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

Primary Purpose

Hutchinson-Gilford Progeria Syndrome

Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Zoledronic acid, pravastatin
Sponsored by
Assistance Publique Hopitaux De Marseille
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hutchinson-Gilford Progeria Syndrome

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Laboratoire de Génétique Moléculaire - Hopital de la Timone

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

1

Arm Description

Zoledronic acid, pravastatin

Outcomes

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

Secondary Outcome Measures

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

Full Information

First Posted
August 1, 2008
Last Updated
July 4, 2013
Sponsor
Assistance Publique Hopitaux De Marseille
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1. Study Identification

Unique Protocol Identification Number
NCT00731016
Brief Title
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
Official Title
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
Study Type
Interventional

2. Study Status

Record Verification Date
July 2013
Overall Recruitment Status
Completed
Study Start Date
October 2008 (undefined)
Primary Completion Date
April 2013 (Actual)
Study Completion Date
July 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique Hopitaux De Marseille

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hutchinson-Gilford Progeria Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Other
Arm Description
Zoledronic acid, pravastatin
Intervention Type
Drug
Intervention Name(s)
Zoledronic acid, pravastatin
Intervention Description
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
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms
Time Frame
48 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nicolas LEVY, MD
Organizational Affiliation
Assistance Publique des Hopitaux de Marseille
Official's Role
Principal Investigator
Facility Information:
Facility Name
Laboratoire de Génétique Moléculaire - Hopital de la Timone
City
Marseille
ZIP/Postal Code
13385
Country
France

12. IPD Sharing Statement

Learn more about this trial

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

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