Informations générales (source: ClinicalTrials.gov)
Study of Intra-monocytic Imiglucerase Kinetic and Its Correlation With Clinical and Biological Gaucher Disease (CIMI)
Interventional
Phase 2
University Hospital, Clermont-Ferrand (Voir sur ClinicalTrials)
novembre 2012
juin 2016
29 juin 2024
Rational: Imiglucerase has been used to treat Gaucher disease since 1997 but data about
its pharmacokinetics have been partial; investigators know that imiglucerase undergoes a
quick clearance from plasma compartment following the infusion (1/2 life: 1-6 min, from
tissue: <24h), an observation apparently contradictory with usual infusion rhythm (one
infusion every two weeks). Furthermore, by going by GD response, the rhythm of Infusion
is sometimes diminished (for example, every 3 or 4 wks) without pharmacological rational
; In parallel, investigators demonstrated that monocytes represent a satisfactory
surrogate of GD target cells and that enzyme activity into monocytes varies between
individuals.
Our hypothesis is that enzyme activity into monocyte compartment could be different and
could be related to GD response.
Primary purpose: to evaluate the pharmacokinetics of Imiglucerase activity into target
cellular compartment depending on dose and frequency of infusions.
Secondary purposes : 1) to establish a possible relationship between the intra-monocytic
activity of glucocerebrosidase and the clinical and biological activity of Gaucher
disease and to define a possible threshold value of enzyme activity; 2) to establish a
better correlation with known biomarkers of disease (routine markers and markers recently
identified), which would better predict and / or monitor response to treatment ; 3) to
compare the residual and natural rate of activity enzyme intra-monocytic for untreated
patients (low severity disease).
Etablissements
| Les établissements hors Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CHU Clermont-Ferrand - 63003 - Clermont-Ferrand - France | Patrick LACARIN | Contact (sur clinicalTrials) | |||
Critères
Tous
Inclusion Criteria:
- Patient older than 12 years old with Gaucher disease type 1 or type 3 and having
signed an informed consent
- Treated with imiglucerase (Cerezyme ®) with a stable therapeutic strategy for at
least 3 months.
OR
- Untreated patient, with no therapeutic indication at the time of inclusion and
having a diagnosis older than 2 years (non progressive disease)
- Patients must have a social security system
- Patient older than 12 years old with Gaucher disease type 1 or type 3 and having
signed an informed consent
- Treated with imiglucerase (Cerezyme ®) with a stable therapeutic strategy for at
least 3 months.
OR
- Untreated patient, with no therapeutic indication at the time of inclusion and
having a diagnosis older than 2 years (non progressive disease)
- Patients must have a social security system
- Age <12 years old
- Gaucher disease unproven
- Gaucher disease treated with therapeutic changes in the previous 3 months, or
current treatment different from imiglucerase
- Gaucher disease untreated whose diagnosis was established for under 2 years