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

NCT04429984 | Completed

• 1 other identifier: SHP669-406
• Study Type: observational
• Target: 21
• Locations: 1 country
• Sites: 2

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.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Adverse Events (AEs)

  An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP).

  Baseline up to approximately 12 months

• Number of Participants With Adverse Drug Reactions (ADRs)

  An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP.

  Baseline up to approximately 12 months

Secondary Outcomes (10)

• Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data

  Baseline up to approximately 12 months

• Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data

  Baseline up to approximately 12 months

• Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data

  Baseline up to approximately 12 months

• Change From Baseline in Liver Size Using Both Retrospective and Prospective Data

  Baseline up to approximately 12 months

• Treatment History Based on Previous VPRIV Treatment

  At Baseline

Study Arms (1)

VPRIV: All Participants

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Shire: lead

Study Sites (2)

Amrita Institute of Medical Sciences & Research Centre (AIMS)

Kochi, Kerala, 682041, India

Location

All India Institute of Medical Sciences

New Delhi, 110029, India

Location

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2020
• First Posted: June 12, 2020
• Study Start: July 28, 2021
• Primary Completion: April 22, 2023
• Study Completion: April 22, 2023
• Last Updated: June 7, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

IN (2)