Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid

Phase 2

Completed

• Conditions: Hutchinson-Gilford Progeria Syndrome
• Interventions: Drug: Zoledronic acid, pravastatin

• Registration Number: NCT00731016
• Lead Sponsor: Assistance Publique Hopitaux De Marseille

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 delay the gravest infringements of the disease, to prolong the life of the children, and in a more general way, aim at improving their living conditions.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2008-08-01
• Study First Submitted QC: 2008-08-05
• Study First Posted: 2008-08-08
• Study Start: 2008-10
• Primary Completion: 2013-04
• Status Verified: 2013-07
• Study Completion: 2013-07
• Last Update Submitted: 2013-07-04
• Last Update Posted: 2013-07-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

• 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 Criteria

• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Zoledronic acid, pravastatin	Zoledronic acid, pravastatin

Primary Outcome Measures

Name	Time	Method
To evaluate the tolerance and efficacy of pravastatin and zoledronic acid in combination on the patient's weight, height and bone metabolism in Progeria treatment	48 months

Secondary Outcome Measures

Name	Time	Method
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms	48 months

Trial Locations

Locations (1)

Laboratoire de Génétique Moléculaire - Hopital de la Timone; 🇫🇷 Marseille, France