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

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ClinicalTrials.gov Identifier: NCT00731016
Recruitment Status : Completed
First Posted : August 8, 2008
Last Update Posted : July 8, 2013
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille

Tracking Information
First Submitted Date  ICMJE August 1, 2008
First Posted Date  ICMJE August 8, 2008
Last Update Posted Date July 8, 2013
Study Start Date  ICMJE October 2008
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 5, 2008)
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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 5, 2008)
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
Official Title  ICMJE Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hutchinson-Gilford Progeria Syndrome
Intervention  ICMJE 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
Study Arms  ICMJE 1
Zoledronic acid, pravastatin
Intervention: Drug: Zoledronic acid, pravastatin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 5, 2008)
15
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2013
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00731016
Other Study ID Numbers  ICMJE 2008-002471-27
2008-15
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Assistance Publique Hopitaux De Marseille
Study Sponsor  ICMJE Assistance Publique Hopitaux De Marseille
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Nicolas LEVY, MD Assistance Publique des Hopitaux de Marseille
PRS Account Assistance Publique Hopitaux De Marseille
Verification Date July 2013

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