Birt-Hogg-Dubé SyndromePhase 3NCT00928798
Topical Rapamycin for Fibrofolliculomas
A Phase 3 study testing Rapamycin in people with Birt-Hogg-Dubé Syndrome, run by Maastricht University Medical Center at 2 sites in Netherlands.
Trial summary
The purpose of the study is to determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary we evaluate rapamycin safety, formula acceptance and patient satisfaction. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Birt-Hogg-Dubé Syndrome
- What is being tested?
- Rapamycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 2 sitesNetherlands
- Study dates
- Jan 2010to Aug 2011
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Minimum age of 18 years.
- At least 10 facial fibrofolliculomas, histologically confirmed.
- Entered in a screening program and free of malignancy as determined during screening (already had a baseline MRI or CT-scan).
- Being able to understand instructions.
- Mutation status must be known.
- For females: not pregnant and willing to use both oral and barrier contraceptives during the treatment period.
Who cannot join
- Not capable of informed consent.
- Age under 18 years.
- Pregnancy or failure to comply with contraceptive measures.
- Proven or suspected malignancy of skin or other organs.
- No histological confirmation.
- Skin lesions other than fibrofolliculoma that might worsen under sirolimus such as active infections.
Show all 14Show fewer
- Not able to comprehend instructions.
- No proven mutation.
- Less than 10 fibrofolliculomas.
- Planned facial surgery in the treatment period.
- Concomitant disease requiring systemic immunosuppressive treatment
- Concomitant disease requiring facial topical immunosuppressive treatment or facial topical drugs that interfere with rapamycin during trail period or in the 30 days before start trial.
- Tendency to form keloids or hypertrophic scars.
- Drug or alcohol abuse.
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
VU Medical Centre Amsterdam
Amsterdam, 1081 HV
Maastricht University Medical Centre
Maastricht, 6229 HX
About this study
Official title: Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
The purpose of the study is to determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary we evaluate rapamycin safety, formula acceptance and patient satisfaction.
- Conditions
- Birt-Hogg-Dubé Syndrome
- Being tested
- Rapamycin, placebo
- Medical subject headings
- Birt-Hogg-Dube Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RapamycinDrug
Also called Rapamune, Sirolimus
Application of rapamycin 1 mg/ml oral solution to one predefined skin area on one facial half, twice daily 0,25 ml, during 6 months. The other facial half will be similarly treated with a placebo.
placeboDrug
Application of placebo to one predefined skin area on one facial half, during 6 months. The other facial half will be similarly treated with rapamycin 1 mg/ml oral solution
| Study group | Role | What they receive |
|---|---|---|
| rapamycin | Experimental | one facial side rapamycin and one facial side placebo Receives: Drug: Rapamycin; Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.
Measured: 3 and 6 months
Secondary outcomes (1)
Side effects
Measured: 3 and 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2009 (estimated)
- Study startJan 2010
- Primary completionFeb 2011
- Study completionAug 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 Birt-Hogg-Dubé SyndromeStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Maastricht University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Myrovlytis Trust
- Responsible party
- Sponsor
- Investigators
- Maurice van Steensel, Dr., Principal investigator, Department of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00928798
- Sponsor's protocol ID
- 09-2-058
- Status last verified
- Mar 2012
- First submitted
- 25 Jun 2009
- Last updated
- 8 Mar 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Gijezen LM, Vernooij M, Martens H, Oduber CE, Henquet CJ, Starink TM, Prins MH, Menko FH, Nelemans PJ, van Steensel MA. Topical rapamycin as a treatment for fibrofolliculomas in Birt-Hogg-Dube syndrome: a double-blind placebo-controlled randomized split-face trial. PLoS One. 2014 Jun 9;9(6):e99071. doi: 10.1371/journal.pone.0099071. eCollection 2014.
PubMed 24910976
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
- Topical Rapamycin to Treat Fibrofolliculomas in Birt-Hogg-Dubé Syndrome
- ClinicalTrials.gov ID
- NCT00928798
- Acronym / sponsor ID
- 09-2-058
- Conditions
- Birt-Hogg-Dubé Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Maastricht University Medical CenterUniversity, hospital or other
- Collaborators
- Myrovlytis Trust
- Study start
- Jan 2010
- Primary completion
- Feb 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 26 Jun 2009 (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 the study is to determine whether topical application of rapamycin can lead to reduction in size and/or number of fibrofolliculomas in BHD patients and may prevent the growth of new ones. Secondary we evaluate rapamycin safety, formula acceptance and patient satisfaction.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind (participant, investigator)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rapamycin | Experimental | Drug: Rapamycin, Drug: placeboone facial side rapamycin and one facial side placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Minimum age of 18 years.
- At least 10 facial fibrofolliculomas, histologically confirmed.
- Entered in a screening program and free of malignancy as determined during screening (already had a baseline MRI or CT-scan).
- Being able to understand instructions.
- Mutation status must be known.
Key exclusion criteria (14 listed)
- Not capable of informed consent.
- Age under 18 years.
- Pregnancy or failure to comply with contraceptive measures.
- Proven or suspected malignancy of skin or other organs.
- No histological confirmation.
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 |
|---|---|---|
| RapamycinAlso: Rapamune, SirolimusApplication of rapamycin 1 mg/ml oral solution to one predefined skin area on one facial half, twice daily 0,25 ml, during 6 months. The other facial half will be similarly treated with a placebo. | Drug | rapamycin |
| placeboApplication of placebo to one predefined skin area on one facial half, during 6 months. The other facial half will be similarly treated with rapamycin 1 mg/ml oral solution | Drug | rapamycin |
Primary outcome
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area.
Time frame: 3 and 6 months
1 secondary outcome
Side effects
Time frame: 3 and 6 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 Mar 2012.
- Derived from this study: PubMed 24910976Gijezen LM, Vernooij M, Martens H, Oduber CE, Henquet CJ, Starink TM, Prins MH, Menko FH, Nelemans PJ, van Steensel MA. Topical rapamycin as a treatment for fibrofolliculomas in Birt-Hogg-Dube syndrome: a double-blind placebo-controlled randomized split-face trial. PLoS One. 2014 Jun 9;9(6):e99071. doi: 10.1371/journal.pone.0099071. eCollection 2014.
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.
Maurice van Steensel, Dr. principal investigator
Department of Dermatology
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Rapamycin in people with Birt-Hogg-Dubé Syndrome, run by Maastricht University Medical Center at 2 sites in Netherlands.
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 14 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.
Where is the study taking place?
The record lists 2 sites in Netherlands. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Significant regression of lesions (reduction of fibrofolliculoma size and count) in the treated area. (time frame: 3 and 6 months). 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 NCT00928798, 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.