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Intra-monocyte Imiglucerase Kinetics in Gaucher Disease

Information source: University Hospital, Clermont-Ferrand
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Gaucher Disease

Intervention: Imiglucérase (drug) pharmacokinetics (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: University Hospital, Clermont-Ferrand

Official(s) and/or principal investigator(s):
Marc BERGER, Principal Investigator, Affiliation: University Hospital, Clermont-Ferrand

Overall contact:
Patrick LACARIN, Phone: 04 73 75 11 95, Email: placarin@chu-clermontferrand.fr

Summary

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).

Clinical Details

Official title: Study of Intra-monocytic Imiglucerase Kinetic and Its Correlation With Clinical and Biological Gaucher Disease

Study design: Allocation: Non-Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Enzyme intra-monocyte activity (sum of endogenous enzyme activities and therapeutic enzyme)in patients treated with imiglucerase

Secondary outcome:

Residual rate

Endogeneous intra-monocyte glucocérébrosidase activity from untreated patients

Biomarker dosages will provide serum concentration values

Detailed description: 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).

Eligibility

Minimum age: 12 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

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

Exclusion Criteria:

- 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

Locations and Contacts

Patrick LACARIN, Phone: 04 73 75 11 95, Email: placarin@chu-clermontferrand.fr

CHU Clermont-Ferrand, Clermont-Ferrand 63003, France; Recruiting
Patrick LACARIN, Phone: 04 73 75 11 95, Email: placarin@chu-clermontferrand.fr
Marc BERGER, Principal Investigator
Nadia BELMATOUG, Sub-Investigator
Agathe MASSEAU, Sub-Investigator
Christine SERRATRICE, Sub-Investigator
Christian ROSE, Sub-Investigator
Vassili VALAYANNOPOULOS, Sub-Investigator
Thierry BILLETTE DE VILLEMEUR, Sub-Investigator
Olivier LIDOVE, Sub-Investigator
Christian LAVIGNE, Sub-Investigator
Pierre KAMINSKY, Sub-Investigator
Yves-Marie PERS, Sub-Investigator
Catherine CAILLAUD, Sub-Investigator
Additional Information

Starting date: November 2012
Last updated: September 24, 2013

Page last updated: August 20, 2015

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