Selenium in the Treatment of Complicated Lymphatic Malformations

Lymphatic Malformations Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01212965
• Other study ID #: 10/93
• Status: Terminated
• Phase: Phase 1
• First received: September 24, 2010
• Last updated: April 22, 2013
• Start date: September 2010
• Est. completion date: November 2012

Study information

• Verified date: April 2013
• Source: Medical College of Wisconsin
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 5
• Est. completion date: November 2012
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 14 Years to 30 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphatic Abnormalities
• Lymphatic Malformations

Intervention

Drug:
• Selenium
400 µg once a day for 6 months by mouth

Locations

Country	Name	City	State
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Medical College of Wisconsin

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Determine a preliminary rate to oral selenium in adolescents and young adults with symptomatic lymphatic malformations	Disease will be assessed by volumetric MRI and patient quality of life assessments at 6 months of therapy and compared to pretreatment values.	pretreatment and at 6 months	No
Primary	Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations		throughout duration of study	Yes
Secondary	Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome	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	throughout duration of study	No