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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:
Assistance Publique Hopitaux De Marseille
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
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
| Hutchinson-Gilford Progeria Syndrome | Drug: Zoledronic acid, pravastatin | Phase 2 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
| Official Title: | Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid |
Resource links provided by NLM:
Further study details as provided by Assistance Publique Hopitaux De Marseille:
Primary Outcome Measures:
- 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 ]
Secondary Outcome Measures:
- To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms [ Time Frame: 48 months ]
| Enrollment: | 15 |
| Study Start Date: | October 2008 |
| Study Completion Date: | July 2013 |
| Primary Completion Date: | April 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1
Zoledronic acid, pravastatin
|
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 :
|
Eligibility| Ages Eligible for Study: | 3 Years and older (Child, Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00731016
Please refer to this study by its ClinicalTrials.gov identifier: NCT00731016
Locations
| France | |
| Laboratoire de Génétique Moléculaire - Hopital de la Timone | |
| Marseille, France, 13385 | |
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
| Principal Investigator: | Nicolas LEVY, MD | Assistance Publique des Hopitaux de Marseille |
More Information
| Responsible Party: | Assistance Publique Hopitaux De Marseille |
| ClinicalTrials.gov Identifier: | NCT00731016 History of Changes |
| Other Study ID Numbers: |
2008-002471-27 2008-15 |
| Study First Received: | August 1, 2008 |
| Last Updated: | July 4, 2013 |
Additional relevant MeSH terms:
|
Syndrome Progeria Disease Pathologic Processes Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases Zoledronic acid Diphosphonates Pravastatin |
Bone Density Conservation Agents Physiological Effects of Drugs Anticholesteremic Agents Hypolipidemic Agents Antimetabolites Molecular Mechanisms of Pharmacological Action Lipid Regulating Agents Hydroxymethylglutaryl-CoA Reductase Inhibitors Enzyme Inhibitors |
ClinicalTrials.gov processed this record on September 21, 2017