Clinical trial NCT00010452: Study of Picibanil (OK432) Sclerotherapy in Children With Macrocystic Lymphatic Malformations
Official title: Treatment of Cystic Hygroma (Lymphangiomas) in Children- Picibanil(OK432) Sclerotherapy-Multicenter Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Lymphatic Malformations
Keywords: cystic hygroma
lymphangiomas
lymphatic malformations
Lead sponsor: FDA Office of Orphan Products Development
Sponsor class: FED
First posted: 2001-02-02
Enrollment: 150
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: OBJECTIVES:

I. Determine the efficacy of picibanil sclerotherapy in children with macrocystic lymphangioma.

Detailed description: PROTOCOL OUTLINE: This is a multicenter study. Patients are stratified according to prior treatment (no prior treatment vs prior surgical treatment) and geographic area.

Patients who meet all study criteria are put in the "Immediate Treatment Group." Patients receive an intralesional injection of picibanil (OK432) with the aid of ultrasonography or transillumination for localization of cysts. Treatment repeats every 6-8 weeks for a total of up to 4 injections.

After completion of treatment, patients are followed at 6 months, 1 year, and 2 years.

Completion date provided represents the completion date of the grant per OOPD records

Eligibility criteria: PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

* Diagnosis of macrocystic lymphangioma of the head and/or neck Cystic spaces at least 2.0 mL confirmed by radiographic imaging (MRI or CT) Mixed lymphangiomas (macrocystic and microcystic disease) allowed if macrocystic component comprises at least 50% of the total disease burden
* No mixed hemangioma-lymphangioma lesions
* At least 6 months since prior surgery for lymphangioma

--Patient Characteristics--

* Hematopoietic: No clinically significant hematologic disease No hemodynamic instability
* Hepatic: No clinically significant hepatic disorder
* Renal: No clinically significant renal disease No personal or family history of post-streptococcal glomerulonephritis
* Cardiovascular: No personal or family history of rheumatic heart disease
* Pulmonary: No respiratory failure

Other:

* Not pregnant or nursing
* Negative pregnancy test
* No history of allergy to penicillin
* No concurrent temperature of 100.5 degrees or greater
* No active upper respiratory infection
* No personal or family history of obsessive-compulsive or tic disorders
* No personal or family history of PANDA (pediatric autoimmune neuro- psychiatric disorder associated with streptococcal infections)
* No history of hypersensitivity to iodine, Omnipaque, or gadolinium (if fluoroscopy is considered necessary)
* No history of poor health (including congenital disorders, chronic diseases, or immunologic dysfunction)

Primary outcomes:
measure: to establish whether OK432 sclerotherapy is an effective form of treatment for lymphatic malformations versus the traditional form of treatment, which is surgical excision.
timeFrame: indefinate
Secondary outcomes:
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Children's Associated Medical Group
state: California
city: Denver
country: United States
facility: Children's Hospital of Denver
state: Colorado
city: Washington
country: United States
facility: Childrens National Medical Center
state: District of Columbia
city: St. Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Minneapolis
country: United States
facility: Children's Hospitals and Clinics - Minneapolis
state: Minnesota
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Portland
country: United States
facility: Oregon Health Sciences University
state: Oregon
city: Nashville
country: United States
facility: Vanderbilt University
state: Tennessee
city: Houston
country: United States
facility: Texas Pediatric Otolaryngology Center
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the Kings Daughter
state: Virginia
city: Madison
country: United States
facility: University of Wisconsin Hospital and Clinics
state: Wisconsin
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
Study officials:
affiliation: University of Iowa
name: Richard J Smith
role: STUDY_CHAIR
References:
citation: Smith MC, Zimmerman MB, Burke DK, Bauman NM, Sato Y, Smith RJ; OK-432 Collaborative Study Group. Efficacy and safety of OK-432 immunotherapy of lymphatic malformations. Laryngoscope. 2009 Jan;119(1):107-15. doi: 10.1002/lary.20041.