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Trial record 5 of 10 for:    "Klippel-Trenaunay-Weber syndrome"

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Clinica Universidad de Navarra, Universidad de Navarra
ClinicalTrials.gov Identifier:
NCT02080624
First received: April 18, 2013
Last updated: March 4, 2014
Last verified: October 2013
  Purpose

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.


Condition Intervention Phase
Sturge- Weber Syndrome
Drug: Drug: Topical Rapamycin
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
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.

Resource links provided by NLM:


Further study details as provided by Clinica Universidad de Navarra, Universidad de Navarra:

Primary Outcome Measures:
  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 6 [ Time Frame: Baseline, Week 6 ] [ Designated as safety issue: No ]
    Change Outcome Measure

  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 12 [ Time Frame: Baseline, Week 12 ] [ Designated as safety issue: No ]
    Change Outcome Measure

  • Change from baseline in morphologic, chromatographic and spectrometric scores at week 18 [ Time Frame: Baseline, Week 18 ] [ Designated as safety issue: No ]
    Change Outcome Measure

  • Histological response at 12 weeks. [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Efficacy Outcome Measure


Secondary Outcome Measures:
  • Adverse events at baseline [ Time Frame: At the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Adverse events at 6 weeks [ Time Frame: 6 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Adverse events at 12 weeks [ Time Frame: 12 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Adverse events at 18 weeks [ Time Frame: 18 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Total blood cholesterol level (mg/dL) at baseline. [ Time Frame: At the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Total blood cholesterol level (mg/dL) at 6 weeks. [ Time Frame: 6 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Blood concentration of triglycerides (mg/dL) at baseline. [ Time Frame: At the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Blood concentration of triglycerides (mg/dL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Blood concentration of hemoglobin (g/dL) at baseline. [ Time Frame: At the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Blood concentration of hemoglobin (g/dL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention ] [ Designated as safety issue: Yes ]
  • Blood count of leukocytes (number of cells/mL) at baseline. [ Time Frame: At the beginning of the intervention. ] [ Designated as safety issue: Yes ]
  • Blood count of leukocytes (number of cells/mL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ] [ Designated as safety issue: Yes ]
  • Blood platelet count (number of platelets/mL) at baseline. [ Time Frame: At the beginning of the intervention. ] [ Designated as safety issue: Yes ]
  • Blood concentration of rapamycin (ng/ml) at baseline. [ Time Frame: At the beginning of the intervention. ] [ Designated as safety issue: Yes ]
  • Blood concentration of rapamycin (ng/ml) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ] [ Designated as safety issue: Yes ]
  • Blood platelet count (number of platelets/mL) at 6 weeks. [ Time Frame: At 6 weeks after the beginning of the intervention. ] [ Designated as safety issue: Yes ]

Enrollment: 23
Study Start Date: January 2011
Study Completion Date: December 2012
Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Rapamycin
Topical rapamycin applied once a day
Drug: Drug: Topical Rapamycin

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 Name: topical rapamycin 1%

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)

  Eligibility

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02080624

Locations
Spain
Clinica Universidad de Navarra
Pamplona, Navarra, Spain, 31008
Sponsors and Collaborators
Clinica Universidad de Navarra, Universidad de Navarra
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
  More Information

No publications provided

Responsible Party: Clinica Universidad de Navarra, Universidad de Navarra
ClinicalTrials.gov Identifier: NCT02080624     History of Changes
Other Study ID Numbers: RSW2011, 2010-024078-20
Study First Received: April 18, 2013
Last Updated: March 4, 2014
Health Authority: Spain: Agencia Española de Medicamentos y Productos Sanitarios

Additional relevant MeSH terms:
Klippel-Trenaunay-Weber Syndrome
Sturge-Weber Syndrome
Brain Stem Infarctions
Syndrome
Angiomatosis
Brain Diseases
Brain Infarction
Brain Ischemia
Cardiovascular Diseases
Central Nervous System Diseases
Cerebrovascular Disorders
Disease
Hemangioma
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Vascular Tissue
Nervous System Diseases
Neurocutaneous Syndromes
Pathologic Processes
Stroke
Vascular Diseases
Everolimus
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antifungal Agents
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents

ClinicalTrials.gov processed this record on November 20, 2014