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
Protocol serial number	NTR734
Sponsor	Academic Medical Center (AMC) (The Netherlands)
Funder	Academic Medical Center (AMC) (The Netherlands)

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

Academic Medical Center (AMC)
Department of Internal Medicine, F4-279
P.O. Box 22660
Amsterdam
1100 DD
Netherlands

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

Study information

Primary study design	Interventional
Study design	Randomised controlled trial
Secondary study design	Randomised controlled trial
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
Phase	Not Specified
Drug / device / biological / vaccine name(s)	Imiglucerase
Primary outcome measure(s)	Stabilisation of liver ratio (mL liver volume/kg body weight)
Key secondary outcome measure(s)	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
Completion date	01/11/2004

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	All
Target sample size at registration	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

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan