Gaucher`s diseases type 1 and bone manifestations (patients)
Conditions
Interventions
Trade Name: Zavesca
Pharmaceutical Form: Capsule*
INN or Proposed INN: MIGLUSTAT
CAS Number: 72599-27-0
Concentration unit: mg milligram(s)
Product Name: PET tracer [11C]-miglustat
Pharmaceutical Fo
Sponsors
Actelion Pharmaceuticals Sverige AB
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Age 18 years and older -Male or Female -Non-pregnant woman who is remain non-pregnant for 3 months after the end of the study: only women who are surgically sterile, who are in the menopause (no menstruation for at least one year) or those of childbearing potential who are using a reliable method of contraception. Reliable methods of contraception for female patients include the following: a. barrier type devices (e.g., female condom, diaphragm and contraceptive sponge) used ONLY in combination with a spermicidal b. intrauterine devices c. oral contraceptive agent d. Depo-Provera TM (medroxyprogesterone acetate) e. evonorgelestrel implants -Gaucher`s disease type 1 and typical bone manifestations diagnosed with a radiological examination (x-ray, MR) -Signed inform consent prior to any study-mandated procedure 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
Exclusion criteria
Exclusion criteria: -Severe renal impairment (creatinine clearance <30ml/min) F-emale paient who are not menopause (no menstruation for at least one year) and will not undergo a pregnancy test prior to enrollment in the study. -Known hypersensitivity to drugs of the same class as the study drug, or any of their excipients -Treatment with miglustat within 1 month prior to randomisation T-reatment with ERT, Enzyne Replace Therapy, within 1 month prior to randomization -Planned treatment or treatment with another investigational drug or therapy within 1 month prior to randomization.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objective of the study is to determine the biodistribution and time course of (11C)miglustat in bone tissue and brain at the therapeutic dose level of Miglustat.;Secondary Objective: ;Primary end point(s): The objective for the study is to determine the biodistrubution and time cousre of (11C)miglustat in bone tissue and brain at the therapeutic dose level og Miglustat. General uptake data, in the form of Time-Activity_Curves (TAC) for relevant tissues (i.e. bone, and brain) will be generated. If possible the PET data will be anayzed using kinetic medeling, e.g. determination of the distrubution volume (DV) of (11C)miglustat or the rate constant for transport of (11C) miglustat from plasma to tissue (K1). | — |
Countries
Sweden
Outcome results
None listed