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

Hutchinson-Gilford Progeria Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00731016
• Other study ID #: 2008-002471-27
• Secondary ID: 2008-15
• Status: Completed
• Phase: Phase 2
• First received: August 1, 2008
• Last updated: July 4, 2013
• Start date: October 2008
• Est. completion date: July 2013

Study information

• Verified date: July 2013
• Source: Assistance Publique Hopitaux De Marseille
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: July 2013
• Est. primary completion date: April 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years and older

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Hutchinson-Gilford Progeria Syndrome
• Progeria
• Syndrome

Intervention

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

Locations

Country	Name	City	State
France	Laboratoire de Génétique Moléculaire - Hopital de la Timone	Marseille

Sponsors (1)

Lead Sponsor	Collaborator
Assistance Publique Hopitaux De Marseille

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		48 months	No
Secondary	To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms		48 months	No