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

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.

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Key information

Conditions

Hutchinson-Gilford Progeria Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Laminopathies Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Progeria

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Laboratoire de Génétique Moléculaire - Hopital de la Timone

Marseille, 13385, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Zoledronic acid, pravastatin

Drug

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

Primary outcomes

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 outcomes

To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms

Time frame: 48 months

Sponsors and collaborators

Lead sponsor

Assistance Publique Hopitaux De Marseille

Other

Registry information

Important dates

Study start: 2008
Primary completion: 2013
Study completion: 2013
First posted: Aug 8, 2008
Registry last updated: Jul 8, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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