Clinical Trial: Sildenafil for the Treatment of Lymphatic Malformations

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations

Brief Summary:

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


Detailed Summary: Lymphatic malformations (LMs) are localized areas of abnormal development of the lymphatic system that commonly occur in the head and neck of children. LMs are considered a rare or orphan disease which causes complications including pain, ulceration, secondary infection, infiltration of other organs, and death. Current therapies involve surgical excision or methods of chemical or physical destruction of portions of lesions. No therapies are uniformly effective and all have the risk of significant adverse events. We recently witnessed almost complete resolution of a LM lesion in a child who was treated with sildenafil oral therapy for pulmonary arterial hypertension. We have subsequently evaluated additional subjects who improved with sildenafil. The goal of this clinical research trial is to document the benefit or absence of benefit of sildenafil therapy for LMs and identify which type of patient will benefit from sildenafil. This study is a double-blind placebo-controlled trial which involves precise documentation of volume changes associated with therapy or placebo by using MRI segmentation techniques. We will also observe and document the clinical response to sildenafil or placebo using clinical evaluation scores and surveys. The results of the study should identify characteristics of LM lesions which may suggest a beneficial response to sildenafil therapy. Sildenafil has very low risk of side effects in the dosage used in this trial. Documentation of an effective response of LMs to sildenafil will accelerate the interest in, and the ability to understand, the mechanisms of LM formation and treatment.
Sponsor: Stanford University

Current Primary Outcome: Change in lesion volume of the test medication as evaluated by MRI examination. [ Time Frame: 20 weeks ]

Participants will be followed for the duration of the study, an expected average of 20 weeks.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Change in lesion volume estimated using a soft tape measure to measure the length, width, and hemispheric measurement of each of the lymphatic malformations. [ Time Frame: 20 weeks ]
    Participants will be followed for the duration of the study, an expected average of 20 weeks.
  • Subject's evaluation of the change in lesion clinical characteristics. [ Time Frame: 20 weeks ]
    Participants will be followed from baseline to 20 weeks.


Original Secondary Outcome: Same as current

Information By: Stanford University

Dates:
Date Received: January 7, 2015
Date Started: May 23, 2015
Date Completion: December 2022
Last Updated: May 9, 2017
Last Verified: May 2017