Study of Picibanil (OK432) Sclerotherapy in Children With Macrocystic Lymphatic Malformations

This study is ongoing, but not recruiting participants.

First Received: February 2, 2001 Last Updated: March 28, 2008 History of Changes

Sponsors and Collaborators:	University of Iowa Chugai Pharmaceutical
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00010452

Purpose

OBJECTIVES:

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

Condition	Intervention	Phase
Lymphatic Malformations	Drug: picibanil	Phase II Phase III

Drug Information available for: Picibanil

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study
Official Title:	Treatment of Cystic Hygroma (Lymphangiomas) in Children- Picibanil(OK432) Sclerotherapy-Multicenter Trial

Further study details as provided by FDA Office of Orphan Products Development:

Primary Outcome Measures:

to establish whether OK432 sclerotherapy is an effective form of treatment for lymphatic malformations versus the traditional form of treatment, which is surgical excision. [ Time Frame: indefinate ] [ Designated as safety issue: Yes ]

Enrollment:	150
Study Start Date:	April 2000
Primary Completion Date:	June 2006 (Final data collection date for primary outcome measure)

Intervention Details:

Up to .2 mg per injection, given intralesionally every 6-8 weeks

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.

Eligibility

Ages Eligible for Study:	6 Months to 18 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00010452

Locations

United States, California
Children's Associated Medical Group
San Diego, California, United States, 92123
United States, Colorado
Children's Hospital of Denver
Denver, Colorado, United States, 80218
United States, District of Columbia
Childrens National Medical Center
Washington, District of Columbia, United States
United States, Florida
All Children's Hospital
St. Petersburg, Florida, United States, 33701
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Minnesota
Children's Hospitals and Clinics - Minneapolis
Minneapolis, Minnesota, United States, 55404
United States, New York
SUNY Upstate Medical University
Syracuse, New York, United States, 13210
United States, Oregon
Oregon Health Sciences University
Portland, Oregon, United States
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States
United States, Texas
Texas Pediatric Otolaryngology Center
Houston, Texas, United States, 77030
United States, Virginia
Children's Hospital of the Kings Daughter
Norfolk, Virginia, United States, 23507
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53201
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States, 53792-0001

Sponsors and Collaborators

University of Iowa

Chugai Pharmaceutical

Investigators

Study Chair:

Richard J Smith

University of Iowa

More Information

No publications provided by FDA Office of Orphan Products Development

Additional publications automatically indexed to this study by National Clinical Trials Identifier (NCT ID):

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.

Responsible Party:	University of Iowa Health Care ( Richard JH Smith, MD )
Study ID Numbers:	199/15706, UIHC-FDR001774
Study First Received:	February 2, 2001
Last Updated:	March 28, 2008
ClinicalTrials.gov Identifier:	NCT00010452 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

cystic hygroma
lymphangiomas
lymphatic malformations

Study placed in the following topic categories:

Lymphatic Abnormalities
Lymphatic Diseases
Picibanil
Congenital Abnormalities
Lymphangioma, Cystic

Additional relevant MeSH terms:

Lymphatic Vessel Tumors
Lymphatic Abnormalities
Lymphatic Diseases
Neoplasms
Neoplasms by Histologic Type
Antineoplastic Agents

Therapeutic Uses
Picibanil
Congenital Abnormalities
Pharmacologic Actions
Lymphangioma
Lymphangioma, Cystic

ClinicalTrials.gov processed this record on May 06, 2009