Selenium in the Treatment of Complicated Lymphatic Malformations

Sponsor

Medical College of Wisconsin (Other)

Overall Status

Terminated

CT.gov ID

NCT01212965

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Lymphatic Malformations	Drug: Selenium	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

5 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Pilot Clinical Trial to Estimate the Safety and Efficacy of Selenium in the Treatment of Complicated Lymphatic Malformations in Adolescents and Young Adults

Study Start Date :

Sep 1, 2010

Actual Primary Completion Date :

Sep 1, 2011

Actual Study Completion Date :

Nov 1, 2012

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

14 Years to 30 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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