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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Hutchinson-Gilford Progeria Syndrome	Drug: Zoledronic acid, pravastatin	Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	15 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
Study Start Date :	October 2008
Actual Primary Completion Date :	April 2013
Actual Study Completion Date :	July 2013

Arms and Interventions

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Arm	Intervention/treatment
1; Zoledronic acid, pravastatin	Drug: Zoledronic acid, pravastatin; Pravastatin : 10 mg daily Zoledronic acid : slow (30 mn) intravenous injections, diluted into 50 ml of saline solution following this schedule : injection 1, S1: 0.0125 mg/kg of zoledronic acid; injection 2, S6: 0.025 mg/kg of zoledronic acid; injection 3, S12 and following, trimestrial basis, 0.05 mg/kg of zoledronic acid

Outcome Measures

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Primary Outcome Measures :

1. 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 [ Time Frame: 48 months ]

Secondary Outcome Measures :

1. To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ]

Eligibility Criteria

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Ages Eligible for Study:	3 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

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Locations

France
Laboratoire de Génétique Moléculaire - Hopital de la Timone
Marseille, France, 13385

Sponsors and Collaborators

Assistance Publique Hopitaux De Marseille

Investigators

Principal Investigator:	Nicolas LEVY, MD	Assistance Publique des Hopitaux de Marseille

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:	NCT00731016 History of Changes
Other Study ID Numbers:	2008-002471-27; 2008-15
First Posted:	August 8, 2008
Last Update Posted:	July 8, 2013
Last Verified:	July 2013

Additional relevant MeSH terms:

Syndrome; Progeria; Disease; Pathologic Processes; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Zoledronic acid; Diphosphonates; Pravastatin	Bone Density Conservation Agents; Physiological Effects of Drugs; Anticholesteremic Agents; Hypolipidemic Agents; Antimetabolites; Molecular Mechanisms of Pharmacological Action; Lipid Regulating Agents; Hydroxymethylglutaryl-CoA Reductase Inhibitors; Enzyme Inhibitors