No registrations found.
ID
Source
Brief title
Health condition
Type I Gaucher disease.
Sponsors and support
Intervention
Outcome measures
Primary outcome
Stabilization of liver ratio (mL livervolume/kg bodyweight).
Secondary outcome
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.
Background summary
Gaucher 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).
Study objective
To 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.
Study design
N/A
Intervention
Lowering of the frequency of enzyme replacement therapy to once every four weeks.
Department of Internal Medicine, F4-279,
P.O. Box 22660
C.E.M. Hollak
Meibergdreef 9
Amsterdam 1100 DD
The Netherlands
+31 (0)20 5666071
c.e.hollak@amc.uva.nl
Department of Internal Medicine, F4-279,
P.O. Box 22660
C.E.M. Hollak
Meibergdreef 9
Amsterdam 1100 DD
The Netherlands
+31 (0)20 5666071
c.e.hollak@amc.uva.nl
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
N/A
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| NTR-new | NL724 |
| NTR-old | NTR734 |
| Other | : N/A |
| ISRCTN | ISRCTN51027260 |