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

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	Complete list of historical versions of study NCT00731016 on ClinicalTrials.gov Archive Site
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 Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	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	Phase 2
Study Design ICMJE	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	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	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	Sexes Eligible for Study: All
Ages	3 Years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers	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	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