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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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30 June 2014 |
Main ID: |
EUCTR2010-022583-13-IT |
Date of registration:
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27/04/2011 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA. - EFFECTS STUDY
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Scientific title:
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An open-label, multi-center, expanded access study of RAD001 in patients with subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC). The EFFECTS STUDY: Everolimus For Fast Expanded aCcess in TSC SEGA. - EFFECTS STUDY |
Date of first enrolment:
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06/07/2011 |
Target sample size:
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250 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-022583-13 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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Controlled: no
Randomised: no
Open: no
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product:
Placebo:
Other:
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Phase:
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Countries of recruitment
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Belgium
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Czech Republic
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Germany
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Greece
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Hungary
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Italy
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Spain
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Contacts
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Key inclusion & exclusion criteria
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Inclusion criteria: • 1.Male or female 3 years or older; 2.Clinically definite diagnosis of tuberous sclerosis according to the modified Gomez criteria. Clinically definite diagnosis is defined as either of the following: - Two Major Features from Table 5-1 - One Major Feature plus two Minor Features from Table 5-1; 3.Presence of at least one SEGA lesion identified by MRI or CT (according to local requirements) Note: SEGA lesions are only diagnosed in patients with TSC. They arise in the subependymal layer of the lateral ventricle and are usually located near the foramen of Monro and enhance homogeneously with contrast on MRI with no evidence of surrounding edema; 4. If female and of child-bearing potential, documentation of negative pregnancy test prior to enrollment. Sexually active pre-menopausal female patients (and female partners of male patients) must use adequate contraceptive measures while on study and for up to 8 weeks after ending treatment; 5.Written informed consent according to local guidelines Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria: 1. Patients for whom SEGA-related surgery is required and planned; 2. History of myocardial infarction, angina or stroke related to atherosclerosis; 3. Known impaired lung function (e.g. FEV1 or DLCO = 70% of predicted); 4. Significant hematological or hepatic abnormality (i.e. transaminase levels > 2.5 x ULN or serum bilirubin > 1.5 x ULN, hemoglobin < 9 g/dL, platelets < 80,000/mm3, absolute neutrophil count < 1,000/mm3); 5. Ongoing or active infection at time of enrollment; 6. Patients with Hepatitis B or C; 7. Prior history of organ transplantation; 8.Recent surgery (involving entry into a body cavity or requiring sutures) within one month prior to enrollment; 9. Use of an investigational drug within the 30 days prior to enrollment; 10. Uncontrolled hyperlipidemia: Fasting serum cholesterol > 300 mg/dL OR >7.75 mmol/L AND Fasting triglycerides > 2.5 x ULN; 11. Uncontrolled diabetes mellitus as defined by fasting serum glucose > 1.5 x ULN; 12. Patients with bleeding diathesis or on oral anti-vitamin K medication; 13. Serum creatinine > 1.5 x ULN; 14.Any severe and/or uncontrolled medical conditions which could cause unacceptable safety risks: - Uncontrolled hypercholesterolemia/hypertriglyceridemia; - Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of study drug (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome); 15. Patients with a known hypersensitivity to RAD001 or other rapamycin analogs (sirolimus, temsirolimus), or to its excipients; 16. For the purpose of MRI assessments: - Ferromagnetic metal implants other than those approved as safe for use in MR scanner (e.g., braces, some types of aneurysm clips, shrapnel); Patients suffering from uncontrollable claustrophobia or physically unable to fit into the machine (e.g. obesity); 17. Patients with known history of HIV seropositivity; 18. Inability to attend scheduled clinic visits and noncompliance with scheduled clinic visits required for dose monitoring and adjustment and toxicity management; 19. Female patients who are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are used, they must be continued throughout the study by both sexes.
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
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Health Condition(s) or Problem(s) studied
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Subependymal giant cell astrocytomas (SEGA)associated with Tuberous Sclerosis Complex (TSC) MedDRA version: 13.1
Level: PT
Classification code 10045138
Term: Tuberous sclerosis
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Intervention(s)
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Product Name: everolimus Product Code: RAD001 Pharmaceutical Form: Tablet INN or Proposed INN: everolimus CAS Number: 159351-69-6 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5-
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Primary Outcome(s)
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Main Objective: To evaluate safety of RAD001 in patients with SEGA associated with TSC.
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Primary end point(s): Grade 3 and 4 Adverse Events, Serious Adverse Events.
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Secondary Objective: To evaluate the investigator’s best overall response rate of RAD001 in patients with SEGA associated with TSC.
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Secondary ID(s)
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CRAD001MIC02
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2010-022583-13-DE
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Source(s) of Monetary Support
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Results
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Results available:
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Date Posted:
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Date Completed:
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