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Trial record 4 of 102 for:    Gaucher Disease

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

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ClinicalTrials.gov Identifier: NCT02528617
Recruitment Status : Withdrawn (No subjects accrued and no potential subjects were identified..)
First Posted : August 19, 2015
Last Update Posted : March 2, 2018
Sponsor:
Collaborator:
Texas Scottish Rite Hospital for Children
Information provided by (Responsible Party):
Baylor Research Institute

Brief Summary:
The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

Condition or disease Intervention/treatment Phase
Gaucher Disease Type 1 Gaucher Disease Type 3 Drug: Velaglucerase alfa Phase 4

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Estimated Study Start Date : July 2015
Actual Primary Completion Date : October 2017
Actual Study Completion Date : October 2017


Arm Intervention/treatment
Gaucher Type 1 or 3
Velaglucerase alfa IV 60 units/kg every other week for duration of the study.
Drug: Velaglucerase alfa
Enzyme replacement therapy
Other Name: VPRIV




Primary Outcome Measures :
  1. Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa. [ Time Frame: Baseline pre-intervention and yearly thereafter for 3 years ]

Secondary Outcome Measures :
  1. Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa. [ Time Frame: Baseline pre intervention and yearly thereafter for 3 years ]
  2. Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa. [ Time Frame: Baseline pre intervention and yearly thereafter for 3 years ]
  3. Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa. [ Time Frame: Baseline pre-intervention and yearly thereafter for 3 years ]
  4. Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa. [ Time Frame: Baseline pre-intervention and yearly thereafter for 3 years. ]


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Ages Eligible for Study:   4 Years to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Enzyme Replacement Therapy naive,
  • confirmed diagnosis of Gaucher disease type 1 or 3,
  • able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
  • able to tolerate all study procedures,
  • skeleton not fully formed as confirmed by DXA and MRI),
  • and willing to receive velaglucerase alfa infusions every other week for the duration of the study.

Exclusion Criteria:

  • Clinically unstable,
  • taking or have taken bisphosphonates,
  • Gaucher type 2,
  • pregnant female,
  • or deemed inappropriate for participation by the principal investigator.

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): NCT02528617


Locations
United States, Texas
Baylor Research Institute
Dallas, Texas, United States, 75226
Sponsors and Collaborators
Baylor Research Institute
Texas Scottish Rite Hospital for Children
Investigators
Principal Investigator: Raphael Schiffmann, M.D.,M.H.Sc. Baylor Research Institute/Institute of Metabolic Disease

Responsible Party: Baylor Research Institute
ClinicalTrials.gov Identifier: NCT02528617     History of Changes
Other Study ID Numbers: 014-285
First Posted: August 19, 2015    Key Record Dates
Last Update Posted: March 2, 2018
Last Verified: February 2018

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