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

NCT ID: NCT00731016

Last Updated: 2013-07-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-10-31
• Study Completion Date: 2013-07-31

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.

Conditions

Hutchinson-Gilford Progeria Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

1

Zoledronic acid, pravastatin

Group Type: OTHER

Zoledronic acid, pravastatin

Intervention Type: 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

Interventions

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

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique Hopitaux De Marseille

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nicolas LEVY, MD

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique des Hopitaux de Marseille

Locations

Laboratoire de Génétique Moléculaire - Hopital de la Timone

Marseille, , France

Countries

France

Other Identifiers

2008-15

Identifier Type: -

Identifier Source: secondary_id

2008-002471-27

Identifier Type: -

Identifier Source: org_study_id