Phase 3 N=5 Treatment

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Gaucher Disease

Bottom Line

View on ClinicalTrials.gov: NCT01842841 ↗

Enrolled (actual)

Serious AEs

40.0%

Results posted

Dec 2015

Primary outcome: Primary: Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) — 2; 0; 2 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: velaglucerase alfa (Drug)
Age: Pediatric, Adult, Older Adult · 2+ yrs
Sex: All
Sponsor: Shire
Primary completion: Oct 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs)	2; 0; 2	—
PRIMARY Number of Participants Using Concomitant Medication	5	—
PRIMARY Number of Participants With Abnormal and Clinically Significant Laboratory Test Results	1	—
PRIMARY Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies	—	—
SECONDARY Change From Baseline in Hemoglobin Concentration at Week 101	0.22	—
SECONDARY Change From Baseline in Platelet Count at Week 101	9.8	—
SECONDARY Change From Baseline in Liver Volume Normalized to Body Weight at Week 103	0.01	—
SECONDARY Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103	0.04	—
SECONDARY Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score	—	—
SECONDARY Change From Baseline in Bone Mineral Density (BMD) at Week 103: T-Score	—	—
SECONDARY Change From Baseline in Bone Marrow Burden (BMB) Score at Week 103	-1.2; 0.0; -1.2	—
SECONDARY Change From Baseline in Growth Velocity at Week 101 : Height Z-Score	—	—
SECONDARY Change From Baseline in Skeletal Age at Week 103: Z-Score	—	—
SECONDARY Change From Baseline in Plasma Chitotriosidase Levels at Week 101	-181.0	—
SECONDARY Number of Participants With Change From Baseline in Neurological Status at Week 103	—	—
SECONDARY Change From Baseline in Chemokine [C-C Motif] Ligand 18 (CCL18) Levels at Week 101	-11.4	—

Summary

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with Type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with Type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

Eligibility Criteria

Inclusion Criteria

The patient has completed treatment with EOW velaglucerase alfa through Week 51 of study HGT-GCB-087.
Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study.
The patient, the patient's parent(s)or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee(IRB/IEC)
The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

Exclusion Criteria

The patient has received treatment with any investigational drug, other than velaglucerase alfa, or investigational device within 30 days prior to study entry; such use during the study is not permitted.
The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
The patient has a significant comorbidity, as determined by the Investigator that might affect study data or confound the study results.
The patient is unable to comply with the protocol as determined by the Investigator.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01842841). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.