Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Freeline Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Gaucher Disease, Type 1

Treatments: Genetic: FLT201

Study type: Interventional

Funder types: Industry

Identifiers

NCT06545136

FLT201-02

Details and patient eligibility

About

This is a multicenter, long-term, follow-up trial of participants with Gaucher disease type 1 who received FLT201 treatment in a preceding clinical trial. Participants will be followed for 5 years post-treatment.

Enrollment: 75 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 1. Participants who have previously received FLT201 (including those who may have required recommencement or initiation of ERT/substrate reduction therapy [SRT]).
• 2. Participants able to give full informed consent and able to comply with all requirements of the trial.

Exclusion criteria

• n/a

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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75 participants in 1 patient group

Dosed with FLT201

Experimental group

Treatment:

Genetic: FLT201