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

NCT02528617 | Withdrawn Phase 4

• 1 other identifier: 014-285
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Gaucher Disease Type 1; Gaucher Disease Type 3

Outcome Measures

Primary Outcomes (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.

  Baseline pre-intervention and yearly thereafter for 3 years

Secondary Outcomes (4)

• Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.

  Baseline pre intervention and yearly thereafter for 3 years

• Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.

  Baseline pre intervention and yearly thereafter for 3 years

• Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.

  Baseline pre-intervention and yearly thereafter for 3 years

• Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.

  Baseline pre-intervention and yearly thereafter for 3 years.

Study Arms (1)

Gaucher Type 1 or 3

OTHER

Velaglucerase alfa IV 60 units/kg every other week for duration of the study.

Drug: Velaglucerase alfa

Interventions

Velaglucerase alfa DRUG

Enzyme replacement therapy

Also known as: VPRIV

Gaucher Type 1 or 3

Eligibility Criteria

• Age: 4 Years - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Baylor Research Institute: lead
• Texas Scottish Rite Hospital for Children: collaborator

Study Sites (1)

Baylor Research Institute

Dallas, Texas, 75226, United States

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Officials

• Raphael Schiffmann, M.D.,M.H.Sc.

  Baylor Research Institute/Institute of Metabolic Disease

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2015
• First Posted: August 19, 2015
• Study Start: July 1, 2015
• Primary Completion: October 1, 2017
• Study Completion: October 1, 2017
• Last Updated: March 2, 2018

Record last verified: 2018-02

Locations

US (1)