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 2


Study Type:	Interventional
Study Design:	Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid

Resource links provided by NLM:

Genetics Home Reference related topics: Hutchinson-Gilford progeria syndrome

Drug Information available for: Pravastatin Pravastatin sodium Zoledronic acid

Genetic and Rare Diseases Information Center resources: Progeria

U.S. FDA Resources

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 ]


Enrollment:	15
Study Start Date:	October 2008
Study Completion Date:	July 2013
Primary Completion Date:	April 2013 (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 (Child, Adult, Senior)
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

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


Responsible Party:	Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:	NCT00731016 History of Changes
Other Study ID Numbers:	2008-002471-27 2008-15
Study First Received:	August 1, 2008
Last Updated:	July 4, 2013
Health Authority:	France: Ministry of Health

Additional relevant MeSH terms:


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

ClinicalTrials.gov processed this record on September 26, 2016