Hutchinson-Gilford Progeria SyndromePhase 2NCT00731016
Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
A Phase 2 study testing Zoledronic acid, pravastatin in people with Hutchinson-Gilford Progeria Syndrome, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Hutchinson-Gilford Progeria Syndrome
- What is being tested?
- Zoledronic acid, pravastatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2008to Jul 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
France1 site
Laboratoire de Génétique Moléculaire - Hopital de la Timone
Marseille, 13385
About this study
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.
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Being tested
- Zoledronic acid, pravastatin
- Medical subject headings
- Progeria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zoledronic acid, pravastatinDrug
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 group | Role | What they receive |
|---|---|---|
| 1 | Other | Zoledronic acid, pravastatin Receives: Drug: Zoledronic acid, pravastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 48 months
Secondary outcomes (1)
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2008 (estimated)
- Study startOct 2008
- Primary completionApr 2013
- Study completionJul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hutchinson-Gilford Progeria SyndromeStudies of the same condition, open and under way.
- More trials run by Assistance Publique Hopitaux De MarseilleThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Nicolas LEVY, MD, Principal investigator, Assistance Publique des Hopitaux de Marseille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00731016
- Sponsor's protocol ID
- 2008-002471-27
- Other IDs
- 2008-15, 2008
- Status last verified
- Jul 2013
- First submitted
- 1 Aug 2008
- Last updated
- 8 Jul 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of the Hutchinson-Gilford Progeria Syndrome With a Combination of Pravastatin and Zoledronic Acid
- ClinicalTrials.gov ID
- NCT00731016
- Acronym / sponsor ID
- 2008-002471-27
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- Oct 2008
- Primary completion
- Apr 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 8 Aug 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Drug: Zoledronic acid, pravastatinZoledronic acid, pravastatin |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Zoledronic acid, pravastatinPravastatin : 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 | Drug | 1 |
Primary outcome
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
1 secondary outcome
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms
Time frame: 48 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2013.
Related trials for Hutchinson-Gilford Progeria Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nicolas LEVY, MD principal investigator
Assistance Publique des Hopitaux de Marseille
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Zoledronic acid, pravastatin in people with Hutchinson-Gilford Progeria Syndrome, run by Assistance Publique Hopitaux De Marseille at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2013.