A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease
- Conditions
- Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol: 1BACKGROUND AND RATIONALE 1.1Disease
- Registration Number
- EUCTR2005-001651-37-GB
- Lead Sponsor
- Actelion Pharmaceuticals Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 50
Eligible patients must meet all of the following inclusion criteria:
1.Patients aged 18 years or older at the study entry with a confirmed diagnosis of type 1 mild to moderate Gaucher disease at the time of enrollment. Diagnosis will be confirmed on glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene.
2.Patients treated with ERT for at least 3 years and who are receiving their current dose for a minimum of last six months.
3.Clinically and biologically stable for the previous 2 years, i.e. (with at least 3 timepoint assessments available for each parameters)
a.Stable organomegaly, defined by <= 10% variation.
b.Free of progressive symptomatic bone disease (no proven bone disease progression for the last 2 years)
c.Controlled hemoglobin level above 11 g/dl ,
d.Controlled platelets counts with a mean of at least 100x10 Power 9 /l
e.Stable chitotriosidase activity, defined by <= 20% variation.
4.Patients who provide written informed consent to participate in the study
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Eligible patients must meet none of the following exclusion criteria:
1.Patients (males and females) who do not agree to use reliable contraception throughout the study and for three months after cessation of miglustat treatment.
2.Female patients who are pregnant or breast feeding, or will not undergo a pregnancy test prior to enrollment into the study.
3.Patients with a history of significant lactose intolerance.
4.Patients with a confirmed neuropathy.
5.Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 6 months of Visit 1, or who have a history of significant gastrointestinal disorders (e.g., Irritable Bowel Syndrome).
6.Patients with history of cataracts or known increased risk of cataract formation.
7.Patients with severe renal impairment i.e., with a creatinine clearance <30 ml/min/1.73mPower2
8.Patients who are not ambulant, or have severe bone disease according to the bone history and symptom list (see appendix 9)
9.Patients splenectomised before 18 years old, because of massive splenomegaly or severe cytopenia.
10.Patients with an intercurrent medical condition that would render them unsuitable for study.
11.Patients currently undergoing therapy with other investigational product, or who have received an investigational product within 3 months prior to study start.
12.Patients with an intercurrent active medical condition such as HIV or Hepatitis B/C that would render them unsuitable for study.
13.Patients who for whatever reason are, in the opinion of the Investigator, thought to be unsuitable for the study.
14.Patients who have previously received treatment with miglustat.
15.Patients with a history of evidence of oculomotor gaze palsies, ataxia or other manifestations typically associated with type 3 (neuronopatic”) Gaucher disease.
16.Predicted hypersensitivity to miglustat or any excipients.
17.Patients with current alcohol or drug abuse or dependence.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term (i.e. over a 24 month period) effect on liver volume of miglustat (100mg TID) as maintenance therapy in adult patients with stable mild to moderate Type 1 Gaucher disease after a switch from Enzyme Replacement Therapy (ERT) ;<br> Secondary Objective: To evaluate the long-term (i.e. over a 24 month period) effects of miglustat (100mg TID) on spleen volume and other markers of Type 1 Gaucher disease<br><br> To evaluate the long-term safety and tolerability with particular reference to any changes in the neurological assessments (including cognitive function)<br> ;<br> Primary end point(s): Percent change from baseline to Month 24 in liver volume<br><br> For the primary endpoint, the clinically acceptable difference is a mean percentage increase from Baseline to Month 24 of no greater than 10% in liver volume.<br>
- Secondary Outcome Measures
Name Time Method