Gaucher Disease, Type 1 Clinical Trial
Official title:
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study
Verified date | October 2022 |
Source | Shaare Zedek Medical Center |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Background: In order to allow our satisfied patients, who have successfully completed 24 months of rapid intravenous infusion of Velaglucerase alfa (VPRIV), to continue with the 10 minutes IV therapy, the clinical trial framework must be extended; and this extension is important for the assessment of long term benefit (up to 5 years) of this regimen of administration of Velaglucerase alfa.. Suggested trial: An additional 36 months home therapy follow up of safety and efficacy of rapid intravenous infusion of Velaglucerase alfa (VPRIV) in adult patients with type 1 Gaucher disease. Patients must have completed the prior 4 parts / 24 months of the protocol before enrolling into this extension phase ("Part 5") and have provided a new consent before entering PART 5 of the study. Patients must not have experienced clinically significant AEs, including allergic reactions, in any of the prior study parts of this protocol to be eligible to participate, and have maintained stability in the key disease features. All infusions of 10' will be given in the context of home therapy. "Clinically significant" AEs will be determined by the PI using standard description of AEs as previously described at phase 3, and if necessary will support withdrawal of the patient from the study.
Status | Completed |
Enrollment | 15 |
Est. completion date | January 1, 2022 |
Est. primary completion date | January 20, 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years to 75 Years |
Eligibility | Inclusion Criteria: - Aged 18-75 years, non-splenectomized Enzymatic diagnosis & molecular analysis indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3 months) prior to Baseline at a constant dose and frequency and without clinically significant AEs including allergic reactions Exclusion Criteria: - Experience of a clinically significant AE to VPRIV at any time in the past Existence of a clinically significant co-morbidity |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Shaare Zedek Medical Center | Shire |
Zimran A, Revel-Vilk S, Becker-Cohen M, Chicco G, Arbel N, Rolfs A, Szer J. Rapid intravenous infusion of velaglucerase-alfa in adults with type 1 Gaucher disease. Am J Hematol. 2018 Sep;93(9):E246-E248. doi: 10.1002/ajh.25205. Epub 2018 Aug 9. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence of change from baseline in blood pressure for rapid infusion-1 | measured by blood pressure pre and post infusion | 9 months | |
Primary | Incidence of change from baseline in heart rate for rapid infusion-1 | measured by heart rate pre and post infusion | 9 months | |
Primary | Incidence of change from baseline in temperature for rapid infusion-1 | measured by temperature pre and post infusion | 9 months | |
Secondary | Non deterioration in Gaucher manifestations- stability in platelet counts | Efficacy secondary endpoints are non-deterioration (defined as stability) in platelet count | 9 months | |
Secondary | Non deterioration in Gaucher manifestations- stability in hemaglobin count | Efficacy secondary endpoints are non-deterioration (defined as stability) in hemaglobin count | 9 months | |
Secondary | Non deterioration in Gaucher manifestations- measured by liver volume | Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of liver volume (as previously defined in the TKT034 clinical trial) | 9 months | |
Secondary | Non deterioration in Gaucher manifestations- measured by spleen volumes | Efficacy secondary endpoints are non-deterioration (defined as stability) in organ volumes of spleen volume (as previously defined in the TKT034 clinical trial) | 9 months | |
Secondary | Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1 | Efficacy secondary endpoints are non-deterioration (defined as stability) by lack of sustained increases in the biomarkers. | 9 months |
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