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

Baylor Scott and White Health (BSWH)

Status and phase: Withdrawn; Phase 4

Conditions: Gaucher Disease Type 3; Gaucher Disease Type 1

Treatments: Drug: Velaglucerase alfa

Study type: Interventional

Funder types: Other

Identifiers

NCT02528617

014-285

Details and patient eligibility

About

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.

Sex: All

Ages: 4 to 14 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Gaucher Type 1 or 3

Other group

Description:

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

Treatment:

Drug: Velaglucerase alfa