Clinical trial NCT04429984: Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
Official title: A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Gaucher Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2020-06-12
Enrollment: 21
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with confirmed type 1 Gaucher disease irrespective of any age or gender who have been prescribed VPRIV therapy according to the investigator's judgment will be included in this study.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Baseline up to approximately 12 months
measure: Number of Participants With Adverse Drug Reactions (ADRs)
timeFrame: Baseline up to approximately 12 months
Secondary outcomes:
measure: Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Liver Size Using Both Retrospective and Prospective Data
timeFrame: Baseline up to approximately 12 months
measure: Treatment History Based on Previous VPRIV Treatment
timeFrame: At Baseline
measure: Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Platelet Count Based on Previous VPRIV Treatment
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Spleen Size Based on Previous VPRIV Treatment
timeFrame: Baseline up to approximately 12 months
measure: Change From Baseline in Liver Size Based on Previous VPRIV Treatment
timeFrame: Baseline up to approximately 12 months
measure: Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment
timeFrame: Baseline up to approximately 12 months
Other outcomes:
Locations:
city: Kochi
country: India
facility: Amrita Institute of Medical Sciences & Research Centre (AIMS)
state: Kerala
city: New Delhi
country: India
facility: All India Institute of Medical Sciences
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: