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
| ISRCTN | ISRCTN51027260 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN51027260 |
| Secondary identifying numbers | NTR734 |
- Submission date
- 12/10/2006
- Registration date
- 12/10/2006
- Last edited
- 15/10/2008
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr C E M Hollak
Scientific
Scientific
Academic Medical Center (AMC)
Department of Internal Medicine, F4-279
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
| Phone | +31 (0)20 5666071 |
|---|---|
| c.e.hollak@amc.uva.nl |
Study information
| Study design | Randomised controlled trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| Scientific title | |
| Study acronym | Q2Q4 |
| Study objectives | 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. |
| Ethics approval(s) | Received from the local medical ethics committee |
| Health condition(s) or problem(s) studied | Gaucher disease |
| Intervention | Lowering of the frequency of enzyme replacement therapy to once every four weeks: 1. Imiglucerase once every four weeks 2. Imiglucerase once every one or two weeks (normal therapy) |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Imiglucerase |
| Primary outcome measure | Stabilisation of liver ratio (mL liver volume/kg body weight) |
| Secondary outcome measures | 1. Stabilisation of chitotriosidase (in patients who are not deficient for the chitotriosidase gene, 6% of population) 2. Stabilisation of haemoglobin and platelet count 3. Stabilisation of hexosaminidase 4. Stabilisation of spleen volume 5. Stabilisation of QCSI 6. Change in quality of life (QOL) 7. Stabilisation of aspartate aminotransferase (ASAT), alanine aminotransferase (ALAT), gamma-glutamyl transferase (y-GT), lactate dehydrogenase (LDH), alkaline phosphatase (AF), angiotensin converting fnzyme (ACE), ferritin |
| Overall study start date | 28/05/2003 |
| Completion date | 01/11/2004 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 11 |
| Key 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: 3.1. Haemoglobin levels within normal limits (male more than 8.0 mmol/L, female more than 7.5 mmol/L) 3.2. Platelet count more than 100 x 10^9/L 3.3. No or asymptomatic organomegaly 3.4. No evidence of clinical bone disease, such as avascular necrosis, pathologic fractures, orthopaedic replacement or bone-crises 3.5. Quantitative Chemical Shift Imaging (QCSI) levels of more than 23% 3.6. 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 |
| Key exclusion criteria | Does not comply with the above inclusion criteria |
| Date of first enrolment | 28/05/2003 |
| Date of final enrolment | 01/11/2004 |
Locations
Countries of recruitment
- Netherlands
Study participating centre
Academic Medical Center (AMC)
Amsterdam
1100 DD
Netherlands
1100 DD
Netherlands
Sponsor information
Academic Medical Center (AMC) (The Netherlands)
Hospital/treatment centre
Hospital/treatment centre
Department of Internal Medicine
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
"ROR" |
https://ror.org/03t4gr691 |
|---|
Funders
Funder type
Hospital/treatment centre
Academic Medical Center (AMC) (The Netherlands)
Private sector organisation / Universities (academic only)
Private sector organisation / Universities (academic only)
- Alternative name(s)
- Academic Medical Center, AMC
- Location
- Netherlands
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
