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

• Status: Completed
• Trial end date: 2013-07-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique Hopitaux De Marseille
• Treatments: Diphosphonates; Pravastatin; Zoledronic Acid

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