Selenium in the Treatment of Complicated Lymphatic Malformations

This study has been terminated.
(The investigators chose to terminate the study due to participant attrition. Of the limited data evaluable, none of the patients experienced adverse events.)
Sponsor:
Information provided by (Responsible Party):
Michael Kelly, Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT01212965
First received: September 24, 2010
Last updated: April 22, 2013
Last verified: April 2013
  Purpose

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 Intervention Phase
Lymphatic Malformations
Drug: Selenium
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Medical College of Wisconsin:

Primary Outcome Measures:
  • Determine a preliminary rate to oral selenium in adolescents and young adults with symptomatic lymphatic malformations [ Time Frame: pretreatment and at 6 months ] [ Designated as safety issue: No ]
    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 [ Time Frame: throughout duration of study ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome [ Time Frame: throughout duration of study ] [ Designated as safety issue: No ]
    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


Enrollment: 5
Study Start Date: September 2010
Study Completion Date: November 2012
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Selenium
    400 μg once a day for 6 months by mouth
    Other Name: Selenase
  Eligibility

Ages Eligible for Study:   14 Years to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01212965

Locations
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Medical College of Wisconsin
Investigators
Principal Investigator: Michael E Kelly, MD, PhD Medical College of Wisconsin
  More Information

No publications provided

Responsible Party: Michael Kelly, Principal Investigator, Medical College of Wisconsin
ClinicalTrials.gov Identifier: NCT01212965     History of Changes
Other Study ID Numbers: 10/93
Study First Received: September 24, 2010
Last Updated: April 22, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Medical College of Wisconsin:
Selenium
Lymphatic Malformations
Adolescents

Additional relevant MeSH terms:
Congenital Abnormalities
Lymphatic Abnormalities
Lymphatic Diseases
Selenium
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs
Trace Elements
Micronutrients
Growth Substances

ClinicalTrials.gov processed this record on August 21, 2014