OK432 (Picibanil) in the Treatment of Lymphatic Malformations

NCT03427619 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 201107741
• Study Type: interventional
• Target: 275
• Locations: 1 country
• Sites: 14

Brief Summary

Standard of care for Lymphatic Malformations has been surgical excision. We have been using OK432/Picibanil (generously supplied by Chugai Pharmaceuticals in Japan) since 1992 with great success for macrocystic disease. The objective of the study was to provide OK-432 immunotherapy to subjects with macrocystic or mixed (\> 50% macrocystic) lymphatic malformations (LMs) and investigate the efficacy and safety of OK 432 as a treatment option in subjects with LMs.

Conditions

Lymphatic Malformations

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Clinical Success at 1 to 6 Months Post-Therapy as Assessed by Imaging

  Clinical success was defined as having either a complete (90% 100%) or substantial (60% 89%) reduction in lymphatic malformation (LM) volume after treatment. Response was determined using post treatment imaging studies at approximately 1 to 6 months after completion of treatment

  1 to 6 Months Post-Therapy

Secondary Outcomes (3)

• Number of Participants With Clinical Response 1 to 6 Months Post-Therapy as Assessed by Imaging

  1 to 6 Months Post-Therapy

• Number of Participants With Investigator-Evaluated Overall Response

  1 to 6 Months Post-Therapy

• Change From Baseline in Lesion Volume

  Baseline and 1 to 6 Months Post-Therapy

Study Arms (1)

OK432 (Picibanil)

EXPERIMENTAL

There is no control in this study. All participants will receive the actual drug -OK432. With each injection they may receive 0.01 to 0.05mg/mL 6-12 weeks apart up to 4 injections total.

Drug: OK432

Interventions

OK432 DRUG

OK432 will be injected at dosage of 0.01 to 0.05 mg/mL 6-12 weeks apart up to 4 injections total.

Also known as: Picibanil

OK432 (Picibanil)

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• To be eligible to receive OK432 immunotherapy
• Patients must be ages 6 months to 17 years
• Patients must have a macrocystic Lymphatic Malformation
• Patients may have had surgical treatment for their Lymphatic Malformation
• Patients must have an imaging study to confirm the diagnosis of a macrocystic or mixed Lymphatic Malformation An MRI is preferred over a CT scan (an ultrasound may be used between injections if warranted, however an MRI or CT should be done pre and post treatment)

You may not qualify if:

• Penicillin allergy
• Women who are pregnant or nursing
• Patients who present with a temperature of 100.5 degrees F or greater
• Patients with mixed hemangioma-lymphangioma lesions
• Patients with a history OR a family history of rheumatic heart disease or post-streptococcal glomerulonephritis
• Patients with hemodynamic instability and respiratory failure
• Patients with a history OR a family history of obsessive-compulsive, tic disorders, or PANDA (pediatric autoimmune neuro-psychiatric disorder associated with streptococcal infections)
• Patients who demonstrate abnormalities in the history, physical examination or laboratory analysis which may indicate significant hepatic, hematologic, or renal disease
• Patients who are not in "good general health" (including patients with congenital disorders, chronic diseases, immunologic dysfunction, transplant recipients)

Sponsors & Collaborators

• Richard JH Smith: lead

Study Sites (14)

Rady Children's Hospital & Health Center San Diego

San Diego, California, 92123, United States

Location

The Children's Hospital of Denver

Denver, Colorado, 80218, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Richard Smith, MD

Iowa City, Iowa, 52242, United States

Location

Spectrum Health-SHMG Ear, Nose, & Throat

Grand Rapids, Michigan, 49546, United States

Location

Children's Hospitals & Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States

Location

SUNY Health Science Center

Syracuse, New York, 13210, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Vanderbilt University Hospital

Nashville, Tennessee, 37232, United States

Location

Children's ENT of Houston

Houston, Texas, 77030, United States

Location

Children's Hospital of the Kings Daughter

Norfolk, Virginia, 23507, United States

Location

University of Wisconsin Hospital & Clinic

Madison, Wisconsin, 53279, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Lymphatic Abnormalities

Interventions

Picibanil

Condition Hierarchy (Ancestors)

Lymphatic Diseases; Hemic and Lymphatic Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Biological Products; Complex Mixtures

Limitations and Caveats

Results are based on a retrospective analysis of source-verified data that include subjects enrolled in the open label study. This study was conducted to provide patients with continued access to OK-432 on a compassionate use basis. Response data were not well documented for everyone. Subjects with missing data included those who were lost to follow up, had incomplete or missing CRFs or imaging studies.

Results Point of Contact

• Title: Dr. Richard Smith
• Organization: University of Iowa

richard-smith@uiowa.edu

319-356-2177

Study Officials

• Richard JH Smith, MD

  University of Iowa

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Model Details: All eligible participants receive the actual drug.

Study Record Dates

• First Submitted: January 10, 2018
• First Posted: February 9, 2018
• Study Start: October 5, 2005
• Primary Completion: April 30, 2018
• Study Completion: April 30, 2018
• Last Updated: November 23, 2021
• Results First Posted: October 28, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (14)