Phase 2 N=7 Treatment

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Gaucher Disease, Type 3

Bottom Line

View on ClinicalTrials.gov: NCT01685216 ↗

Enrolled (actual)

Serious AEs

16.7%

Results posted

Oct 2015

Primary outcome: Primary: Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration — 2.15 g/dL

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: velaglucerase alfa (Biological)
Age: Pediatric · 2+ yrs
Sex: All
Sponsor: Shire
Primary completion: Mar 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration	2.15	—
SECONDARY Change From Baseline to 12 Months (Week 53) in Platelet Count	136.6	—
SECONDARY Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI)	-30.12	—
SECONDARY Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI)	-62.27	—
SECONDARY Number of Participants With Abnormal Neurological Status During The Study	6	—
SECONDARY Number of Participants Who Experienced a Treatment-Emergent Adverse Event	6; 1; 1	—
SECONDARY Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study	0; 1; 1; 1; 1	—

Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and the severity of these neurological symptoms. Patients with type 2 Gaucher disease present with acute neurological deterioration, and those with type 3 disease typically display a more sub acute neurological course. Type 1 Gaucher disease, the most common form accounting for more than 90% of all Gaucher disease cases, does not involve the central nervous system. The purpose of this clinical research study is to investigate the safety and effectiveness of velaglucerase alfa in patients with type 3 Gaucher disease.

Eligibility Criteria

Inclusion Criteria: Each patient must meet the following criteria to be enrolled in this study.

The patient has a confirmed diagnosis of type 3 Gaucher disease.
The patient is ≥ 2 and < 18 years of age at the time of enrollment.
The patient is either näive to treatment or has not received treatment (investigational or approved) for Gaucher disease within 12 months prior to study entry.
The patient has Gaucher disease-related anemia, defined as hemoglobin concentration below the lower limit of normal for age and sex.

AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA

The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation.
The patient has Gaucher disease-related thrombocytopenia, defined as platelet count < 120 x 10,000 platelets/cubic mm.
The patient has a Gaucher disease-related readily palpable enlarged liver.
Patients who have undergone splenectomy may still be eligible to participate in the study.
Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy testing will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the Investigator.
The patient's parent(s) or the patient's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

Exclusion Criteria: Patients who meet any of the following criteria will be excluded from this study.

The patient is suspected of having type 2 or type 1 Gaucher disease.
The patient is < 2 years of age.
The patient has experienced a severe (Grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any enzyme replacement therapy for Gaucher disease (approved or investigational).
The patient has received any non-Gaucher disease-related treatment with an investigational drug within 30 days prior to study entry.
The patient is a pregnant and/or lactating female.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01685216). 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.