Clinical trial NCT01212965: 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
Overall status: TERMINATED
Phase: PHASE1
Conditions: Lymphatic Malformations
Keywords: Selenium
Lymphatic Malformations
Adolescents
Lead sponsor: Medical College of Wisconsin
Sponsor class: OTHER
First posted: 2010-10-01
Enrollment: 5
Sex: ALL
Minimum age (years): 14
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Determine a preliminary rate to oral selenium in adolescents and young adults with symptomatic lymphatic malformations
timeFrame: pretreatment and at 6 months
measure: Determine the safety of oral selenium in adolescents and young adults with symptomatic lymphatic malformations
timeFrame: throughout duration of study
Secondary outcomes:
measure: Correlate selenium drug levels, expression of selenoproteins and IL-20with outcome
timeFrame: throughout duration of study
Other outcomes:
Locations:
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
Study officials:
affiliation: Medical College of Wisconsin
name: Michael E Kelly, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: