A Gene Therapy Study in Patients With Gaucher Disease Type 1 (NCT05324943) | Clinical Trial Compass
CompletedPhase 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
United States, Brazil, Germany6 participantsStarted 2022-04-15
Plain-language summary
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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Adult ≥ 18 years of age.
✓. Diagnosis of Gaucher disease Type 1 with deficient GCase enzyme activity ≤30% of normal in leukocytes at diagnosis.
✓. All female patients of childbearing potential must not be lactating and must have a negative serum pregnancy test at screening and confirmed negative by urine testing prior to dosing on Day 1. Female patients of childbearing potential and male patients must be willing to follow protocol guidelines for barrier protection/contraception.
✓. Able to give full informed consent for the trial.
✓. Treatment status at screening (screening period is 16 weeks):
Exclusion criteria
✕. Diagnosed or suspected Type 2 or Type 3 Gaucher disease (including any patient with eye movement abnormality on clinical examination).
✕. Positive for neutralising antibodies to AAVS3 at screening.
✕. Evidence of significant and persistent liver dysfunction at Screening defined as \>1.5 x upper limit of normal (ULN) in alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin.
✕. Evidence of any of the following at screening:
✕. Hb \<8 g/dL.
✕. Platelets \<45,000/mm3.
✕. Pulmonary hypertension.
What they're measuring
1
Incidence of treatment-emergent adverse events
Timeframe: Day 1 (dosing) through the final follow-up visit at Week 38