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Sturge- Weber SyndromePhase 2RSWNCT02080624

Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome

A Phase 2 study testing Drug: Topical Rapamycin in people with Sturge- Weber Syndrome, run by Clinica Universidad de Navarra, Universidad de Navarra at 1 site in Spain.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Sturge-Weber syndrome (SWS) is a rare congenital neuro-cutaneous disorder considered as a rare or orphan disease. SWS is characterized by a capillary vascular malformation (CM) localized on the skin of the face, eyes and central nervous system. Read more about this study

Who is it for?
Ages 16 to 65 years
Conditions the study is for
Sturge- Weber Syndrome
What is being tested?
Drug: Topical Rapamycin
Could I receive a placebo?
No, everyone receives an active treatment
How many people
23 participants
Where
1 siteSpain
Study dates
Jan 2011to Dec 2012
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 16 to 65 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis: All patients must have the diagnostic criteria for Sturge-Weber syndrome.
  • Age: patients must be greater than 16 years and less than or equal to 21 years of age at the time of study entry.
  • Capillary malformation: patients must have CM on the face.
  • Investigational drug: Patients must not have received an investigational drug within 3 months.
  • Females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method during the time they are receiving the study drug and for 3 months thereafter. Abstinence is an acceptable method of birth control. Women of childbearing potential will be given a pregnancy test prior to administration of rapamycin and must have a negative pregnancy test.
  • Intellectual capacity to understand the information given and able to comply with the protocol and safety monitoring requirements of the study in the opinion of the investigator.
  • Signed informed consent/assent.

Who cannot join

  • Patients with diagnosis of Sturge-Weber syndrome without facial CM.
  • Patients with another cutaneous disease on the CM area.
  • Patients that will be applying another topical cream on the CM area.
  • Chronic treatment with systemic steroids or another immunosuppressive agent. Patients with endocrine deficiencies are allowed to receive physiologic or stress doses of steroids if necessary.
  • Patients who:
  • have had a major surgery or significant traumatic injury within 2 weeks of start of study drug;
Show all 22Show fewer
  • have not recovered from the side effects of any major surgery (defined as requiring general anesthesia but excluding a procedure for insertion of central venous access), or
  • may require major surgery during the course of the study.
  • Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
  • Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
  • symptomatic congestive heart failure of New York heart Association Class III or IV.
  • unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction within 6 months of start of study drug, serious uncontrolled cardiac arrhythmia or any other clinically significant cardiac disease.
  • severely impaired lung function.
  • uncontrolled diabetes as defined by fasting serum glucose greater than 1.5 upper limit of normal.
  • active (acute or chronic) or uncontrolled severe infections.
  • liver disease such as cirrhosis, chronic active hepatitis or chronic persistent hepatitis.
  • Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (i.e., uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration).
  • A known history of HIV seropositivity or known immunodeficiency.
  • Women who are pregnant or breast feeding.
  • Patients who have received prior treatment with an inhibitor of mammalian target of rapamycin.
  • History of noncompliance to medical regimens.
  • Patients unwilling to or unable to comply with the protocol or who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.

Trial locations and contacts

1 site in 1 country.

Spain1 site

  • Clinica Universidad de Navarra

    Pamplona, Navarre, 31008

Other trials in Spain

About this study

Official title: Phase II, Randomized, Triple Blind, Intra-individually Placebo-controlled Clinical Trial to Assess the Efficacy and Safety of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome.

Sturge-Weber syndrome (SWS) is a rare congenital neuro-cutaneous disorder considered as a rare or orphan disease. SWS is characterized by a capillary vascular malformation (CM) localized on the skin of the face, eyes and central nervous system. Given the localization and the extent of the CM, children with SWS are particularly prone to developing severe psychological problems. The standard treatment for CM is pulsed dye laser (PDL) although in these cases whitening of the lesion is rarely achieved. Combining topical rapamycin, a specific inhibitor of the mammalian target of rapamycin, with PDL is hypothesised to be a good therapeutic option in these patients.

Read the detailed description

Patients with SWS will be treated with 2 sessions of PDL in the lateral part of the CM separated by an interval of 6 weeks and with 1% topical rapamycin or placebo in the superior or inferior half, both applied once a day for 12 weeks. The clinical response will be analyzed using a morphologic and chromatographic computerised system and with spectrometry. Histological response will be evaluated also. For that purpose, we will make 4 biopsies, one in each quadrant (quadrant treated with PDL and placebo, quadrant treated with PDL and rapamycin, quadrant treated only with rapamycin and quadrant treated only with placebo)

Being tested
Drug: Topical Rapamycin
Medical subject headings
Brain Stem Infarctions

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Drug: Topical RapamycinDrug

    Also called topical rapamycin 1%

    After signing this consent form, you will be asked to undergo some screening tests or procedures to find out if you can be in the research study.

    A medical history, which involves questions about your health history, any medications you are taking or plan to take, any allergies and the treatments you received for your CM.

    A physical exam, during which you will be asked about any problems that you might be having. Additionally, your clinician will check your vital signs (blood pressure, heart rate, weight and height). The doctor will also evaluate your performance status, which indicates how much your illness affects your activity level.

    Blood tests, which will be done to make sure your hemogram, triglyceride and cholesterol levels are normal.

    A pregnancy test for females will be done to check that you are not pregnant. If theses tests show that you are eligible to participate in the research study, you will begin the study treatment.

Study groups in NCT02080624
Study groupRoleWhat they receive
RapamycinExperimentalTopical rapamycin applied once a day
Receives: Drug: Drug: Topical Rapamycin

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change from baseline in morphologic, chromatographic and spectrometric scores at week 6

    Change Outcome Measure

    Measured: Baseline, Week 6

  2. Change from baseline in morphologic, chromatographic and spectrometric scores at week 12

    Change Outcome Measure

    Measured: Baseline, Week 12

  3. Change from baseline in morphologic, chromatographic and spectrometric scores at week 18

    Change Outcome Measure

    Measured: Baseline, Week 18

  4. Histological response at 12 weeks.

    Efficacy Outcome Measure

    Measured: 12 weeks

Secondary outcomes (16)

  1. Adverse events at baseline

    Measured: At the beginning of the intervention

  2. Adverse events at 6 weeks

    Measured: 6 weeks after the beginning of the intervention

  3. Adverse events at 12 weeks

    Measured: 12 weeks after the beginning of the intervention

  4. Adverse events at 18 weeks

    Measured: 18 weeks after the beginning of the intervention

  5. Total blood cholesterol level (mg/dL) at baseline.

    Measured: At the beginning of the intervention

  6. Total blood cholesterol level (mg/dL) at 6 weeks.

    Measured: 6 weeks after the beginning of the intervention

  7. Blood concentration of triglycerides (mg/dL) at baseline.

    Measured: At the beginning of the intervention

  8. Blood concentration of triglycerides (mg/dL) at 6 weeks.

    Measured: At 6 weeks after the beginning of the intervention

  9. Blood concentration of hemoglobin (g/dL) at baseline.

    Measured: At the beginning of the intervention

  10. Blood concentration of hemoglobin (g/dL) at 6 weeks.

    Measured: At 6 weeks after the beginning of the intervention

  11. Blood count of leukocytes (number of cells/mL) at baseline.

    Measured: At the beginning of the intervention.

  12. Blood count of leukocytes (number of cells/mL) at 6 weeks.

    Measured: At 6 weeks after the beginning of the intervention.

  13. Blood platelet count (number of platelets/mL) at baseline.

    Measured: At the beginning of the intervention.

  14. Blood concentration of rapamycin (ng/ml) at baseline.

    Measured: At the beginning of the intervention.

  15. Blood concentration of rapamycin (ng/ml) at 6 weeks.

    Measured: At 6 weeks after the beginning of the intervention.

  16. Blood platelet count (number of platelets/mL) at 6 weeks.

    Measured: At 6 weeks after the beginning of the intervention.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Mar 2014 (estimated)
  2. Study startJan 2011
  3. Primary completionSep 2012
  4. Study completionDec 2012

Resources

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
Allocation
Not applicable
Intervention model
Single group
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02080624
Sponsor's protocol ID
RSW2011
Other IDs
2010-024078-20
Acronym
RSW
Status last verified
Oct 2013
First submitted
18 Apr 2013
Last updated
6 Mar 2014
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Drug: Topical Rapamycin in people with Sturge- Weber Syndrome, run by Clinica Universidad de Navarra, Universidad de Navarra at 1 site in Spain.

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 7 inclusion and 22 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 6 Mar 2014. 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 Spain. 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?

Change from baseline in morphologic, chromatographic and spectrometric scores at week 6 (time frame: Baseline, Week 6). The study lists 4 primary outcomes in all. 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 NCT02080624.

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 6 Mar 2014; AzTrials last refreshed its copy on 7 Nov 2026.

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