Selenium in the Treatment of Complicated Lymphatic Malformations

NCT01212965 | Terminated Phase 1 DMC

• 1 other identifier: 10/93
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

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.

Conditions

Lymphatic Malformations

Keywords

Selenium; Lymphatic Malformations; Adolescents

Outcome Measures

Primary Outcomes (2)

• 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

• Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations

  throughout duration of study

Secondary Outcomes (1)

• Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome

  throughout duration of study

Interventions

Selenium DRUG

400 μg once a day for 6 months by mouth

Also known as: Selenase

Eligibility Criteria

• Age: 14 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Medical College of Wisconsin: lead

Study Sites (1)

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Lymphatic Abnormalities

Interventions

Selenium

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Chalcogens; Elements; Inorganic Chemicals; Minerals

Study Officials

• Michael E Kelly, MD, PhD

  Medical College of Wisconsin

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 24, 2010
• First Posted: October 1, 2010
• Study Start: September 1, 2010
• Primary Completion: September 1, 2011
• Study Completion: November 1, 2012
• Last Updated: April 23, 2013

Record last verified: 2013-04

Locations

US (1)