Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

Shire

Status: Completed

Conditions: Gaucher Disease

Study type: Observational

Funder types: Industry

Identifiers

NCT04429984

SHP669-406

Details and patient eligibility

About

The main aim of this study is to measure the safety and to find out the effects of VPRIV in participants with Gaucher disease using both retrospective and prospective data when used in the post-marketing setting and to collect genetic mutation data from participants with Gaucher disease.

This study is about collecting data available in the participant's medical record as well as data from each participant's ongoing treatment. No study medicines will be provided to participants in this study.

When the participants start the study, they will visit the study clinic close to approximately 12 months.

Enrollment: 21 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participants with type 1 Gaucher disease prescribed VPRIV according to the investigator's judgment and current Indian Prescribing information (PI) are eligible for this study.
• Participants or legally authorized representative must provide written informed consent to participate.

Exclusion criteria

• Participants will be excluded from this study if the participant met any of the contraindications included in the current Indian PI for VPRIV.

Trial design

21 participants in 1 patient group

VPRIV: All Participants

Description:

Participants with type 1 Gaucher disease who as part of standard or routine clinical practice, have already received VPRIV retrospectively and will further receive VPRIV therapy according to the investigator's judgment for approximately 12 months will be observed prospectively in this PMS study.