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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
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

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
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

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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, Older Adult)
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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Information from the National Library of Medicine

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


Locations
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France
Laboratoire de Génétique Moléculaire - Hopital de la Timone
Marseille, France, 13385
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
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Principal Investigator: Nicolas LEVY, MD Assistance Publique des Hopitaux de Marseille
More Information
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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
Additional relevant MeSH terms:
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Progeria
Syndrome
Disease
Pathologic Processes
Metabolism, Inborn Errors
Genetic Diseases, Inborn
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