Phase 3 N=40 Treatment

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Gaucher Disease

Bottom Line

View on ClinicalTrials.gov: NCT00478647 ↗

Enrolled (actual)

Serious AEs

10.0%

Results posted

Sep 2010

Primary outcome: Primary: Participants Who Experienced at Least One Adverse Event — 34 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: GA-GCB (velaglucerase alfa) (Biological)
Age: Pediatric, Adult, Older Adult · 2+ yrs
Sex: All
Sponsor: Shire
Primary completion: Jun 2009

Outcome Measures

Outcome	Result	p-value
PRIMARY Participants Who Experienced at Least One Adverse Event	34	—
SECONDARY Change From Baseline to Week 53 in Hemoglobin Concentration	-0.101	—
SECONDARY Percent Change From Baseline to Week 53 in Platelet Count	7.04	—
SECONDARY Percent Change From Baseline to Week 51 in Normalized Liver Volume	-0.03	—
SECONDARY Percent Change From Baseline to Week 51 in Normalized Spleen Volume	-5.56	—

Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every other week dosing of GA-GCB (velaglucerase alfa) in participants with type 1 Gaucher disease who were previously treated with imiglucerase.

Eligibility Criteria

Inclusion Criteria

Includes:

The participant has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and the participant/legal guardian is willing and able to provide written informed consent prior to initiating any study-related procedures
The participant has received consistent treatment with imiglucerase at a dose ≤ 60 U/kg and ≥ 15 U/kg every other week for a minimum of 30 consecutive months. Participants who are anti-imiglucerase antibody positive will be allowed to enter this study
The participant is at least 2 years of age
Female participants of child-bearing potential agree to use a medically acceptable method of contraception. Male participants must agree to use a medically acceptable method of birth control
Participant must be sufficiently co-operative to participate in the study as judged by the Investigator.

Exclusion Criteria

Includes:

The participant has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease
The participant has received treatment with any investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted
Participant is HIV positive
Participant is hepatitis B/C positive
The participant presents with sustained iron, folic acid and/or vitamin B12 deficiency-related anemia during Screening
The participant, participant's parent(s), or participant's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
The participant has a significant comorbidity that might affect study data or confound the study results
The participant is unable to comply with the protocol or is otherwise unlikely to complete the study, as determined by the Investigator
The participant has experienced an anaphylactic/anaphylactoid reaction during treatment with imiglucerase
The participant has received miglustat during the 6 months prior to study enrollment
The participant has an active, clinically significant spleen infarction
The participant has active, progressive bone necrosis
The participant is a pregnant and/or lactating female

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00478647). 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.