- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02080624
Efficacy and Safety Study of Topical Rapamycin Associated With Pulsed Dye Laser in Patients With Sturge-Weber Syndrome (RSW)
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.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Navarra
-
Pamplona, Navarra, Spain, 31008
- Clinica Universidad de Navarra
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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;
- 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.
Study Plan
How is the study designed?
Design Details
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Rapamycin
Topical rapamycin applied once a day
|
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.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change from baseline in morphologic, chromatographic and spectrometric scores at week 6
Time Frame: Baseline, Week 6
|
Change Outcome Measure
|
Baseline, Week 6
|
Change from baseline in morphologic, chromatographic and spectrometric scores at week 12
Time Frame: Baseline, Week 12
|
Change Outcome Measure
|
Baseline, Week 12
|
Change from baseline in morphologic, chromatographic and spectrometric scores at week 18
Time Frame: Baseline, Week 18
|
Change Outcome Measure
|
Baseline, Week 18
|
Histological response at 12 weeks.
Time Frame: 12 weeks
|
Efficacy Outcome Measure
|
12 weeks
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Adverse events at baseline
Time Frame: At the beginning of the intervention
|
At the beginning of the intervention
|
Adverse events at 6 weeks
Time Frame: 6 weeks after the beginning of the intervention
|
6 weeks after the beginning of the intervention
|
Adverse events at 12 weeks
Time Frame: 12 weeks after the beginning of the intervention
|
12 weeks after the beginning of the intervention
|
Adverse events at 18 weeks
Time Frame: 18 weeks after the beginning of the intervention
|
18 weeks after the beginning of the intervention
|
Total blood cholesterol level (mg/dL) at baseline.
Time Frame: At the beginning of the intervention
|
At the beginning of the intervention
|
Total blood cholesterol level (mg/dL) at 6 weeks.
Time Frame: 6 weeks after the beginning of the intervention
|
6 weeks after the beginning of the intervention
|
Blood concentration of triglycerides (mg/dL) at baseline.
Time Frame: At the beginning of the intervention
|
At the beginning of the intervention
|
Blood concentration of triglycerides (mg/dL) at 6 weeks.
Time Frame: At 6 weeks after the beginning of the intervention
|
At 6 weeks after the beginning of the intervention
|
Blood concentration of hemoglobin (g/dL) at baseline.
Time Frame: At the beginning of the intervention
|
At the beginning of the intervention
|
Blood concentration of hemoglobin (g/dL) at 6 weeks.
Time Frame: At 6 weeks after the beginning of the intervention
|
At 6 weeks after the beginning of the intervention
|
Blood count of leukocytes (number of cells/mL) at baseline.
Time Frame: At the beginning of the intervention.
|
At the beginning of the intervention.
|
Blood count of leukocytes (number of cells/mL) at 6 weeks.
Time Frame: At 6 weeks after the beginning of the intervention.
|
At 6 weeks after the beginning of the intervention.
|
Blood platelet count (number of platelets/mL) at baseline.
Time Frame: At the beginning of the intervention.
|
At the beginning of the intervention.
|
Blood concentration of rapamycin (ng/ml) at baseline.
Time Frame: At the beginning of the intervention.
|
At the beginning of the intervention.
|
Blood concentration of rapamycin (ng/ml) at 6 weeks.
Time Frame: At 6 weeks after the beginning of the intervention.
|
At 6 weeks after the beginning of the intervention.
|
Blood platelet count (number of platelets/mL) at 6 weeks.
Time Frame: At 6 weeks after the beginning of the intervention.
|
At 6 weeks after the beginning of the intervention.
|
Collaborators and Investigators
Investigators
- Principal Investigator: Maider Pretel, MD PhD, Clinica Universidad de Navarra
- Principal Investigator: Leyre Aguado, MD PhD, Clinica Universidad de Navarra
- Principal Investigator: Laura Marqués, MD, Clinica Universidad de Navarra
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Ischemia
- Pathologic Processes
- Necrosis
- Cardiovascular Diseases
- Vascular Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Disease
- Brain Ischemia
- Infarction
- Stroke
- Brain Infarction
- Hemangioma
- Neoplasms, Vascular Tissue
- Neurocutaneous Syndromes
- Angiomatosis
- Syndrome
- Klippel-Trenaunay-Weber Syndrome
- Sturge-Weber Syndrome
- Brain Stem Infarctions
- Physiological Effects of Drugs
- Anti-Infective Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Anti-Bacterial Agents
- Antibiotics, Antineoplastic
- Antifungal Agents
- Sirolimus
Other Study ID Numbers
- RSW2011
- 2010-024078-20 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Sturge- Weber Syndrome
-
Johns Hopkins UniversityVascular Birthmarks FoundationCompletedEpilepsy | Sturge Weber SyndromeUnited States
-
University of California, IrvineMainz UniversityCompletedSturge - Weber Syndrome (SWS)United States
-
Wills EyeUniversity of Medicine and Dentistry of New JerseyTerminatedSturge Weber Syndrome | Port-wine MarkUnited States
-
Baylor College of MedicineNovartis PharmaceuticalsWithdrawnSturge Weber SyndromeUnited States
-
Qlaris Bio, Inc.CompletedOcular Hypertension | Glaucoma | Glaucoma Congenital | Sturge-Weber Syndrome (SWS)United States
-
University of Illinois at ChicagoWayne State University; Boston Children's Hospital; Duke University; University... and other collaboratorsRecruitingSturge-Weber SyndromeUnited States
-
Anne Comi, MDJazz Pharmaceuticals; Faneca 66 FoundationCompletedSturge-Weber SyndromeUnited States
-
University Hospital, ToursCompletedPort Wine Stain | Parkes Weber Syndrome | Klippel Trenaunay SyndromeFrance
-
Wayne State UniversityNational Institutes of Health (NIH)Recruiting
-
Hugo W. Moser Research Institute at Kennedy Krieger...Baylor College of Medicine; New York University; National Institute of Neurological... and other collaboratorsActive, not recruitingSturge-Weber SyndromeUnited States
Clinical Trials on Drug: Topical Rapamycin
-
LEO PharmaCompleted
-
The University of Texas Health Science Center at...National Institute on Aging (NIA)CompletedAging | Epigenetics | Inflammatory MediatorsUnited States
-
Nemours Children's ClinicRecruitingVascular AnomalyUnited States
-
Qassim UniversityCairo UniversityCompleted
-
Universidad de AntioquiaINNOVATION CORPORATION FOR THE DEVELOPMENT OF PRODUCTS FOR TROPICAL DISEASES... and other collaboratorsWithdrawnLeishmaniasis, CutaneousColombia
-
Melanoma and Skin Cancer Trials LimitedMonash University; The University of QueenslandNot yet recruitingSkin Cancer | Solid Organ Transplant RecipientsAustralia
-
Novan, Inc.CompletedAcne VulgarisUnited States
-
Stanford UniversityUniversity of Pennsylvania; University of California, Davis; Oregon Health and... and other collaboratorsRecruiting
-
Abramson Cancer Center of the University of PennsylvaniaCompletedCutaneous T Cell LymphomaUnited States