Back to resultsSelenium in the Treatment of Complicated Lymphatic Malformations
Primary Purpose
Lymphatic Malformations
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Selenium
Sponsored by
About this trial
This is an interventional treatment trial for Lymphatic Malformations focused on measuring Selenium, Lymphatic Malformations, Adolescents
Eligibility 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
Sites / Locations
- Children's Hospital of Wisconsin
Outcomes
Primary Outcome Measures
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.
Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations
Secondary Outcome Measures
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
Full Information
NCT ID
NCT01212965
First Posted
September 24, 2010
Last Updated
April 22, 2013
Sponsor
Medical College of Wisconsin
1. Study Identification
Unique Protocol Identification Number
NCT01212965
Brief Title
Selenium in the Treatment of Complicated Lymphatic Malformations
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 Type
Interventional
2. Study Status
Record Verification Date
April 2013
Overall Recruitment Status
Terminated
Why Stopped
The investigators chose to terminate the study due to participant attrition. Of the limited data evaluable, none of the patients experienced adverse events.
Study Start Date
September 2010 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
November 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Medical College of Wisconsin
4. Oversight
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lymphatic Malformations
Keywords
Selenium, Lymphatic Malformations, Adolescents
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Selenium
Other Intervention Name(s)
Selenase
Intervention Description
400 μg once a day for 6 months by mouth
Primary Outcome Measure Information:
Title
Determine a preliminary rate to oral selenium in adolescents and young adults with symptomatic lymphatic malformations
Description
Disease will be assessed by volumetric MRI and patient quality of life assessments at 6 months of therapy and compared to pretreatment values.
Time Frame
pretreatment and at 6 months
Title
Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations
Time Frame
throughout duration of study
Secondary Outcome Measure Information:
Title
Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome
Description
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
Time Frame
throughout duration of study
10. Eligibility
Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michael E Kelly, MD, PhD
Organizational Affiliation
Medical College of Wisconsin
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Selenium in the Treatment of Complicated Lymphatic Malformations
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