Selenium in the Treatment of Complicated Lymphatic Malformations

Phase 1

Terminated

• Conditions: Lymphatic Malformations

• Registration Number: NCT01212965
• Lead Sponsor: Medical College of Wisconsin

Brief Summary

The investigators propose a pilot trial to obtain preliminary information regarding the safety and response rate of patients with symptomatic lymphatic malformations treated with oral Selenium. Information obtained in this pilot trial will be used to plan future phase 2 clinical trials.

Hypotheses:

* Selenium will be safe and efficacious in the treatment of adolescents and young adults with symptomatic lymphatic malformations

* Disease response will correlate with serum levels of selenium and blood levels of antioxidants essential to selenium metabolism.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-09
• Study First Submitted: 2010-09-24
• Study First Submitted QC: 2010-09-29
• Study First Posted: 2010-10-01
• Primary Completion: 2011-09
• Study Completion: 2012-11
• Status Verified: 2013-04
• Last Update Submitted: 2013-04-22
• Last Update Posted: 2013-04-23

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• Patients must have clinical and radiographic features consistent with a lymphatic malformation (LM). Patients with combined venous lymphatic malformations (CVLM) will be included if the predominant componet is lymphatic based on MRI. Both macrocystic and microcystic LM will be eligible. Tissue biopsy is required to confirm the presence of an abnormal lymphatic malformation.
• All patients diagnosed with lymphatic malformations between 14 and 30 years of age will be eligible.
• Patients must have lymphatic malformations with potential to cause morbidity including one or more of the following: Lymphedema, Coagulopathy, Chronic Pain, Recurrent Cellulitis (> 1 episodes/year), Ulceration and/or bleeding, Impairment of organ function, Visceral and/or bone involvement, or Disfigurement.
• Patients receiving previous systemic therapy, surgical, debulking, or sclerotherapy will be eligible eight weeks following completion of therapy if they meet all inclusion criteria.
• All patients and/or their parents or legal guardians must sign a written informed consent. All institutional FDA requirements for human studies must be met.

Exclusion Criteria

• Younger than 14 years of age or older than 30 years of age
• Life-threatening complications related to LM
• Patients with preexisting renal, hepatic, or thyroid disorders
• Patients receiving a daily multivitamin supplement or other natural products that include selenium
• Patients that have received previous selenium therapy will not be eligible
• Women who are pregnant or breastfeeding

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Determine a preliminary rate to oral selenium in adolescents and young adults with symptomatic lymphatic malformations	pretreatment and at 6 months	Disease will be assessed by volumetric MRI and patient quality of life assessments at 6 months of therapy and compared to pretreatment values.
Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations	throughout duration of study

Secondary Outcome Measures

Name	Time	Method
Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome	throughout duration of study	Whole blood will be drawn prior to therapy and at specific time intervals after initiating selenium. Serum concentrations of selenium, selenium-dependent tripeptide glutathione and IL-20 will be measured and correlated with outcome

Trial Locations

Locations (1)

Children's Hospital of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States