Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome (NCT03047980) | Clinical Trial Compass
CompletedPhase 2/3
Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome
United States10 participantsStarted 2017-01
Plain-language summary
The purpose of this research study is to gain a preliminary understanding of the safety of sirolimus in Sturge-Weber syndrome (SWS) and determine best outcomes to be used to assess the utility of sirolimus for the treatment of cognitive impairments related to Sturge-Weber syndrome.
Who can participate
Age range3 Years – 31 Years
SexALL
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Inclusion criteria
✓. Male or female patients ages 3 to 31 years of age, inclusive.
✓. Cognitive impairment as defined by the following:
✓. Ability to participate in direct neuropsychological and developmental testing.
✓. English as primary language.
✓. Stable anti-epileptic drugs (no changes in medications except dose for \>3 months).
✓. Adequate renal function. GFR must be greater than 50 ml/min/m2 as determined by the Schwartz Formula for children and MDRD for adults: http://www.nkdep.nih.gov/professionals/gfr\_calculators/index.htm
✓. If female and of child bearing potential, documentation of a negative pregnancy test prior to enrollment determined by a urine test is required. Sexually active pre-menopausal female patients (and female partners of male patients) must use adequate contraceptive measures, excluding estrogen containing contraceptives, while on the study drug. Abstinence will be considered an adequate contraceptive measure.
✓. INR ≤1.5 (Anticoagulation is allowed if target INR ≤ 1.5 on a stable dose of warfarin or on a stable dose of LMW heparin for \>2 weeks.)
Exclusion criteria
✕. Allergy to sirolimus or other rapamycin analogues.
✕. Patients with seizures secondary to metabolic, toxic, infectious or psychogenic disorder, drug abuse or current seizures related to an acute medical illness.
What they're measuring
1
Change in Cognitive Function as Assessed by the National Institute of Health (NIH) Toolbox Cognitive Battery
Timeframe: Baseline and at 6 months on the study drug
. Inability to keep follow-up appointments, maintain close contact with Principal Investigators, and/or complete all necessary studies to maintain safety.
✕. Patients in need of immediate major surgical intervention.
✕. Concurrent severe and/or uncontrolled medical disease, which could compromise participation in the pilot study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration, impaired or restrictive pulmonary function, pneumonitis or pulmonary infiltrates).
✕. 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. Inhaled steroids are allowed.
✕. Known history of HIV seropositivity or known immunodeficiency. Testing is not required unless a condition is suspected.
✕. Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of sirolimus (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A gastric tube or nasogastric tube is allowed.