An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease
- Conditions
- Gaucher DiseaseMedDRA version: 18.1Level: PTClassification code 10075697Term: Gaucher's disease type ISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2015-001578-17-ES
- Lead Sponsor
- Shire Human Genetic Therapies, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 21
Each patient must meet the following criteria to be enrolled in this study.
1. The patient 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 dry blood spot test is not sufficient. Diagnosis may be based on results obtained
prior to screening if documented in the patient?s medical history.
2. Patients must have a LS BMD Z-score <-1 as measured by DXA during the screening phase.
3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to
enrollment.
4. The patient is ?16 and ?65 years of age. For patients ?16 and <18 years of age a Tanner
stage of ?4 must be established.
5. Female patients of child bearing potential must agree to use a medically acceptable method
of contraception at all times during the study.
6. The patient, and the patient?s parent(s) or legal guardian(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).
7. The patient must be sufficiently cooperative to participate in this clinical study as judged by
the investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Patients who meet any of the following criteria will be excluded from the study.
1. Neurological symptoms indicating that the patient has type 3 Gaucher disease.
2. 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).
3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin
(or erythropoietin-like substances) during the past year.
4. 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
(eg, pain).
5. The patient is pregnant or lactating.
6. The patient has had a splenectomy.
7. The patient 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the effect of VPRIV therapy (60 U/kg every other week [EOW]) in treatment-naïve<br>patients with type 1 Gaucher disease on change from baseline in lumbar spine (LS) BMD<br>Z-score as measured by DXA after 12 months of treatment.;Secondary Objective: To evaluate the effect of VPRIV therapy (60 U/kg EOW) on BMD in treatment-naïve patients<br>with type 1 Gaucher disease as measured by the change from baseline in LS BMD Z-score at<br>24 months, and BMB as measured by MRI of the LS and femur after 12 and 24 months of<br>treatment.;Primary end point(s): Change from baseline to 12 months (Week 51) in LS BMD Z-score as measured by DXA.<br>Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of<br>study).;Timepoint(s) of evaluation of this end point: Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of<br>study).
- Secondary Outcome Measures
Name Time Method Secondary end point(s): The key secondary endpoints of this study are:<br>? Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in<br>BMB score (MRI of lumbar spine and femur). Assessments will be performed at the baseline<br>visit and Weeks 25, 51, 77, and 103 (end of study).<br>? Change from baseline in LS BMD Z-score at 24 months (Week 103 [end of study]).<br>Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of<br>study).;Timepoint(s) of evaluation of this end point: . Assessments will be performed at the baseline visit and Weeks 25, 51, 77, and 103 (end of study).<br><br>. Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of<br>study).