Clinical Trial: A Study to Evaluate Sildenafil for the Treatment of Lymphatic Malformations

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary: Lymphatic malformations can be challenging to treat. Mainstay interventions including surgery and sclerotherapy are invasive and can result in local recurrence and complications. We sought to assess the effect of 20 weeks of oral sildenafil on reducing lymphatic malformation volume and symptoms in children. Seven children (4 boys, 3 girls; ages 13-85 months) with lymphatic malformations were given oral sildenafil for 20 weeks in this open-label study. The volume of the lymphatic malformation was calculated blindly using magnetic resonance imaging performed before and after 20 weeks of sildenafil. Lymphatic malformations were assessed clinically on weeks 4, 12, 20, and 32. Both the physician and parents evaluated the lymphatic malformation in comparison with baseline. Sildenafil can reduce lymphatic malformation volume and symptoms in some children. ( J Am Acad Dermatol 2014;70:1050-7.)
Sponsor: Stanford University

Current Primary Outcome: Change in Volume of Lymphatic Malformation [ Time Frame: Baseline, 20 weeks ]

Participants were given sildenafil for 20 weeks. Participants weighing more than 20 kg were given 20 mg 3 times daily (60 mg/day). Participants weighing between 8 kg and 20 kg were given 10 mg 3 times daily (30 mg/day).


Original Primary Outcome: Change in lymphatic malformation size [ Time Frame: 12 weeks ]

Current Secondary Outcome:

Original Secondary Outcome:

  • Clinical lesion measurements [ Time Frame: 12 weeks ]
  • Physician and participant assessments of lesional change from baseline [ Time Frame: 12 weeks ]


Information By: Stanford University

Dates:
Date Received: February 3, 2011
Date Started: December 2010
Date Completion:
Last Updated: May 22, 2015
Last Verified: May 2015