Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease



Status:Recruiting
Conditions:Metabolic
Therapuetic Areas:Pharmacology / Toxicology
Healthy:No
Age Range:18 - 70
Updated:4/5/2019
Start Date:June 29, 2016
End Date:January 30, 2021
Contact:Shire Contact
Email:ClinicalTransparency@shire.com
Phone:+1 866 842 5335

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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

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 DXA after 24 months of treatment.


Inclusion Criteria:

- 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.

Exclusion criteria

- Neurological symptoms indicating that the participant may have type 3 Gaucher disease.

- A significant comorbidity, which, as determined by the investigator, might affect
study data or confound the study results (eg, malignancies, primary biliary cirrhosis,
autoimmune liver disease, etc).

- Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with
erythropoietin (or erythropoietin-like substances) during the past year.

- Structural, joint-associated bone damage of such extent and severity that the
investigator deems it could impact participation in the study and assessment of
relevant study endpoints (example, pain).

- The participant is pregnant or lactating.

- The participant has had a splenectomy.

- The participant is enrolled in another clinical study that involves clinical
investigations or use of any investigational product (drug or device) within 30 days
prior to study enrollment or at any time during the study.

- Severe vitamin D deficiency to the level that would be expected to result in
osteomalacia (vitamin D < 10 nanograms per milliliter [ng/mL] [25 nanomoles per liter
{nmol/L}]). If there is mild vitamin D insufficiency at screening (vitamin D greater
than [>] 10 and < 30 ng/mL) treat with 4000 IU vitamin D per day for 1 month and
rescreen.

- The participant has previously interrupted ERT for safety reasons.

- The participant has had hypersensitivity to the active substance or to any of the
excipients.
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Los Angeles, California 90027
Principal Investigator: Divya Vats, MD
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 215-590-1000
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Principal Investigator: Jaime Vengoechea Barrios, MD
Phone: 404-778-3543
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Beverly Hills, California 90211
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225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Joel Charrow, MD
Phone: 312-227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Priya Kishnani, MD
Phone: 919-681-9854
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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3959 Pender Drive
Fairfax, Virginia 22030
Principal Investigator: Ozlem Goker-Alpan
Phone: 571-308-1900
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8 HaAliya HaShniya Street
Haifa, 31096
Principal Investigator: Hagit Baris Feldman
Phone: +972-4-7772456
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New York, New York 10016
Principal Investigator: Heather Lau, MD
Phone: 212-263-8344
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