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

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Assistance Publique Hopitaux De Marseille

ClinicalTrials.gov Identifier:

NCT00731016

First received: August 1, 2008

Last updated: July 4, 2013

Last verified: July 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

August 1, 2008

Last Updated Date

July 4, 2013

Start Date ^ICMJE

October 2008

Primary Completion Date

April 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00731016 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

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 Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Hutchinson-Gilford Progeria Syndrome

Intervention ^ICMJE

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

Study Arm (s)

Zoledronic acid, pravastatin

Intervention: Drug: Zoledronic acid, pravastatin

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

July 2013

Primary Completion Date

April 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

3 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Listed Location Countries ^ICMJE

France

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT00731016

Other Study ID Numbers ^ICMJE

2008-002471-27, 2008-15

Has Data Monitoring Committee

Responsible Party

Assistance Publique Hopitaux De Marseille

Study Sponsor ^ICMJE

Assistance Publique Hopitaux De Marseille

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Nicolas LEVY, MD

Assistance Publique des Hopitaux de Marseille

Information Provided By

Assistance Publique Hopitaux De Marseille

Verification Date

July 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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