AngiomyolipomaPhase 4NCT01217125
Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis
A Phase 4 study testing Sirolimus in people with Angiomyolipoma, run by Fundacio Puigvert.
Trial summary
The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Angiomyolipoma
- What is being tested?
- Sirolimus
- How many people
- 18 participants
- Where
- No sites listed yet
- Study dates
- Oct 2008to May 2011
- Phase
- Phase 4
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of tuberousclerosis Angiomyolipoma >2cm age>10 years
Who cannot join
- Creatinine >4 No recent AML bleeding No hepatic abnormalities (liver tests 2fold) Hematocrit<27% Thrombocytopenia (<100.000/mm3) Leukopenia (<3000/mm3) Ischemic cardiopathy Recent surgery (2 months prior to enrollment) Pregnancy Serum cholesterol over 7.8 mmol/l or hypertriglyceridemia fasten(>4.6 mmol/l) non controlled with drugs malignancy in the previous 2 years allergy to macrolides
About this study
Official title: CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS
The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis.
- Conditions
- Angiomyolipoma
- Being tested
- Sirolimus
- Keywords
- tuberousclerosis, angiomyolipoma, rapamycin
- Medical subject headings
- Angiomyolipoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Also called Rapamune
plasma levels between 4 and 8
What is being measured
The primary outcome is the main question the study is designed to answer.
Angiomyolipoma volume
Measured by MRI
Measured: 2 years
Secondary outcomes (1)
skin lesions, AML complications,
Evaluate skin lesions Collect event realted to AML
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2010 (estimated)
- Study startOct 2008
- Primary completionMay 2011
- Study completionMay 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AngiomyolipomaStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundacio Puigvert
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, Spain
- Responsible party
- Principal investigator, Roser Torra, Fundacio Puigvert
- Investigators
- Roser Torra, MD,PhD, Principal investigator, Fundacio Puigvert, Barcelona
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01217125
- Sponsor's protocol ID
- EudraCT 2007-005978-30
- Status last verified
- Mar 2012
- First submitted
- 6 Oct 2010
- Last updated
- 8 Mar 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Cabrera-Lopez C, Marti T, Catala V, Torres F, Mateu S, Ballarin J, Torra R. Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial. Orphanet J Rare Dis. 2012 Nov 11;7:87. doi: 10.1186/1750-1172-7-87.
PubMed 23140536
Study at a glance
From the registry record, last updated by the sponsor on 8 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- CLINICAL TRIAL TO DETERMINE THE EFFICACY AND SAFETY OF RAPAMYCIN IN ANGIOMYOLIPOMAS IN PATIENTS WITH TUBEROUS SCLEROSIS
- ClinicalTrials.gov ID
- NCT01217125
- Acronym / sponsor ID
- EudraCT 2007-005978-30
- Conditions
- Angiomyolipoma
- Keywords
- tuberousclerosis, angiomyolipoma, rapamycin
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Fundacio PuigvertUniversity, hospital or other
- Collaborators
- Ministry of Health, Spain
- Study start
- Oct 2008
- Primary completion
- May 2011 (actual)
- Study completion
- May 2011 (actual)
- First posted
- 8 Oct 2010 (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.
The purpose of this study is to determine whether rapamycin is safe and effective in the treatment of renal angiomyolipomas in patients with tuberousclerosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Diagnosis of tuberousclerosis Angiomyolipoma >2cm age>10 years
Key exclusion criteria (1 listed)
- Creatinine >4 No recent AML bleeding No hepatic abnormalities (liver tests 2fold) Hematocrit<27% Thrombocytopenia (<100.000/mm3) Leukopenia (<3000/mm3) Ischemic cardiopathy Recent surgery (2 months prior to enrollment) Pregnancy Serum cholesterol over 7.8 mmol/l or hypertriglyceridemia fasten(>4.6 mmol/l) non controlled with drugs malignancy in the previous 2 years allergy to macrolides
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 |
|---|---|---|
| SirolimusAlso: Rapamuneplasma levels between 4 and 8 | Drug | – |
Primary outcome
Angiomyolipoma volume
Time frame: 2 years
Measured by MRI
1 secondary outcome
skin lesions, AML complications,
Time frame: 2 years
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 Mar 2012.
- Derived from this study: PubMed 23140536Cabrera-Lopez C, Marti T, Catala V, Torres F, Mateu S, Ballarin J, Torra R. Assessing the effectiveness of rapamycin on angiomyolipoma in tuberous sclerosis: a two years trial. Orphanet J Rare Dis. 2012 Nov 11;7:87. doi: 10.1186/1750-1172-7-87.
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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.
Roser Torra, MD,PhD principal investigator
Fundacio Puigvert, Barcelona
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Sirolimus in people with Angiomyolipoma, run by Fundacio Puigvert.
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 1 inclusion and 1 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 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Angiomyolipoma volume (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01217125, which also lists 1 linked publication.
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 Mar 2012.