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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.


- candidate number2196
- NTR NumberNTR734
- ISRCTNISRCTN51027260
- Date ISRCTN created12-okt-2006
- date ISRCTN requested11-okt-2006
- Date Registered NTR12-sep-2006
- Secondary IDsN/A 
- Public TitleAn 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.
- Scientific TitleAn 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."
- ACRONYMQ2Q4
- hypothesisTo compare the efficacy of maintenance therapy with an equal monthly dose of imiglucerase when administered at a frequency of once every four weeks versus once every one or two weeks, in adult type I Gaucher disease patients in stable and good condition during a minimum of two years on enzyme supplementation therapy.
- Healt Condition(s) or Problem(s) studiedGaucher's disease
- Inclusion criteria1. 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 criteriaN/A
- mec approval receivedyes
- multicenter trialno
- randomisedyes
- masking/blindingNone
- controlActive
- groupParallel
- Type2 or more arms, randomized
- Studytypeintervention
- planned startdate 28-mei-2003
- planned closingdate1-nov-2004
- Target number of participants11
- InterventionsLowering of the frequency of enzyme replacement therapy to once every four weeks.
- Primary outcomeStabilization of liver ratio (mL livervolume/kg bodyweight).
- Secondary outcome1. 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.
- TimepointsN/A
- Trial web siteN/A
- statusstopped: trial finished
- CONTACT FOR PUBLIC QUERIESDr. C.E.M. Hollak
- CONTACT for SCIENTIFIC QUERIESDr. M. Fost, de
- Sponsor/Initiator Academic Medical Center (AMC), Department of Internal Medicine
- Funding
(Source(s) of Monetary or Material Support)
Academic Medical Center (AMC)
- PublicationsHaematologica. 2007 Feb;92(2):215-21.
- Brief summaryGaucher disease type I can be successfully treated with enzyme replacement therapy. In order to reduce the burden of the intravenously administered enzyme, low frequency of infusion will be prospectively studied in patients with stable and minor disease following ERT. Patients will be randomly assigned to continue their original regimen (in a once every week or fortnightly schedule) or to lower the rate of infusion to once every four weeks, at the same cumulative dose. Primary endpoint is change in liver ratio (ml/kg body weight) and secondary endpoints are spleen volume, haemoglobin level, platelet count, lumbar bone marrow fat content measured with QCSI, white cell count, and plasma levels of ferritin, chitotriosidase, liver enzymes and angiotensin converting enzyme (ACE).
- Main changes (audit trail)
- RECORD12-sep-2006 - 16-jun-2008


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