A Gaucher Disease Gene Therapy Trial With FLT201

Conditions

• Gaucher Disease Type 1

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• FLT201 — GENETIC
  FLT201 is a replication-incompetent single-stranded (ss) recombinant adeno-associated virus (AAV) vector. The vector is composed of a ss DNA genome packaged in an AAV-derived protein capsid.

Study Details

This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.

Key Dates

Start date

Apr 7, 2026

Status verified

Apr 2026

Primary completion

Jul 1, 2028

Completion

Oct 1, 2032

Study Design

Enrollment

45 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

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

Primary Outcome Measure

To evaluate the efficacy of FLT201 [ Time Frame: 1 Year ]

Central Contacts

• Spur Clinical Trials Contact
  +44 (0)1438 906870
  [email protected]

Locations (3)

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