Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
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ClinicalTrials.gov Identifier: NCT00731016 |
Recruitment Status :
Completed
First Posted : August 8, 2008
Last Update Posted : July 8, 2013
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Condition or disease | Intervention/treatment | Phase |
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Hutchinson-Gilford Progeria Syndrome | Drug: Zoledronic acid, pravastatin | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | N/A |
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 |

Arm | Intervention/treatment |
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1
Zoledronic acid, pravastatin
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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 :
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- 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 ]
- To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ]

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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00731016
France | |
Laboratoire de Génétique Moléculaire - Hopital de la Timone | |
Marseille, France, 13385 |
Principal Investigator: | Nicolas LEVY, MD | Assistance Publique des Hopitaux de Marseille |
Responsible Party: | Assistance Publique Hopitaux De Marseille |
ClinicalTrials.gov Identifier: | NCT00731016 |
Other Study ID Numbers: |
2008-002471-27 2008-15 |
First Posted: | August 8, 2008 Key Record Dates |
Last Update Posted: | July 8, 2013 |
Last Verified: | July 2013 |
Progeria Syndrome Disease Pathologic Processes Laminopathies Genetic Diseases, Inborn Metabolism, Inborn Errors Metabolic Diseases Zoledronic Acid 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 |