Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations
A Phase 2 study to evaluate safety and efficacy of sildenafil taken orally to improve or resolve lymphatic malformations in children. Subjects may receive either placebo or treatment in an oral dosage with an open label extension for subjects who received placebo. The study treatment assignment will be randomized in a double blind fashion. MRI examination will evaluate change in lesion volume due to treatment. Other safety and efficacy measures will be taken through the 32-week study duration. Funding Source - FDA OOPD
Stanford is now accepting new patients for this trial. Please contact Elidia C Tafoya, MPH at 650-724-1982 for more information.
Investigator(s):
Intervention(s):
- drug : Sildenafil 20 mg tablets
- other : Placebo tablets (resembling Revatio)
Phase: Phase 2
Eligibility
Ages Eligible For Study:
Inclusion Criteria
Subjects must: - Be legally authorized representative of subjects willing and able to give consent. Assent obtained for subjects 7 - 10 years old. - Be between the ages of 6 months - 10 years of age at the time of entry into the study. - Be at the minimum weight of 8 kg at the time of enrollment. - Be required to have the clinical diagnosis of lymphatic malformation that appears to be over 3 cm in greatest diameter in order to be evaluated for entry. A review of a previous MRI examination may help confirm the entry criteria on subjects selected to come to Stanford for the MRI screening. - Have the lymphatic malformation cause enough disability for the subject that requires them to consider systemic therapy. - For female subjects: must not be pregnant or breast-feeding. - Have a parent or legally authorized representative willing and able to ensure subject is present for all required study visits. - Have a required MRI examination to confirm that the lymphatic malformation is present and is greater than 3 cm in diameter in order for the subjects to receive medication, which happens during the initial screening evaluation portion of the trial. - Have no contraindications for the use of sildenafil. - Have a normal eye examination. - Have normal liver and kidney function. - Have no contraindication to MRI examinations such as metal implants, etc. - Not be a smoker.
External Links
Contact information
Primary Contact:
Elidia C Tafoya, MPH 650-724-1982
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305