Long-term follow-up for Gaucher disease patients after FLT201 treatment
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects With Gaucher Disease (GALILEO-2)
This study looks at how people with Type 1 Gaucher disease are doing five years after receiving the FLT201 treatment to see how it has affected their health over time.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 75 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Spur Therapeutics Industry-sponsored |
| Locations | 4 sites (Fairfax, Virginia and 3 other locations) |
| Trial ID | NCT06545136 on ClinicalTrials.gov |
What this trial studies
This multicenter trial follows participants with Type 1 Gaucher disease who previously received FLT201 treatment in an earlier clinical trial. The study aims to monitor the long-term effects and outcomes of the treatment over a period of five years. Participants will be assessed for their health status and any changes in their condition post-treatment. The study is designed to gather valuable data on the efficacy and safety of FLT201 in the long term.
Who should consider this trial
Good fit: Ideal candidates are individuals who have previously received FLT201 treatment for Type 1 Gaucher disease.
Not a fit: Patients who have not received FLT201 treatment or those who do not meet the inclusion criteria will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide insights into the long-term effectiveness and safety of FLT201 for patients with Type 1 Gaucher disease.
How similar studies have performed: Other studies on long-term follow-up of treatments for Gaucher disease have shown promising results, indicating that this approach is supported by previous findings.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Fairfax, Virginia and 3 other locations
- Lysosomal Rare Disorders Research and Treatment Center — Fairfax, Virginia, United States (Not_yet_recruiting)
- Hospital de Clinicas de Porto Alegre (HCPA) — Porto Alegre, Brazil (Not_yet_recruiting)
- Hospital Quironsalud Zaragoza — Zaragoza, Spain (Recruiting)
- Salford Royal Hospital — Salford, United Kingdom (Not_yet_recruiting)
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.