Status:
COMPLETED
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
Lead Sponsor:
Takeda
Conditions:
Gaucher Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE4
Brief Summary
The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means the par...
Eligibility Criteria
Inclusion
- Participant eligibility is determined according to the following criteria prior to entry into the study:
- In the opinion of the investigator, the participant is capable of understanding and complying with protocol requirements.
- Participant either signs and dates a written, informed consent form or completes an e-consent process prior to the initiation of any study procedures.
- Participant has been diagnosed with GD type I; diagnosis was confirmed biochemically and/or genetically.
- Participant has been treated with SRT for at least 3 months prior to switch to VPRIV.
- Participant has been treated with VPRIV at least 3 months prior to enrollment (Baseline \[Day 0\]).
- Participant is aged 18 or older.
- Arm A: Participant is able to use mobile application based on clinician's judgment, (e.g., owns an iPhone version 5 or later or smartphones with Android operating systems, have an active data plan or regular Wi-Fi access).
- Arm A: The participant's primary language is English.
Exclusion
- Any participant who meets any of the following criteria will not qualify for entry into the study:
- Participant is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in conduct of this study (e.g., spouse, parent, child, sibling) or may consent under duress.
- Participant is judged by the investigator as being ineligible for any other reason.
- Participant has L444P/L444P GBA1 genotype (c.1448T greater than \[\>\] C).
- Participant has Parkinson's disease, a history of central nervous system \[CNS\] manifestations, or any other neurological disorder (e.g. Lewy Body Disease, Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Multiple sclerosis).
Key Trial Info
Start Date :
April 22 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 16 2023
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT04718779
Start Date
April 22 2021
End Date
February 16 2023
Last Update
December 21 2023
Active Locations (1)
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1
The Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, United States, 22030