CompletedPhase 3

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

China20 participantsStarted 2023-01-03

Plain-language summary

The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT). Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.

Who can participate

Age range2 Years

SexALL

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Inclusion criteria

✓. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
✓. Decreased GCB activity level that is \>30% of normal, but with confirmation of genetic mutation test
✓. Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
✓. A platelet count of \<90 × 10\^9/L below the lower limit of normal for their age and gender

What they're measuring

Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE)

Timeframe: Up to 56.2 weeks

Trial details

NCT IDNCT05529992

SponsorTakeda

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08-05

Results submitted2025-02-04

View on ClinicalTrials.gov More Gaucher Disease trials