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

NCT00731016 | Completed Phase 2

• 2 other identifiers: 2008-002471-272008-15
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (1)

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

  48 months

Study Arms (1)

1

OTHER

Zoledronic acid, pravastatin

Drug: Zoledronic acid, pravastatin

Interventions

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

1

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Molecularly characterised patients with a known mutation of their LMNA gene leading to the production of a farnesylated prelamin A, whether truncated or not
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique Hopitaux De Marseille: lead

Study Sites (1)

Laboratoire de Génétique Moléculaire - Hopital de la Timone

Marseille, 13385, France

Location

MeSH Terms

Conditions

Progeria

Interventions

Zoledronic Acid; Pravastatin

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Polycyclic Compounds

Study Officials

• Nicolas LEVY, MD

  Assistance Publique des Hopitaux de Marseille

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2008
• First Posted: August 8, 2008
• Study Start: October 1, 2008
• Primary Completion: April 1, 2013
• Study Completion: July 1, 2013
• Last Updated: July 8, 2013

Record last verified: 2013-07

Locations

FR (1)