Condition category
Nutritional, Metabolic, Endocrine
Date applied
12/10/2006
Date assigned
12/10/2006
Last edited
15/10/2008
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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

Sponsor information

Organisation

Academic Medical Center (AMC) (The Netherlands)

Sponsor details

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

Sponsor type

Hospital/treatment centre

Website

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

Publication citations

Additional files

Editorial Notes