An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

Status: Completed
Location: See all (15) locations...
Intervention Type: Dietary Supplement, Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY

The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: No
View:

• The participant has a documented diagnosis of type 1 Gaucher disease, as documented by deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts. Diagnosis by only dry blood spot test is insufficient. Diagnosis may be based on results obtained prior to screening if documented in the participant's medical history.

• Participants must have a LS BMD Z-score less than (<) -1 or BMD T-score of < -1 as measured by DXA during the screening phase.

• Participant is treatment-naive, that is (ie,) has not received ERT or SRT in the 12 months prior to enrollment.

• The participant is greater than or equal to (>=) 18 and less than or equal to (<=) 70 years of age.

• Female participants of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study.

• The participant, or participant's legally authorized representative(s), if applicable, understands the nature, scope, and possible consequences of the study and has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

• The participant must be sufficiently cooperative to participate in this clinical study as judged by the investigator.

Locations
United States
California
Cedars Sinai Medical Center
Beverly Hills
Kaiser Permanente
Los Angeles
Georgia
Emory Genetics
Atlanta
Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago
North Carolina
Duke University Medical Center
Durham
New York
NYU School of Medicine
New York
Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia
Virginia
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax
Other Locations
Israel
Rambam Health Care Campus
Haifa
Shaare Zedek Medical Center
Jerusalem
Rabin Medical Center
Petah Tikva
Spain
Hospital Universitario Ramon y Cajal
Madrid
Hospital Quironsalud Zaragoza
Zaragoza
United Kingdom
Addenbrooke's Hospital
Cambridge
Royal Free Hospital
London
Time Frame
Start Date: June 29, 2016
Completion Date: November 30, 2020
Participants
Target number of participants: 21
Treatments
Experimental: Velaglucerase alfa 60 U/kg
Participants will receive 60-minute intravenous infusion of 60 units per kilogram (U/kg) velaglucerase alfa every other week (EOW) and an oral daily dose of 800 IU vitamin D for 24 months (101 weeks).
Authors
Divya Vats, Can Ficicioglu, Heather Lau, Rebecca Mardach, Priya Kishnani, Joel Charrow, Ozlem Goker-Alpan, Noa Ruhrman Shahar, Patrick B Deegan, Derralynn A Hughes, Ari Zimmerman, Jesus Villarrubia Espinosa, Jaime Vengoechea Barrios, Divya Divya, Pilar Pilar, Hagit Baris Feldman, Ari Zimran, Pilar Giraldo
Sponsors
Leads: Shire

This content was sourced from clinicaltrials.gov

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