Gene Therapy (FLT201) for Gaucher Disease

(GALILEO-1 Trial)

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

18 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

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

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)

Treatment Details

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.

Participant Groups

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+