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Phase 4 Study to Evaluate the Effect of Velaglucerase Alfa (VPRIV®) on Patients With Type 1 Gaucher Disease Through the IV Administration of VPRIV® Over 2 Years

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ClinicalTrials.gov Identifier: NCT02574286
Recruitment Status : Recruiting
First Posted : October 12, 2015
Last Update Posted : May 22, 2018
Sponsor:
Information provided by (Responsible Party):
Shire

October 9, 2015
October 12, 2015
May 22, 2018
July 11, 2016
February 26, 2021   (Final data collection date for primary outcome measure)
Change From Baseline in Lumbar Spin (LS) Bone Mineral Density (BMD) Z-Score to Week 103/End of Study [ Time Frame: Baseline, Week 103 (End of Study) ]
Bone mineral density of the lumbar spine will be measured by dual energy x-ray absorptiometry (DXA), and the results will be converted to Z-scores appropriate for age, sex, and race. Lumbar spine T-scores/WHO classifications of normal bone density, osteopenia, osteoporosis.
Change from baseline to 12 months (Week 51) in lumbar spine (LS) and Bone Mineral Density (BMD) Z-score as measured by DXA. [ Time Frame: Screening and Weeks 25, 51, 77, and 103 (end of study) ]
Complete list of historical versions of study NCT02574286 on ClinicalTrials.gov Archive Site
  • Change From Baseline in Bone Marrow Burden (BMB) Score at Week 51 and Week 103 (end of Study) [ Time Frame: Baseline, Week 51, and 103 (end of study) ]
    Bone marrow burden scores will be calculated from magnetic resonance imaging (MRI) of the LS and femurs, and will be converted to BMB scores between 0 and 8.
  • Change From Baseline in Lumbar Spin (LS) Bone Mineral Density (BMD) Z-score at Week 51 [ Time Frame: Baseline, Week 51, and 103 (end of study) ]
    Bone mineral density of the lumbar spine will be measured by dual energy x-ray absorptiometry (DXA), and the results will be converted to Z-scores appropriate for age, sex, and race. Lumbar spine T-scores/WHO classifications of normal bone density, osteopenia, osteoporosis.
  • Change From Baseline Over Time in Hemoglobin Concentration [ Time Frame: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and 103 (end of study) ]
    Hemoglobin Concentration will be measured from the collected blood samples at the baseline visit and Weeks 13, 25, 37, 51, 65, 77, 89, and 103. The measurement of hemoglobin concentration will be included as a component of hematology laboratory testing.
  • Change From Baseline Over Time in Platelet Count [ Time Frame: Baseline, Week 13, 25, 37, 51, 65, 77, 89, and 103 (end of study) ]
    Platelet count will be measured from the collected blood samples at baseline and week 13, 25, 37, 51, 65, 77, 89, and 103. The measurement of platelet count will be included as a component of hematology laboratory testing.
  • Change From Baseline in Normalized Liver Volume at Week 51 and Week 103 (end of Study) [ Time Frame: Baseline, Week 51 and 103 (end of study). ]
    Normalized liver volume will be measured by abdominal MRI.
  • Change From Baseline in Normalized Spleen Volume at Week 51 and Week 103 (end of Study) [ Time Frame: Baseline, Week 51 and 103 (end of study). ]
    Normalized spleen volume will be measured by MRI.
  • Change From Baseline in Bone Pain at Week 51 and Week 103 (end of Study) [ Time Frame: Baseline, Week 51 and 103 (end of study) ]
    Bone pain will be measured by the Brief Pain Inventory (BPI). The BPI is developed to assess the severity of pain and the impact of pain on daily functions. It will assess the severity of pain, impact of pain on daily function, location of pain, pain medications, and amount of pain relief in the past 24 hours or the past week.
  • Change From Baseline in Overall Fatigue at Week 51 and Week 103 (end of study) [ Time Frame: Baseline, Week 51 and 103 (end of study) ]
    Overall fatigue will be measured by the Brief Fatigue Inventory (BFI).
  • Shifts In World Health Organization (WHO) Bone Mineral Density (BMD) Classifications (Normal Bone Density, Osteopenia, Osteoporosis) Based on Lumbar Spine (LS) T-Scores. [ Time Frame: Baseline, Week 51, and 103 (end of study) ]
    LS T-scores/WHO classification includes normal bone density, osteopenia, and osteoporosis.
  • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in bone marrow burden (BMB) score (MRI of LS and femur). [ Time Frame: Baseline and Weeks 25, 51, 77, and 103 (end of study). ]
  • Change from baseline at 24 months (Week 103 [end of study]) in LS BMD Z-score. [ Time Frame: Baseline and Weeks 25, 51, 77, and 103 (end of study). ]
  • Change from baseline over time in hemoglobin concentration. [ Time Frame: Baseline and Weeks 7, 19, 31, 45, 57, 71, 83, and 95. ]
  • Change from baseline over time in platelet count. [ Time Frame: Baseline and Weeks 7, 19, 31, 45, 57, 71, 83, and 95. ]
  • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in normalized liver volume as measured by abdominal MRI. [ Time Frame: Baseline visit and Weeks 25, 51, 77, and 103 (end of study). ]
  • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in normalized spleen volume as measured by MRI. [ Time Frame: Baseline visit and Weeks 25, 51, 77, and 103 (end of study). ]
  • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in bone pain as measured by the brief pain inventory (BPI). [ Time Frame: Baseline visit and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study). ]
  • Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in overall fatigue as measured by the BFI. [ Time Frame: Baseline visit and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study). ]
  • Shifts in World Health Organization (WHO) BMD classifications (normal bone density, osteopenia, osteoporosis) based on LS T-scores. [ Time Frame: Baseline (screening visit) and Weeks 25, 51, 77, and 103 (end of study). ]
  • Number of participants with Adverse Events (AEs) [ Time Frame: Screening through 30 day safety follow-up ]
Not Provided
Not Provided
 
Phase 4 Study to Evaluate the Effect of Velaglucerase Alfa (VPRIV®) on Patients With Type 1 Gaucher Disease Through the IV Administration of VPRIV® Over 2 Years
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
To evaluate the effect of VPRIV® therapy on patients with type 1 Gaucher disease by measuring the change in the lumbar spine (LS) and bone mineral density (BMD) after 24 months of treatment.
Not Provided
Interventional
Phase 4
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Gaucher Disease
  • Drug: Velaglucerase alfa
    Velaglucerase alfa 60 U/kg every other week (EOW) as a 60-minute intravenous infusion
  • Dietary Supplement: 800 International Unit (IU) Vitamin D
    800 IU vitamin D orally daily starting at the week 1 visit
Experimental: Velaglucerase alfa 60 Unit per kilogram (U/kg)
Participant will be receiving velaglucerase alfa 60 U/kg every other week (EOW) as 60 minute intravenous infusion.
Interventions:
  • Drug: Velaglucerase alfa
  • Dietary Supplement: 800 International Unit (IU) Vitamin D
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
40
Same as current
March 31, 2021
February 26, 2021   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (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 patient's medical history.
  2. Patients must have a lumbar spine (LS) bone mineral density (BMD) Z-score < -1 or BMD T-score of < -1 as measured by dual energy x-ray absorptiometry (DXA) during the screening phase.
  3. Patient is treatment-naïve: should not have received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the 12 months prior to enrollment.
  4. The patient is greater than or equal to (≥) 18 and less than or equal to (≤)70 years of age.
  5. Female patients of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study.
  6. The patient, or patient'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).
  7. The patient must be sufficiently cooperative to participate in this clinical study as judged by the investigator.

Exclusion criteria

  1. Neurological symptoms indicating that the patient may have type 3 Gaucher disease.
  2. A significant comorbidity, which, as determined by the investigator, might compromise study assessment, 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.
  8. Severe vitamin D deficiency to the level that would be expected to result in osteomalacia [vitamin D < 10 nanogram per mililitre (ng/mL) (25 nanomoles per litre (nmol/L)]. If there is mild vitamin D insufficiency at screening [vitamin D > 10 and < 30 ng/mL] treat with 4000 International Unit (IU) vitamin D per day for 1 month and rescreen.
  9. The patient has previously interrupted ERT for safety reasons.
  10. The patient has had hypersensitivity to the active substance or to any of the excipients.
Sexes Eligible for Study: All
18 Years to 70 Years   (Adult, Older Adult)
No
Contact: Shire Contact 1-866-842-5335 clinicaltransparency@shire.com
Israel,   Spain,   United Kingdom,   United States
 
 
NCT02574286
SHP-GCB-402
2015-001578-17 ( EudraCT Number )
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Shire
Shire
Not Provided
Study Director: Shire Physician Shire
Shire
May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP