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

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

Study designRandomised controlled trial
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Not specified
Study typeTreatment
Scientific title
Study acronymQ2Q4
Study objectivesTo 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) studiedGaucher disease
InterventionLowering 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 typeDrug
Pharmaceutical study type(s)
PhaseNot Specified
Drug / device / biological / vaccine name(s)Imiglucerase
Primary outcome measureStabilisation of liver ratio (mL liver volume/kg body weight)
Secondary outcome measures1. 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 date28/05/2003
Completion date01/11/2004

Eligibility

Participant type(s)Patient
Age groupAdult
Lower age limit18 Years
SexBoth
Target number of participants11
Key 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:
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 criteriaDoes not comply with the above inclusion criteria
Date of first enrolment28/05/2003
Date of final enrolment01/11/2004

Locations

Countries of recruitment

  • Netherlands

Study participating centre

Academic Medical Center (AMC)
Amsterdam
1100 DD
Netherlands

Sponsor information

Academic Medical Center (AMC) (The Netherlands)
Hospital/treatment centre

Department of Internal Medicine
P.O. Box 22660
Amsterdam
1100 DD
Netherlands

ROR logo "ROR" https://ror.org/03t4gr691

Funders

Funder type

Hospital/treatment centre

Academic Medical Center (AMC) (The Netherlands)
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 shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan