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

Purpose

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	Intervention	Phase
Hutchinson-Gilford Progeria Syndrome	Drug: Zoledronic acid, pravastatin	Phase II

Genetics Home Reference related topics:

Hutchinson-Gilford progeria syndrome

Drug Information available for:

Pravastatin Pravastatin sodium Zoledronic acid Sodium chloride S 1 (Combination)

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study

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

Further study details as provided by Assistance Publique Hopitaux De Marseille:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ] [ Designated as safety issue: No ]

Estimated Enrollment:	15
Study Start Date:	October 2008
Estimated Study Completion Date:	March 2013
Estimated Primary Completion Date:	September 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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

Eligibility

Ages Eligible for Study:	3 Years and older
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00731016

Contacts


Contact: Nicolas LEVY, MD	0491387787	nicolas.levy@ap-hm.fr

Contact: Sabine SIGAUDY, MD	04 91 3867 49	sabine.sigaudy@ap-hm.fr

Locations


France
	Laboratoire de Génétique Moléculaire - Hopital de la Timone	Recruiting
	MARSEILLE, France, 13385
	Contact: Nicolas LEVY, MD 049138 77 87 nicolas.levy@ap-hm.fr
	Contact: Sabine SIGAUDY, MD 04 91 3867 49 sabine.sigaudy@ap-hm.fr

Sponsors and Collaborators

Assistance Publique Hopitaux De Marseille

Investigators


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

More Information

Responsible Party:	Assistance Publique Hopitaux De Marseille ( Assistance Publique Hopitaux De Marseille )
Study ID Numbers:	2008-002471-27, 2008-15
First Received:	August 1, 2008
Last Updated:	October 8, 2008
ClinicalTrials.gov Identifier:	NCT00731016
Health Authority:	France: Ministry of Health

Study placed in the following topic categories:

Progeria

Pravastatin

Metabolism, Inborn Errors

Hutchinson Gilford progeria syndrome

Diphosphonates

Zoledronic acid

Metabolic Diseases

Genetic Diseases, Inborn

Metabolic disorder

Additional relevant MeSH terms:

Antimetabolites

Disease

Molecular Mechanisms of Pharmacological Action

Antilipemic Agents

Physiological Effects of Drugs

Enzyme Inhibitors

Bone Density Conservation Agents

Anticholesteremic Agents

Hydroxymethylglutaryl-CoA Reductase Inhibitors

Pharmacologic Actions

Pathologic Processes

Syndrome

Therapeutic Uses

ClinicalTrials.gov processed this record on October 31, 2008

Sponsored by:	Assistance Publique Hopitaux De Marseille
Information provided by:	Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:	NCT00731016