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Trial record 27 of 102 for:    Gaucher Disease

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

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Gaucher Disease Drug: Velaglucerase alfa Dietary Supplement: 800 International Unit (IU) Vitamin D Phase 4

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 11, 2016
Estimated Primary Completion Date : February 26, 2021
Estimated Study Completion Date : March 31, 2021


Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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).

  9. 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.



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02574286


Contacts
Contact: Shire Contact 1-866-842-5335 clinicaltransparency@shire.com

Locations
United States, California
Kaiser Permanente Recruiting
Los Angeles, California, United States, 90027
Contact: Divya Vats       dvats@chla.usc.edu   
Principal Investigator: Divya Vats, MD         
United States, Georgia
Emory Genetics Recruiting
Decatur, Georgia, United States, 30033
Contact: Jaime Vengoechea Barrios    404-778-3543      
Principal Investigator: Jaime Vengoechea Barrios, MD         
United States, Illinois
Ann and Robert H Lurie Childrens Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Joel Charrow, MD       jcharrow@northwestern.edu   
Principal Investigator: Joel Charrow, MD         
United States, New York
NYU School of Medicine - Hospital Recruiting
New York, New York, United States, 10016
Contact: Heather Lau, MD    212-263-0139      
Principal Investigator: Heather Lau, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Priya Kishnani    919-681-9854    kishn001@mc.duke.edu   
Principal Investigator: Priya Kishnani, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Can Ficicioglu    215-590-3376    ficicioglu@email.chop.edu   
Principal Investigator: Can Ficicioglu         
Israel
Gaucher Clinical Department of Medicine Recruiting
Jerusalem, Israel, 9103102
Contact: Ari Zimmerman    972-2-6555143      
Principal Investigator: Ari Zimmerman, MD         
Spain
Hospital Universitario Ramon y Cajal Recruiting
Madrid, Spain, 28034
Contact: Jesus Villarrubia Espinosa, MD, PhD    + 34 913 368 686    jesus.vallarrubia@salud.madrid.org   
Principal Investigator: Jesus Villarrubia Espinosa, MD, PhD         
Hospital Quironsalud Zaragoza Recruiting
Zaragoza, Spain, 50006
Contact: Pilar Giraldo    +34976765546      
Principal Investigator: Pilar Pilar, MD         
United Kingdom
Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB20QQ
Contact: Patrick B Deegan, MD    +4401223274634    patrick.deegan@addenbrookes.nhs.uk   
Principal Investigator: Patrick B Deegan, MD         
Royal Free Hospital Recruiting
London, United Kingdom, NW3 2QG
Contact: Derralynn A Hughes, MD    020 77943 4154    rmgvdah@ucl.ac.uk   
Principal Investigator: Derralynn A Hughes, MA, DPhil, BMBCh, MRCP         
Sponsors and Collaborators
Shire
Investigators
Study Director: Shire Physician Shire

Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02574286     History of Changes
Other Study ID Numbers: SHP-GCB-402
2015-001578-17 ( EudraCT Number )
First Posted: October 12, 2015    Key Record Dates
Last Update Posted: May 22, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Vitamins
Vitamin D
Micronutrients
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents