An open randomised study comparing efficacy of maintenance therapy with imiglucerase at a frequency of once every four weeks versus the original schedule (once every one or two weeks) in adult type I Gaucher disease patients.

An open randomised study comparing efficacy of maintenance therapy with imiglucerase at a frequency of once every four weeks versus the original schedule (once every one or two weeks) in adult type I Gaucher disease patients."

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON21048

Enrollment

Registered

2006-09-12

Start date

2003-05-28

Completion date

Unknown

Last updated

2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Type I Gaucher disease.

Interventions

Lowering of the frequency of enzyme replacement therapy to once every four weeks.

Eligibility

Inclusion criteria

Inclusion criteria: 1. Patients, older than 18 years, with proven Gaucher type I disease, as evidenced by decreased plasma glucocerebrosidase activity or genotyping. 2. Patients who have received enzyme therapy for at least two years prior to study enrolment.. 3. Patients with mild, stable Gaucher disease, as defined by having all of the following throughout the 24 months prior to screening: a. haemoglobin levels within normal limits (male >8.0 mmol/L, female >7.5 mmol/L) b. platelet count >100 x 109/L c. no or asymptomatic organomegaly d. no evidence of clinical bone disease, such as avascular necrosis, pathologic fractures, orthopaedic replacement or bone-crises. e. QCSI levels of > 23% f. a maximum variability of 30% in plasma chitotriosidase levels 4. Patients who have provided written informed consent to participate in the study. 5. Patients who are co-operative, able to understand the nature and scope of the study, and who are expected to be generally compliant.

Exclusion criteria

Exclusion criteria: N/A

Design outcomes

Primary

Measure	Time frame
Stabilization of liver ratio (mL livervolume/kg bodyweight).	—

Secondary

Measure	Time frame
1. Stabilization of chitotriosidase (in patients who are not deficient for the chitotriosidase gene, 6% of population); 2. Stabilization of haemoglobin and platelet count; 3. Stabilization of hexosaminidase; 4. Stabilization of spleen volume; 5. Stabilization of QCSI; 6. Change in QOL; 7. Stabilization of ASAT, ALAT, y-GT, LDH, AF, ACE, ferritin.	—

Contacts

Public ContactC.E.M. Hollak

Academic Medical Center (AMC), Department of Internal Medicine, F4-279, P.O. Box 22660

c.e.hollak@amc.uva.nl+31 (0)20 5666071

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)