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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:
NCT00731016
First received: August 1, 2008
Last updated: July 4, 2013
Last verified: July 2013

August 1, 2008
July 4, 2013
October 2008
April 2013   (final data collection date for primary outcome measure)
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 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00731016 on ClinicalTrials.gov Archive Site
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
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.

Not Provided
Interventional
Phase 2
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Hutchinson-Gilford Progeria Syndrome
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
1
Zoledronic acid, pravastatin
Intervention: Drug: Zoledronic acid, pravastatin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
15
July 2013
April 2013   (final data collection date for primary outcome measure)

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
Both
3 Years and older
No
Contact information is only displayed when the study is recruiting subjects
France
 
NCT00731016
2008-002471-27, 2008-15
No
Assistance Publique Hopitaux De Marseille
Assistance Publique Hopitaux De Marseille
Not Provided
Principal Investigator: Nicolas LEVY, MD Assistance Publique des Hopitaux de Marseille
Assistance Publique Hopitaux De Marseille
July 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP