Status:
COMPLETED
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
Lead Sponsor:
Assistance Publique Hopitaux De Marseille
Conditions:
Hutchinson-Gilford Progeria Syndrome
Eligibility:
All Genders
3+ years
Phase:
PHASE2
Brief Summary
We suggest treating the Hutchinson-Gilford Progeria Syndrome by two molecules (zoledronic acid and pravastatin).The therapeutic approach which we propose has for objectives to reduce, to prevent or to...
Eligibility Criteria
Inclusion
- Molecularly characterised patients with a known mutation of their LMNA gene leading to the production of a farnesylated prelamin A, whether truncated or not
- Patients must be able to travel and consult in Marseille, France for necessary explorations planned at the inclusion step, then following the protocol flow
- chart for zoledronic acid injections and follow-up visits
- Patient older than 3 years
- Patients affiliated or beneficiary of a legal medical insurance
- Adult patients certifying they have been properly informed about the protocol, and they signed a written consent form. Children and/or disabled patients whose parents/legal tutor have been informed and have signed a written consent form
Exclusion
- Known hypersensitivity to pravastatin or zoledronic acid
- Seric transaminase levels higher than 3 times of normal value
- CPK level higher than 5 times of normal value
- Creatininemia higher than 0.5mg/dl or 44mM, or creatinin clearance lower than 70ml/min/1.73m3
- Presence of dental troubles, or recent dental trouble
- Maxillary osteonecrosis or bone nakedness antecedent
- Congenital galacosemia, glucose or galactose maladsorption syndrome, lactase deficiency
- Every other pathology thought to be incompatible with proposed treatment by the investigator
- Under treatment that can interfere with pravastatin and/or zoledronate metabolisms
Key Trial Info
Start Date :
October 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2013
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT00731016
Start Date
October 1 2008
End Date
July 1 2013
Last Update
July 8 2013
Active Locations (1)
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1
Laboratoire de Génétique Moléculaire - Hopital de la Timone
Marseille, France, 13385