiveness, and potential to reduce symptoms by improving enzyme levels in the body. It is available for adults diagnosed with Gaucher disease Type 1 who are currently on stable enzyme replacement or substrate reduction therapy. As a Phase 1 trial, participants will be among the first to receive this treatment, aiding researchers in understanding its effects in people.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop your current medications. However, if you are on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), you must have been on a stable dose for at least 3 months before joining the trial.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

A previous study found FLT201 to be safe for over two years. Patients tolerated this gene therapy well, with no serious side effects causing them to stop treatment. Early results also show that FLT201 might address issues like the buildup of certain substances in the body, which current treatments don't completely resolve. This suggests that FLT201 could be a safe and effective option for people with Gaucher disease Type 1. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for Gaucher Disease, which often involve enzyme replacement therapies or substrate reduction therapies, FLT201 is a gene therapy that offers a potentially transformative approach. This treatment is unique because it uses a single intravenous infusion to deliver a therapeutic gene, aiming to provide long-term benefits by addressing the root cause of the disease rather than just managing symptoms. Researchers are excited about FLT201's potential to significantly reduce treatment frequency and improve the quality of life for patients with Gaucher Disease.

What evidence suggests that FLT201 might be an effective treatment for Gaucher disease?

Research has shown that FLT201, a new gene therapy under study in this trial, could be a promising treatment for Gaucher disease Type 1. In early studies, patients experienced a lasting reduction in lyso-Gb1 levels, an important disease marker, by 42% to 96%. This significant drop suggests that the therapy might effectively manage the disease's symptoms. Additionally, some patients continued to benefit for up to two years after stopping their regular treatments. Early research also supports the idea that FLT201 could provide long-lasting benefits by addressing the disease's root causes. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have Gaucher disease Type 1 with low GCase enzyme activity.

I am not pregnant, not breastfeeding, and willing to use birth control as per the study's requirements.

I've been on the same enzyme or substrate therapy for over 2 years without changes in the last 3 months.

Exclusion 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.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of FLT201

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

38 weeks

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• FLT201

Trial Overview 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.

How Is the Trial Designed?

Group I: FLT201 Experimental Treatment 1 Intervention

FLT201 is an advanced therapy investigational medicinal product (ATIMP) administered as a single intravenous infusion.

Who Is Running the Clinical Trial?

Freeline Therapeutics

Lead Sponsor

Trials

7

Recruited

150+

Spur Therapeutics

Lead Sponsor

Trials

7

Recruited

150+

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S1525001625003727

FLT201, a novel liver-directed AAV gene therapy candidate ... Preclinical data indicate that FLT201 could offer a durable treatment for Gaucher disease type 1, addressing unmet needs related to substrate ...

2. cgtlive.com cgtlive.com/view/patients-treated-spur-therapeutics-gaucher-disease-gene-therapy-flt201-maintain-clinical-benefit-2-years

Patients Treated With Spur Therapeutics' Gaucher Disease ... The efficacy data included 4 patients who had discontinued their SOC therapy in a time frame of 4 to 11 weeks after being treated with FLT201.

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40340248/

FLT201, a novel liver-directed AAV gene therapy candidate ... Preclinical data indicate that FLT201 could offer a durable treatment for Gaucher disease type 1, addressing unmet needs related to substrate ...

4. neurologylive.com neurologylive.com/view/gene-therapy-flt201-shows-promise-early-stage-study-gaucher

Gene Therapy FLT201 Shows Promise in Early-Stage ... In the multicenter study, treatment with the gene therapy led to durable reductions, ranging from 42% to 96%, in lyso-Gb1 levels, considered one ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05324943

NCT05324943 | A Gene Therapy Study in Patients With ... An arm type in which a group of participants receives an intervention/treatment considered to be effective (or active) by health care providers. Adverse event.

6. cell.com cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(25)00372-7

FLT201, a novel liver-directed AAV gene therapy candidate ... Preclinical data indicate that FLT201 could offer a durable treatment for Gaucher disease type 1, addressing unmet needs related to substrate accumulation in ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10970163/

Advancements in Viral Gene Therapy for Gaucher Disease ERT is considered very safe, with reported side effects rarely warranting discontinuation of treatment [10]. However, despite its efficacy, ERT ...