An Investigational Pilot Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations

There is an unsatisfied medical need for a first-line treatment of lymphatic malformations with a good benefit/risk profile. Based on a patient experience in the institution, the investigators plan to verify whether or not the medication sildenafil has a beneficial effect on lymphatic malformations. The investigators plan to do this by treating patients with lymphatic malformations with the medication sildenafil for a 20 week period. This is an investigator initiated study funded by an Innovations in Patient Care grant and a SPARK grant.

Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.

Investigator(s):

Intervention(s):

  • drug : Sildenafil

Phase: Phase 1/Phase 2

Eligibility

Ages Eligible For Study:

6 Months - 10 Years

Inclusion Criteria

1. Written informed consent(s) for study participation and (where applicable) the use of the participant's images are obtained according to national regulations from the participant's parent(s) or guardian(s) prior to performing any study procedures. 2. The participant is 6 months to 10 years of age at inclusion. 3. The participant weight is at least 8kg. 4. A diagnosis of LM or mixed venous lymphatic malformation involving the skin and subcutaneous tissue and at least 3cm based on clinical and radiographic criteria. 5. LMs may benefit from systemic therapy based on clinical criteria. 6. Females must not be pregnant or breast-feeding. 7. If participant is a child, parent/guardian must be able to follow instructions and must be willing and able to ensure that the subject is present for all required study visits. 8. Subject has no contraindication for use of sildenafil. 9. LMs may involve any part of the body. 10. Subject will have normal results on screening tests (eye exam, blood tests). 11. Subject has no contraindication for MRI examinations, such as metal implants, etc. 12. Subject must not be a smoker.

External Links

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Contact information

Primary Contact:

Andrea Tichy, Ph.D. 650-724-1982

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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