Contact information
Type
Scientific
Primary contact
Dr C E M Hollak
ORCID ID
Contact details
Academic Medical Center (AMC)
Department of Internal Medicine
F4-279
P.O. Box 22660
Amsterdam
1100 DD
Netherlands
+31 (0)20 5666071
c.e.hollak@amc.uva.nl
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
NTR734
Study information
Scientific title
Acronym
Q2Q4
Study hypothesis
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
Received from the local medical ethics committee
Study design
Randomised controlled trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
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
Phase
Not Specified
Drug names
Imiglucerase
Primary outcome measures
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 trial start date
28/05/2003
Overall trial end date
01/11/2004
Reason abandoned
Eligibility
Participant 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
Participant type
Patient
Age group
Adult
Gender
Both
Target number of participants
11
Participant exclusion criteria
Does not comply with the above inclusion criteria
Recruitment start date
28/05/2003
Recruitment end date
01/11/2004
Locations
Countries of recruitment
Netherlands
Trial participating centre
Academic Medical Center (AMC)
Amsterdam
1100 DD
Netherlands
Funders
Funder type
Hospital/treatment centre
Funder name
Academic Medical Center (AMC) (The Netherlands)
Alternative name(s)
Academic Medical Center, AMC
Funding Body Type
private sector organisation
Funding Body Subtype
academic
Location
Netherlands
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary